P HARMA D EVELOPMENT D IGEST
Issue Date: August 2016
– HEMATOLOGY
Vol. 2 No.8
AN DERSON CON SU LT IN G GROU P
Overview
Reference to any specific commercial product or service does not imply endorsement or recommendation by the Publisher.
The report is divided into two parts:
Top Line Data Section: The topline data is organized by company and provides a quick review of product specifics including;
company name, generic and brand name (only product name is listed for devices), indication, the date and regulatory status of
the product indication (in the European Union, United States), and a brief timeline of significant regulatory and research events
from 2013 forward. Entries are color coded: (Darker shade of green indicates a potential blockbuster. Darker shade of yellow
indicates impending regulatory approval – within six months).
PRODUCT OVERVIEW
Greylock Pharma Development Digest is a surveillance service designed for pharmaceutical industry vendors. It therefore
provides an industry perspective, as it is this perspective that is of most value to vendors. The information contained herein is
primarily gathered and summarized from industry press releases, literature and announcements and is not designed to provide a
balanced perspective. The Publisher does not assume any legal liability or responsibility for the accuracy, completeness, or
usefulness of the information supplied herein, nor any opinion expressed. The Publisher, its agents, and employees will not be
liable for any loss or damage arising directly or indirectly from the possession, publication, use of, or reliance on information
obtained from this report. It is provided in good faith without express or implied warranty.
Green: Entries in dark green indicate an approved product with a large revenue potential. Light green entries are for approved
products with a lower revenue potential.
Yellow: All product entries are yellow until they are approved either in the EU or in the US. There are three shades of yellow:
the palest yellow indicates that there is only clinical trial activity associated with a product; once there is regulatory activity then
it becomes a darker yellow; the darkest indicates a positive CHMP or FDA advisory committee positive opinion indicating
approval is imminent.
Red: A negative clinical trial result or regulatory event has occurred.
Product Detail Section: This information is organized by company and provides a detailed description of the product, including
indication data for prescription drugs, and a more detailed timeline of research and regulatory events from 2013 forward.
© Greylock Pharma Development Digest ®. All rights reserved. Neither this publication nor any part of it may be reproduced,
stored in a retrieval system, or transmitted in any form or by any means, electronic, mechanical, photocopying, recording, or
otherwise, without the prior written permission of Greylock Press LLC. Greylock Pharma Development Digest is a registered
trademark of Greylock Press, LLC.
ISSN 2471-2795
Contact Information: info@greylockpress.com
2
Term
Definition
CMMS NCD
United States (US) Centers for Medicare & Medicaid Services National Coverage Determination
EMA APP
Approved by the European Medicines Agency for marketing within the European Union.
EMA CE
The EMA Conformité Européene Mark indicates that a medical device complies with all of the safety requirements
established by the European Union. The CE Mark is required to market a medical device within the European Union.
EMA CHMP
The EMA Committee for Medicinal Products is the European equivalent of the Food and Drug Administration
Advisory Committee. A positive opinion from the CHMP is the second step in the approval process.
EMA COMP
The EMA Committee for Orphan Medicinal Products adopts a positive opinion recommending a pharmaceutical for
orphan drug status.
EMA DCP
EMA Decentralized Procedure allows companies to obtain the simultaneous authorization of a pharmaceutical in
more than one EU country if it has not yet been authorized in any EU country and it does not fall within the
mandatory scope of the centralized procedure.
EMA MAA
European Medicines Agency – Marketing Authorization Application. This is the first step in obtaining authorization
to market a pharmaceutical within the European Union.
FDA ACR
Food and Drug Administration Advisory Committee Recommendation – drugs and devices must first be
recommended by the advisory committee before final approval by the FDA.
FDA APP
Approved by the FDA to be marketed within the United States
FDA BLA
A Biologics License Application is a request for permission to introduce, or deliver for introduction, a biologic
product into interstate commerce and begins the drug approval process in the US.
FDA BTD
Breakthrough Therapy Designation is granted when a new drug is intended to treat a serious or life-threatening
disease and data indicates that the new drug provides substantial benefits over existing therapy.
FDA CRL
A Complete Response Letter is issued when the FDA requires additional information in the review of an application.
FDA FTD
Fast Track Designation is granted when a drug offers unmet medical needs for life-threatening conditions. The goal is
to grant approval within six months of this designation.
GLOSSARY
Glossary
3
Glossary
Definition
FDA HDE
The Humanitarian Device Exemption is the device version of the orphan drug designation and is granted to devices
designed to treat rare diseases.
FDA IDE
The Investigation Device Exemption is granted to equipment manufacturers prior to marketing authorization. IDE
allows companies to test the product in a limited clinical settings prior to full approval.
FDA IND
An Investigational New Drug application is filed when a pharmaceutical company wishes to transport unapproved
drugs across state lines for the purposes of conducting multi-state clinical trials. Companies must apply for an IND
when a drug is not yet approved for marketing within the United States.
FDA NDA
A New Drug Application begins the process of FDA review of a new drug.
FDA ODD
Orphan Drug Designation is granted for therapies in rare diseases (less than 200,000 cases).
FDA PMA
A Pre-Market Approval application is the first step in the regulatory process for medical devices and allows
manufacturers to test the device in a number of medical centers.
FDA PRD
FDA Priority Review Designation. This designation is given to investigational compounds that treat serious
conditions for which current therapy is inadequate or where a new drug offers a significant potential advancement.
PRD means that the FDA will attempt to take action within 6 months of receipt of the application.
FDA QIPD
QIDP status provides priority review and a five-year extension of market exclusivity following product approval in
the United States.
FDA sNDA
A Supplemental New Drug Application is submitted by a pharmaceutical company for any changes in packaging,
labeling, dosages, ingredients, or new indications to an already approved pharmaceutical product.
UK NICE
The England and Wales National Institute for Health Care Excellence (NICE). NICE looks at pharmaco-economic
data and makes recommendations on use of drugs based on a NICE cost/benefit analysis. NICE recommendation
occur after a product has been approved by the EMA and the UK drug approval authorities.
GLOSSARY
Term
4
Topline Data
Pages 8-37
Detail Data
Abbvie: duvelisib
Page 38
Pages 39-40
AbbVie: ibrutinib
AbbVie: venetoclax, elotuzumab (see BMS)
Acceleron: luspatercept, sotatercept (see Celgene)
Page 41
Ablynx: caplacizumab
Page 42
Alexion: eculizumab
Page 43
Alnylam/Genzyme: ALN-CC5, fitusiran (ALN-AT3)
Ambit: quizartinib (see Daiichi)
Page 44
Amgen: blinatumomab
Page 45
Amgen: carfilzomib
Page 46
Amgen: carfilzomib (continued), darbepoetin alfa, denosumab, romiplostim
Page 47
Ariad: ponatinib
Page 48
Astellas: ASP2215
Astellas: isavuconazole
AstraZeneca: acalabrutinib
AstraZeneca: AZD9150/ISI-STAT3
ASTX: guadecitabine
Page 49
Basilea: isavuconazole (see Astellas)
Baxalta: Advate
Page 50
Baxalta: Adynovate, BAX 817
Page 51
Baxalta: BAX 930, FEIBA NF, HyQvia
Page 52
Baxalta: OBI-1, pegaspargase, rixubis, pacritinib (see CTI Biopharma)
Page 53
Baxalta: Vonvendi (BAX 111)
Page 54
Baxter: Vivia/Amia
TABLE OF CONTENTS
Table of Contents
Bayer: BAY 81-8973, damoctocog, octocog
Page 55
Biogen: rFIXFc (Alprolix)
Page 56
5
Table of Contents
Page 57
BioMarin: BMN270
Boehringer Ingelheim: volasertib
BMS: dasatinib
Page 58
BMS: elotuzumab
Page 59
BMS: ipilimumab, nivolumab
Cellectar: CLR-131
Pages 60
Celgene: azacitidine
Page 61
Celgene: lenalidomide
Pages 62-63
Celgene: luspatercept
Page 64
Celgene: luspatercept (continued), pomalidomide
Page 65
Celgene: sotatercept Coherus: pegfilgrastim biosimilar
netupitant+palonosetron (see Helsinn)
Collegium: oxycodone ER
Chugai: ACE 910 (see Roche),
Page 66
CSL: factor rVIII, factor IX Idelvion
Page 67
CTI Biopharma: pacritinib
Page 68
CTI Biopharma: pixantrone, tosedostat
Page 69
Curis: CUDC-907
Page 70
Daiichi Sankyo: quizartinib
Egalet: Egalet-001
Page 71
Egalet: Egalet-002
Eisai: netupitant+palonosetron, palonosetron (see Helsinn) Eli Lilly: galcanezumab
Genmab:
daratumumab (see J&J Janssen), ofatumumab (see Novartis) Gentium: defitelio (see Jazz)
Geron: imetelstat (see J&J
Janssen) Gilead: entospletinib
Page 72
Gilead: idelalisib
Page 73
Gilead: idelalisib (continued)
Helsinn: anamorelin
GSK: GSK2696273, eltrombopag, ofatumumab (see Novartis)
Helsinn: netupitant+palonosetron, palonosetron
Novartis)
Infinity: duvelisib (see Abbvie)
INSYS Therapeutics: dronabinol
Jazz: defibrotide
TABLE OF CONTENTS
Biogen: rFVIIIFc\
GW Pharma: nabiximols
Imcyte: ponatinib (see Ariad), ruxolitinib (see
Ionis: AZD9150/ISI-STAT3 (see Astra)
J&J Janssen: bortezomib (see Takeda), ibrutinib (see AbbVie), daratumumab
Page 74
Page 75
Page 76
J&J Janssen: daratumumab (cont), imetelstat, siltuximab
Page 77
MSD: fosaprepitant, pembrolizumab
Page 78
Novartis: CTL019, deferasirox,
Page 79
6
Table of Contents
Page 80
Novartis: midostaurin, nilotinib
Page 81
Page 82-83
Novartis: ofatumumab
Novartis: panobinostat, pegfilgrastim biosimilar
Novartis: ruxolitinib
Page 84
Oryzon: ORY-1001 (see Roche)
Novartis: ruxolitinib (continued)
Oxigene: OXi4503
Otsuka: nabiximols (see GW Pharma)
Pfizer: BeneFIX, bosutinib
Page 85
Page 86
Pfizer: gemtuzumab+ozogamicin, inotuzumab+ozogamicin
Page 87
Pfizer/Spark: SPK-9001
Page 88
Prothena: NEOD001
Rigel: fostamatinib ITP
Roche: ACE 910
Page 89
Roche: obinutuzumab
Page 90
Roche: obinutuzumab (continued), ORY-1001, rituximab, venetoclax (see AbbVie)
Page 91
Sandoz: rituximab biosimilar
Page 92
Sanofi: SAR302503
1Seattle Genetics: brentuximab+vedotin (see Takeda), vadastuximab talirine
Spectrum: belinostat
Page 93
Spectrum: melphalan, SPI-2012, vincristine liposome
Page 94
Syros: SY-1425
Page 95
Takeda/Millenium: alisertib
Takeda/Millenium: bortezomib
Page 96
Takeda/Millenium: brentuximab+vedotin
Page 97
Takeda/Millenium: ixazomib, MLN9708
Page 98
Tesaro: rolapitant
Page 99
Triphase: marizomib
TABLE OF CONTENTS
Novartis: eltrombopag
Page 100
Drug List by Indication
Pages 101-105
Product Revenues
Pages 106-107
7
1 – 2 years
to approval
< 1 year
To approval
Approved
Small market
Approved
Large market
Recent negative
event
AbbVie: duvelisib
Baxalta: Vonvendi EU
AbbVie: Venclexta EU
AbbVie: Venclexta US
Abbvie: Imbruvica
CTI: pacritinib US
Ablynx:
caplacizumab
Coherus: pegfilgrastim
biosimilar
Baxalta: Adynovi EU
Alexion: Solaris
Amgen: Blincyto
Gilead: entospletinib
Alnylam: ALN-CC5
Collegium: Xtampza
EU
BMS: Opdivo EU
Amgen: Nplate
Amgen: Kyprolis
Gilead: Zydelig
Alnylam: fitusiran
CTI: pacritinib EU
CSL: Afstyla EU
Baxalta: Advate
Amgen: Aranesp
GW: Sativex
Astellas: ASP2215
CTI: Pixuvri US
Egalet: Arymo US
Baxalta: Adynovate
US
Amgen: Xgeva
Novartis: Arzerra EU
Astra: acalabrutinib
Egalet: Arymo EU
GSK: Strimvelis EU
Baxalta: HyQvia
Ariad: Iclusig
Pfizer: Mylotarg US
Astra: AZD9150
INSYS: Syndros EU
Novartis: pegfilgrastim
EU
Baxalta: Obizur
Astellas: Cresemba
Roche: Gazyva
ASTX: guadecitabine
MSD: Keytruda
Sandoz: rituximab EU
Baxalta: Oncaspar
Biogen: Alprolix
Takeda: alisertib
Baxalta: BAX 817
Novartis: pegfilgrastim
US
Takeda: Ninlaro EU
Baxalta: Vonvendi US
Baxalta: FEIBA NF US
Takeda: Ninlaro: EU
Baxalta: BAX 930
Pfizer: inotuzumab+
ozogamicin
Tesaro: Varubi EU
Baxter: Amia/Vivia
Baxalta: Rixubis
Baxalta: FEIBA NF EU
Spectrum: Beleodaq
EU
Bayer: Kogenate
Bayer: Kovaltry
Bayer: damoctocog
Spectrum: Evomela
EU
Biogen: Eloctate
BMS: Yervoy
BioMarin: BMN270
Spectrum: Marqibo
EU
BMS: Empliciti
BMS: Sprycel
BI: volasertib
BMS: Opdivo US
Celgene: Vidaza
Celgene: luspatercept
Celgene:
Imnovid/Pomalyst
Celgene: Revlimid
Celgene: sotatercept
CTI: Pixuvri EU
Collegium: Xtampza US
Cellectar: CLR-131
Daiichi: Injectafer
CSL: Idelvion
CTI: tosedostat
Helsinn: Akynzeo
CSL: Afstyla US
Curis: CUDC-907
Helsinn: Aloxi
MSD: Emend
TOPLINE SUMMARY - AUGUST
> 2 years
to approval
8
1 – 2 years
to approval
< 1 year
To approval
Approved
Small market
Approved
Large market
Daiichi: quizartinib
INSYS: Syndros US
Novartis:
Revolade/Promacta
Egalet: Egalet-002
Jazz: Defitelio
Pfizer BeneFIX
Eli Lilly: galcanezumab
Janssen: Darzalex
Roche: MabThera
GSK: Strimvelis US
Janssen: Sylvant
Tesaro: Varubi US
Helsinn: anamorelin
Novartis: Jadenu US
INSYS: dronabinol EU
Novartis: Tasigna
Janssen: imetelstat
Novartis: Arzerra US
Novartis: CTL019
Novartis: Farydak
Novartis: Jadenu EU
Novartis: Jakavi
Novartis: midostaurin
Pfizer Bosulif
Oxigene: OXi4503
Spectrum: Beleodaq US
Pfizer: Mylotarg EU
Spectrum: Evomela US
Pfizer: SPK-9001
Prothena: NEOD001
Spectrum: Marqibo US
Rigel: fostamatinib
Takeda: Velcade
Roche:
ACE910/emicizumab
Takeda: Adcetris
Roche: ORY-1001
Takeda: Ninlaro US
Recent negative
event
TOPLINE SUMMARY - AUGUST
> 2 years
to approval
Sandoz: rituximab US
Sanofi: SAR302503
Seattle Genetics:
vadastuximab talirine
Spectrum: SPI-2012
Syros: SY-1425
Takeda: MLN9708
Triphase: marizomib
9
AbbVie
Pg 38
AbbVie/
Janssen
Pg 39-40
Brand
Generic
Name
US
Status
EU
Status
Other
Country
Status
Event Timeline
n/a
duvelisib
16 Jun: Topline data from DYNAMO released
15 May: Ph 1 study presented at ASCO
Imbruvica
ibrutinib
16 Jun: FDA ODD for cGVHD
16 Jun: Updates from RESONATE, RESONATE-2
and HELIOS presented at ASCO
16 May: EMA APP for 1st line therapy
16 May: FDA updates label information and SLL
16 Apr: EMA CHMP for 1st line therapy in CLL
16 Mar: FDA APP for 1st line therapy in CLL
15 Dec: RAY study published in Lancet
15 Dec: Ph 2 NHFL study data presented at ASH
15 Dec: Ph 1/2b PCYC-1119 presented at ASH
15 Dec: RESONATE-2 published in NEJM
15 Nov: FDA sNDA for CLL or SLL
15 Sep: sNDA submitted for treatment-naïve CLL
15 Jul: EMA APP for WM
15 Jun: RESONATE results released
15 May: EMA CHMP for WM
15 May: HELIOS study presented at ASCO
15 Apr: Ph 2 study data published in NEJM
15 Mar: HELIOS study stopped early
15 Jan: FDA APP for WM
14 Dec: RESONATE-17study presented at ASH
14 Oct: EMA APP for MCL and CLL
14 Oct: FDA sNDA for WM
14 Jul: FDA APP for 2nd line CLL
14 Jul: EMA CHMP for CLL and MCL
14 Jul: RESONATE study published in NEJM
14 Apr: sNDA for relapsed/refractory CLL
14 Feb: FDA APP for CLL
14 Jan: Ph 3 RESONATE study ended early
13 Dec: Ph 1b/2 study published in Lancet
13 Nov: FDA APP for MCL
13 Oct: EMA MAA for CLL, SLL & MCL
13 Aug: FDA NDA for CLL, MCL & SLL
FDA APP
CLL, MCL,
WM, SLL
EMA APP
CLL, MCL,
WM
CA: APP
CLL, MCL.
WM
TOPLINE DATA
Company
10
AbbVie
Pg 41
Venclexta
venetoclax
AbbVie
n/a
elotuzumab
US
Status
FDA APP
CLL
EU
Status
EMA MAA
Other
Country
Status
Event Timeline
16 Apr: FDA APP for 2nd line CLL
16 Feb: EMA COMP for AML
16 Jan: FDA BTD for AML
16 Jan: FDA BTD for rrCLL
16 Jan: EMA MAA for CLL
16 Jan: FDA NDA & PRD for CLL
15 Dec: Ph 1 study data published in NEJM
15 Dec: Ph 2 study data presented at ASH
15 Aug: Topline data from M13-982 released
15 Jun: Updated Ph 1b data released
15 May: FDA BTD for CLL
15 May: Two Ph 1b studies presented at ASCO
TOPLINE DATA
Company
Brand
Generic
Name
SEE BRISTOL-MYERS SQUIBB
16 May: Post-hoc analysis of TITAN presented at
ESH
16 Feb: TITAN study published in NEJM
14 Dec: Ph 2 TITAN study presented at ASH
14 Jun: Topline data from Ph 2 TITAN study
released
Ablynx
Pg 42
n/a
caplacizumab
Acceleron
n/a
luspatercept
SEE CELGENE
Acceleron
n/a
sotatercept
SEE CELGENE
FDA ODD
EMA COMP
11
Brand
Generic
Name
Alexion
Pg 43
Solaris
eculizumab
Alnylam/
Genzyme
Pg 44
n/a
ALN-CC5
Alnylam/
Genzyme
Pg 44
Ambit
n/a
fitusiran
ALN-AT3
n/a
quizartinib
US
Status
FDA APP
PNH, aHUS
EU
Status
EMA APP
PNH, aHUS
Other
Country
Status
JP: APP
aHUS
Event Timeline
14 Dec: Studies C10-003 & 004 presented at ASH
14 Nov: UK NICE recommends use for all aHUS
14 Nov: 1-year update of a prospective study
presented at ASN Kidney Week
14 Jun: Supplemental analysis of Ph 2 studies
presented at ERA-EDTA
14 May: FDA sBLA amended for long-term aHUS
14 Mar: UK NICE positive assessment for aHUS
13 Nov: Ph 2 prospective studies presented at ASN
13 Sep: Japan APP for aHUS
TOPLINE DATA
Company
16 Jun: Part C of a Ph 1/2 study presented at ESH
13 Dec: Ph 1/2 MDS study presented at ASH
FDA ODD
EMA COMP
15 Dec: Ph 1 study data presented at ASH
15 Jun: Ph 1 study presented at ISTH
15 Jan: Ph 1 study presented at GCC
14 Dec: Ph 1 study presented at ASH
14 Aug: EMA COMP for hem A and hem B
14 May: Topline data from a Ph 1 study presented at
WFH
13 Aug: FDA ODD for hem A and hem B
SEE DAIICHI
12
Amgen
Pg 45
Amgen
Pgs 46-47
Brand
Generic
Name
Blincyto
blinatumomab
Kyprolis
carfilzomib
US
Status
FDA APP
PH- ALL
FDA APP
MM
EU
Status
EMA APP
EU: PH- ALL
EMA APP
MM
Other
Country
Status
Event Timeline
CA: APP 2015
ALL
16 Jun: TOWER study presented at ESH
16 May: FDA PRD for pediatric PH- ALL
16 Mar: FDA sBLA for pediatric PH- ALL
16 Apr: Ph 1 dosage-escalation study published in
JCO
16 Feb: TOWER study ended early for efficacy
15 Dec: Data from 3 Ph 2 studies presented at ASH
15 Nov: EMA APP PH-ALL (conditional)
15 Sep: EMA CHMP PH-ALL
15 Jul: Ph 2 safety and efficacy data released
14 Dec: 211 Study presented at ASH
14 Dec: FDA APP for PH- ALL
14 Oct: FDA PRD for PH-ALL
14 Sep: FDA BLA for PH-ALL
CA: APP 2016
Q1
MM
16 Jun: EMA APP for MM in combination with
dexamethasone
16 Jun: Ph 1/2 study published in Blood
16 Jun: Post-hoc analysis of ASPIRE and subanalysis of ENDEAVOR presented at ESH
16 Jun: ASPIRE study presented at ASCO
16 May: EMA CHMP for relapsed MM
16 May: K-Gem study published in JME
16 Jan: FDA APP for rrMM
15 Dec: ENDEAVOR study published in Lancet
15 Dec: EMA sMAA for relapsed MM
15 Nov: EMA APP for MM 2nd line therapy
15 Sep: EMA CHMP for MM
15 Sep: FDA sNDA for relapsed MM
15 Jul: FDA APP for sNDA of MM
15 Mar: ENDEAVOR study results released
15 Jan: EMA MAA & FDA sNDA for MM
14 Dec: ASPIRE published in NEJM
14 Aug: Topline results for FOCUS released
TOPLINE DATA
Company
13
Brand
Generic
Name
US
Status
EU
Status
Other
Country
Status
Amgen
Pg 47
Aranesp
darbepoetin
alfa
FDA APP
anemia
EMA APP
anemia
Amgen
Pg 47
Xgeva
denosumab
FDA APP
bone metastases,
GCTB, HCM
EMA APP
bone
metastases
CA: APP
bone metastases
Amgen
Pg 47
Nplate
romiplostim
FDA APP
ITP
EMA APP
ITP
CA: APP
ITP
Event Timeline
16 Feb: Topline data from ARCADE released
14 Dec: FDA APP for HCM
16 Jul: Ph 3 study published in Lancet
TOPLINE DATA
Company
14
US
Status
FDA APP
CML,
PH+ ALL
EU
Status
EMA APP
CML,
PH+ ALL
Other
Country
Status
CA: APP 2015
CML,
PH+ ALL
Event Timeline
16 Jun: Long term follow-up data from PACE
presented at ESH
16 Apr: PACE study published in Blood
16 Jan: Japanese NDA submission for CML and
Ph+ ALL
15 May: 4 year Ph 1 data presented at ASCO
14 Dec: Ph 2 PACE study presented at ASH
14 Oct: EMA PRAC clears ponatinib
13 Dec: US sales resumed
13 Nov: EMA CHMP to continue sales in the EU
13 Oct: Sales in US temporarily suspended
13 Oct: EPIC study discontinued early
Ariad
Pg 48
Iclusig
ponatinib
Astellas
Pg 48
n/a
ASP2215
15 May: Data from a Ph 1/2 study presented at
ASCO
Astellas/
Basilea
Pg 49
Cresemba
isavuconazole
15 Oct: EMA APP for Asp. and Mucor.
15 Jul: EMA CHMP
15 Jul: Topline data from ACTIVE study
released
15 Mar: FDA APP
15 Jan: FDA ACR
14 Nov: FDA ODD for candidiasis
14 Jul: EMA MAA
14 Jul: FDA NDA and QIDP for candidiasis
14 May: SECURE study presented at ECCMID
14 Feb: FDA QIDP mucormycosis
13 Dec: FDA QIDP aspergillosis
13 Nov: FDA ODD
13 Sep: Topline data from SECURE study
released
AstraZeneca
Pg 49
n/a
acalabrutinib
FDA APP
aspergillosis
mucormycosis
EMA APP
aspergillosis
mucormycosis
EMA COMP
16 Feb: EMA COMP for CLL, SLL, MCL &
WM
15 Dec: Ph 1/2 study published in NEJM
TOPLINE DATA
Company
Brand
Generic
Name
15
AstraZeneca
Pg 50
n/a
AZD9150/
ISI-STAT3
ASTX
Pg 50
n/a
guadecitabine
Basilea
Cresemba
isavuconazole
Baxalta
Pg 50
Advate
US
Status
EU
Status
Other
Country
Status
Event Timeline
15 Oct: FDA ODD for AML
15 Aug: Ph 1 study published in Lancet
Oncology
14 Jun: Ph 2 study presented at ESH
FDA ODD
TOPLINE DATA
Company
Brand
Generic
Name
SEE ASTELLAS
FDA APP
hem A
EMA APP
hem A
14 Apr: FDA APP BAXJECT III system
14 Feb: AHEAD study presented at EAHAD
EMA MAA
16 Apr: APP in Japan
16 Mar: EMA MAA for hem A
16 Feb: FDA sBLA for children <12 years and in
surgical settings
15 Dec: Topline date from a Ph 3 study released
15 Nov: FDA APP for hem A
15 Jul: A Ph 2/3 study published in Blood
15 Feb: Ph 3 study presented at EHAD
14 Dec: FDA BLA for hem A
14 Aug: Topline Ph 3 results released
13 Nov: Enrollment for Ph 3 trial complete
Baxalta
Pg 51
Adynovate
Adynovi
Advate
PEGylated
(Bax 855)
Baxalta
Pg 51
n/a
BAX 817
15 Mar: Results from Ph 3 study released
Baxalta
Pg 52
n/a
BAX 930
16 May: Ph 1 data released at ISTH
Baxalta
Pg 52
FEIBA NF
FDA APP
hem A
FDA APP
hem A, hem B
JP: APP
hem A
13 Dec: FDA APP for prophylactic use
16
Baxalta
Pg 52
HyQvia
US
Status
FDA APP
PI
EU
Status
EMA APP
PI, SI
15 Nov: EMA APP for acquired hem A
15 Oct: Canadian APP for acquired hem A
15 Jul: EMA CHMP for acquired hem A
14 Oct: FDA APP for acquired hem A in adults
13 Dec: FDA BLA for acquired hem A
13 Dec: Ph 2/3 study of hem A presented at ASH
Obizur
OBI-1
FDA APP
hem A
EMA APP
hem A
Baxalta
Pg 53
Oncaspar
pegaspargase
FDA APP
ALL
EMA APP
ALL
Baxalta
n/a
pacritinib
Rixubis
Baxalta
Pg 54
Vonvendi
von
Willebrand
factor
(Bax 111)
Baxter
Pg 54
Amia
Vivia
Event Timeline
16 May: EMA CHMP for pediatric PI & SI
14 Sep: FDA APP for PI
14 Jul: FDA ACR for PI
14 May: FDA extends review period for BLA
13 Dec: FDA BLA (amended) for PI
Baxalta
Pg 53
Baxalta
Pg 53
Other
Country
Status
CA: APP 2015
hem A
TOPLINE DATA
Company
Brand
Generic
Name
16 Jan: EMA APP for ALL
15 Nov: EMA CHMP for ALL
SEE CTI BIOPHARMA
FDA APP
hem B
FDA APP
VWD
FDA APP
ESKD
EMA APP
hem B
14 Dec: EMA APP for pediatric and adult hem B
14 Sep: FDA APP for children with hem B
13 Dec: FDA sNDA for hem B in children
13 Oct: EMA MAA for hem B
EMA COMP
15 Dec: FDA APP for VWD
15 Aug: Ph 3 study data published in Blood
15 Apr: Ph 3 data presented at HTRS
14 Dec: FDA BLA for VWD
14 Apr: Topline Ph 3 data released
EMA CE
ESKD
15 Oct: FDA APP for ESKD
14 Jun: Data from two Ph 3 studies presented at
ERA-EDTA
13 Dec: EMA CE for ESKD
17
Bayer
Pg 55
Kovaltry
BAY
81-8973
Bayer
Pg 55
n/a
damoctocog
Bayer
Pg 55
Kogenate FS
Kogenate
Octocog
Biogen
Pg 56
Alprolix
rFIXFc
US
Status
FDA APP
hem A
EU
Status
EMA APP
hem A
Other
Country
Status
CA: APP
hem A
Event Timeline
16 Mar: Approved in Japan for hem A
16 Mar: FDA APP for hem A
16 Feb: EMA APP for hem A prophylaxis
16 Feb: Approved in Canada for hem A
15 Dec: EMA CHMP for hem A
15 Jun: MAA for hem A in Japan
14 Dec: Submission of an FDA BLA for hem A
14 Dec: EMA MAA for hem A
TOPLINE DATA
Company
Brand
Generic
Name
14 May: PROJECT presented at WFH
FDA APP
hem A
FDA APP
hem B
EMA APP
hem A
EMA APP
hem B
CA: APP
Hem A
14 Dec: SPINART study published in Blood
14 May: FDA APP for hem A in adults
13 Dec: EMA CHMP opinion on FVIII inhibitors
CA: APP
hem B
16 May: EMA APP for hem B
16 Feb: EMA CHMP for hem B
15 Dec: Post hoc analysis of B-LONG presented
at ASH
15 Aug: B-YOND study presented at NHF
15 Jun: EMA MAA for hem B
15 Feb: Topline results from Kids B-LONG
released
14 Mar: FDA APP for hem B
14 Mar: Approved in Canada for hem B
13 Dec: FDA asks for more data and extension
18
Brand
Generic
Name
US
Status
EU
Status
Other
Country
Status
Event Timeline
Biogen
Pg 57
Eloctate
Elocta
rFVIIIFc
FDA APP
hem A
EMA APP
hem A
15 Dec: Post hoc analysis of ASPIRE presented
at ASH
15 Nov: EMA APP for hem A
15 Sep EMA CHMP positive opinion for hem A
15 Aug: ASPIRE study published in
Haemophilia
14 Oct: EMA MAA for hem A
14 Jun: FDA APP for hem A
14 Apr: Topline data for Kids A-LONG released
13 Nov: A-LONG study published in Blood
BioMarin
Pg 58
n/a
BMN270
FDA ODD
EMA COMP
16 Mar: EMA COMP for severe hem A
16 Mar: FDA ODD
Boehringer
Ingelheim
Pg 58
n/a
volasertib
FDA ODD
TOPLINE DATA
Company
14 Jul: Ph 2 study was published in Blood
14 Apr: FDA ODD & EMA COMP for AML
13 Sep: FDA BTD for AML
19
BMS
Pg 58
Sprycel
dasatinib
US
Status
FDA APP
PH+ ALL, PH+
CML
EU
Status
16 Jul: JCO published 5-year follow-up data
from DASISSION
13 Dec: DASISION study presented at ASH
16 Jun: Ph 2 study published in Blood
16 May: EMA APP for MM
16 Feb: EMA CHMP for MM 2nd line therapy
15 Dec: 3-year follow-up of ELOQUENT-2
presented at ASH
15 Nov: FDA APP for MM
15 Sep: FDA PRD for MM
15 Jul: EMA MAA for MM
15 Jun: ELOQUENT-2 published in NEJM
14 May: FDA BTD for MM
Empliciti
elotuzumab
FDA APP
MM
EMA APP
MM
BMS
Pgs 60
Yervoy
ipilimumab
FDA APP
melanoma
EMA APP
melanoma
Opdivo
nivolumab
Cellectar
Pg 61
n/a
CLR-131
Celgene
Pg 61
Vidaza
azacitidine
FDA APP
HL
Event Timeline
EMA APP
PH+ ALL,
PH+ CML
BMS
Pg 59
BMS
Pg 60
Other
Country
Status
EMA MAA
CA:APP
mM
TOPLINE DATA
Company
Brand
Generic
Name
16 Jul: Independent Ph 1b study published in
NEJM
16 Jun: CHECKMATE-205 presented at ESH
16 May: FDA APP for HL
16 Apr: FDA sBLA for HL
16 Apr: EMA MAA for HL
14 Dec: CHECKMATE-039 published in NEJM
14 May: FDA BTD for HL
16 Jan: Topline Ph 1 data released
FDA APP
MDS, CMML,
RA
EMA APP
MDS, AML,
CMML
15 Oct: EMA APP for AML (expanded)
15 Sep: EMA CHMP for AML (expanded)
13 Dec: Ph 1/2 study presented at ASH
20
Celgene
Pgs 62-63
Revlimid
lenalidomide
US
Status
FDA APP
MM, MDS, MCL
EU
Status
EMA APP
MM, MDS,
MCL
Other
Country
Status
Event Timeline
16 Jul: EMA APP for MCL
16 Jun: Meta-analysis presented at ASCO
16 May: (see BMS) EMA APP of elotuzumab in
combination with lenalidomide
16 Jan: Lenalidomide included in a new
indication for carfilzomib
15 Dec: Approved in Japan as 1st line for MM
15 Nov: Ph 2 study published in NEJM
15 Jun: SPRINT study update presented at EHA
15 Jun: FIRST study update presented at EHA
15 Feb: EMA APP for MM
15 Feb: FDA APP for MM (expanded)
14 Dec: (see Amgen: carfilzomib) ASPIRE
STUDY published in NEJM
14 Dec: EMA CHMP for MM
14 Dec: SPRINT study presented at ASH
14 Dec: Ph 3 study presented at ASH
14 Sep: FIRST study published in NEJM
14 Sep: Ph 3 study data published in NEJM
14 Aug: Ph 2 study published in JCO
13 Dec: Retrospective analysis of MDS presented
at ASH
13 Aug: The PETHEMA/GEM study published
in NEJM
13 Jul: The ORIGIN study discontinued
TOPLINE DATA
Company
Brand
Generic
Name
21
Celgene/
Acceleron
Pgs 64-65
Brand
Generic
Name
n/a
luspatercept
US
Status
EU
Status
Other
Country
Status
Event Timeline
FDA FTD
16 Jun: Two MDS Ph 2 studies presented at ESH
16 Jun: Two BTH Ph 2 studies presented at ESH
15 Dec: Ph 2 BTH study presented at ASH
15 Dec: FDA FTD for MDS
15 Dec: Ph 2 MDS study presented at ASH
15 Jun: Two Ph 2 studies presented at EHA
15 May: FDA FTD for BTH
15 May: PACE-MDS presented at ISMDS
14 Dec: Interim Ph 2 data presented at ASH
16 May: Ph 2 study published in Blood
14 Feb: Approved in Canada for MM
13 Dec: Retrospective analysis of MM-003
presented at ASH
13 Sep: MM-003 study published in Lancet
13 Aug: EMA APP for MM
Celgene
Pg 65
Pomalyst
Imnovid
Pomalidomide
FDA APP
MM
Celgene/
Acceleron
Pg 66
n/a
sotatercept
FDA ODD
Coherus
Pg 66
n/a
pegfilgrastim
16 Jul: Topline data on a pharmacokinetic/
pharmacodynamic study released
Collegium
Pg 66
Xtampza
oxycodone
16 Apr: FDA APP for severe pain
15 Nov: FDA APP tentative approval pain
15 Sep: FDA ACR for severe pain.
15 Aug: Ph 3 study published in Pain
15 Jun: Ph 3 study published in Pain Medicine
14 Dec: FDA NDA for severe pain
Chugai
n/a
ACE-910
Chugai
Akynzeo
neupitant/
palonosetron
FDA APP
pain
EMA APP
US: MM
CA: APP 2014
MM
TOPLINE DATA
Company
14 Nov: Interim Ph 2 data presented at ASN
14 Apr: Interim Ph 2 data presented at NKF
13 Dec: Interim Ph 2 data presented at ASH
SEE ROCHE
SEE HELSINN
22
Company
CSL Behring
Pg 67
CTI
Biopharma
Pg 68
Afstyla
rFVIII
Idelvion
Factor IX
n/a
pacritinib
US
Status
FDA APP
hem A
FDA APP
hem B
FDA NDA
(withdrawn)
EU
Status
EMA MAA
EMA APP
hem B
EMA COMP
Other
Country
Status
Event Timeline
16 May: FDA APP for adult and pediatric hem A
15 Dec: EMA MAA for hem A
15 Jul: FDA BLA for hem A
15 Jun: Ph 3 AFFINITY study presented at ISTH
16 May: EMA APP for hem B
16 May: FDA grants 7 years marketing
exclusivity
16 Mar: FDA APP for hem B
16 Apr: PROLONG-9FP published in Blood
16 Feb: EMA CHMP for hem B
15 Dec: Ph 3 PROLONG-9FP presented at ASH
15 Jun: Ph 3 study presented at ISTH
15 Mar: EMA MAA for hem B
14 May: Two Ph 3 studies presented at WFH
14 Feb: FDA BLA for hem B
TOPLINE DATA
CSL Behring
Pg 67
Brand
Generic
Name
16 Jun: Updated data from PERSIST presented at
ASCO
16 Feb: FDA places hold on clinical trials and
NDA withdrawn
16 Jan: FDA NDA for myelofibrosis completed
15 Dec: Post hoc analysis of PERSIST-1
presented at ASH
15 Nov: FDA NDA for myelofibrosis
15 May: PERSIST-1 presented at ASCO
15 Mar: Ph 2 study published in Blood
15 Mar: Topline data from PERSIST-1 released
14 Dec: Ph 3 data presented at ASH
14 Dec: Analysis of PERSIST-2 study presented
at ASH
14 Jun: FDA FTD for MF
13 Dec: Two Ph 2 studies presented at ASH
23
US
Status
EU
Status
FDA FTD
EMA APP
NHL
Other
Country
Status
Event Timeline
14 Jan: UK NICE recommends funding
13 Oct: UK NICE rejects for 2nd time
CTI Biopharma
Pg 69
Pixuvri
pixantrone
CTI Biopharma
Pg 69
n/a
tosedostat
15 Dec: Ph 2 study data presented at ASH
15 Jun: Ph 2 data presented at EHA
14 Jan: FDA partial hold lifted
13 Dec: Two Ph 2 studies presented at ASH
Curis
Pg 70
n/a
CUDC-907
16 Apr: Ph 1 data published in Lancet Oncology
15 Apr: FDA ODD for DLBCL
15 Dec: Ph 1 study data presented at ASH
15 May: Ph 1 study data presented at ASCO
14 Apr: Preliminary Ph 1 study data presented at
AACR
Daiichi/Ambit
Pg 71
n/a
quizartinib
FDA ODD
TOPLINE DATA
Company
Brand
Generic
Name
14 Jun: Ph 2 study data presented at ASCO and
EHA
13 Dec: Ph 2b study data presented at ASH
24
US
Status
EU
Status
Other
Country
Status
Event Timeline
FDA NDA
16 May: A Ph 2/3 HAP study presented at
AAPS
15 Dec: FDA NDA
15 Jun: Topline results from study 067-EG-011
announced
14 Feb: FDA FTD
FDA FTD
16 Mar: Topline study results released
15 Jun: Topline data from an alcoholinteraction study released
14 Feb: FDA FTD
Egalet
Pg 71
Arymo
Egalet-001
Egalet
Pg 72
n/a
Egalet-002
Eli Lilly
Pg 72
n/a
galcanezumab
Eisai
Akynzeo
neupitant/
palonosetron
SEE HELSINN
Eisai
Aloxi
palonosetron
SEE HELSINN
Genmab
Arzerra
ofatumumab
SEE NOVARTIS
Genmab
Darzalex
daratumumab
SEE J&J JANSSEN
Gentium
Defitelio
defibrotide
SEE JAZZ
Geron
imetelstat
SEE J&J JANSSEN
Gilead
Pg 72
n/a
entospletinib
TOPLINE DATA
Company
Brand
Generic
Name
16 May: Ph 2 study published in Blood
15 May: Ph 2 study published in Blood
25
Brand
Generic
Name
Gilead
Pgs 73-74
Zydelig
idelalisib
GSK
Revolade
eltrombopag
GSK
Pg 73
Strimvelis
GSK2696273
GSK
Arzerra
ofatumumab
GW Pharma
Pg 74
Sativex
nabiximols
Helsinn
Pg 74
n/a
anamorelin
Helsinn
Pg 75
Akynzeo
netupitant/
palonosetron
US
Status
FDA APP
CLL, FL. SLL
EU
Status
EMA APP
CLL, FL
Other
Country
Status
CA: APP 2015
FL, CLL
Event Timeline
16 Jul: Ph 2 study published in Blood
15 Dec: Interim analysis of STUDY 115
presented at ASH
15 Nov: STUDY 115 ended early
15 May: STUDY 119 presented at ASCO
14 Dec: STUDY 101-09 and STUDY 116
presented at ASH
14 Sep: EMA APP for CLL and FL
14 Jul: EMA CHMP for CLL and FL
14 Jul: FDA APP for CLL, FL, SLL
14 Mar: STUDY 101-09 and STUDY 116
published in NEJM
13 Dec: FDA NDA for CL
13 Dec: STUDY 101-09 presented at ASH
13 Dec: STUDY 116 analysis presented at ASH
13 Oct: STUDY 116 halted early due to high
efficacy in CLL patients
13 Sep: FDA NDA for iNHL
TOPLINE DATA
Company
SEE NOVARTIS
EMA CHMP
16 Apr: EMA CHMP for ADA-SCID
SEE NOVARTIS
15 Oct: Topline data from 2 Ph 3 studies released
15 Jan: Topline Ph 3 study data released
14 Apr: FDA FTD for pain in cancer
FDA FTD
14 Nov: ROMANA 1 & 2 study data released
FDA APP
CINV
EMA APP
CINV
15 Jun: EMA APP for CINV
14 Oct: FDA APP for CINV
14 Jan: EMA MAA for CINV
13 Dec: FDA NDA for CINV
26
Brand
Generic
Name
US
Status
EU
Status
Helsinn
Pg 75
Aloxi
palonosetron
FDA APP
CINV
EMA APP
CINV
Incyte
ponatinib
SEE ARIAD
Incyte
Jakafi
ruxolitinib
SEE NOVARTIS
Infinity
duvelisib
SEE ABBVIE
INSYS
Pg 75
Syndros
dronabinol
Ionis Pharma
AZD9150
Company
Defitelio
defibrotide
Event Timeline
15 May: EMA APP for children aged ≥1 month
15 Jan: EMA CHMP for children aged ≥1 month
14 May: FDA APP for children aged ≥1 month
TOPLINE DATA
Jazz
Pg 76
Other
Country
Status
16 Jul: FDA APP for CINV
15 Jun: FDA NDA for weight loss and nausea
14 Oct: FDA CRL
14 Aug: FDA NDA for weight loss and nausea
FDA APP
CINV
SEE ASTRA
FDA APP
VOD
EMA APP
VOD
16 Mar: FDA APP for VOD
16 Feb: Ph 3 study published in Blood
15 Sep: FDA PRD for VOD
14 Dec: FDA NDA & FTD for severe VOD
13 Oct: EMA APP for severe VOD
27
J&J Janssen
Pgs 76-77
Darzalex
daratumumab
US
Status
FDA APP
MM
EU
Status
EMA APP
MM
Other
Country
Status
Event Timeline
16 Jul: FDA BTD
16 Jul: Pooled data from GEN501 & SIRIUS
published in Blood
16 Jun: POLLUX presented at EHA
16 Jun: CASTOR presented at ASCO
16 May: EMA APP for MM
16 May: POLLUX ended early and presented at
EHA
16 Apr: Ph 2 SIRIUS study published in Lancet
16 Apr: EMA CHMP for MM
16 Mar: Topline data from CASTOR released
15 Nov: FDA APP for MM
15 Sep: EMA MAA for MM
15 Sep: FDA PRD for MM
15 Aug: GEN501 study published in NEJM
15 Jun: FDA BLA for MM
15 May: MMY2002 study presented at ASCO
TOPLINE DATA
Company
Brand
Generic
Name
28
Brand
Generic
Name
US
Status
EU
Status
Other
Country
Status
Event Timeline
J&J Janssen
Velcade
bortezomib
SEE TAKEDA/MILLENIUM
J&J Janssen
Imbruvica
ibrutinib
SEE ABBVIE
J&J Janssen
Pg 77
n/a
imetelstat
J&J Janssen
Pg 77
Sylvant
siltuximab
MSD
Pg 78
MSD
Pg 78
Novartis
Pg 79
Emend
fosaprepitant
Keytruda
pembrolizumab
n/a
CTL019
15 Sep: Two Ph 2 studies published in NEJM
14 Nov: FDA lifts full clinical hold
14 Jun: Partial hold on MF trials lifted
14 Mar: FDA imposes a clinical hold on MF and
ET trials
FDA APP
MCD
FDA APP
CINV
FDA BTD
FDA BTD
EMA APP
US: MCD
14 Apr: FDA APP for MCD
14 Jun: EMA APP for MCD
14 Mar: EMA CHMP for MCD
13 Sep: EMA MAA and FDA BLA for MCD
EMA APP
US: CINV
16 Feb: FDA APP nausea prophylactic caused by
MEC
15 Sep: FDA APP for pediatric use
15 Jun: Ph 3 study presented at MASCC
14 Jun: Ph 3 study presented at MASCC
TOPLINE DATA
Company
16 Jun: KEYNOTE-87 presented at ASCO
16 Apr: FDA BTD for HL
15 Dec: KEYNOTE-23 presented at ASH
14 Dec: KEYNOTE-013 presented at ASH
15 Dec: Ph 2a study data presented at ASH
15 Jun: Ph 2 study data presented at ASCO
14 Dec: Two Ph 2 studies presented at ASH
14 Oct: Two Ph 2 studies published in NEJM
14 Jul: FDA BTD for relapsed/refractory ALL
13 Dec: Two Ph 2 studies presented at ASH
29
Novartis
Pg 79
Jadenu
deferasirox
Novartis
Pg 80
Promacta
Revolade
Eltrombopag
Novartis
Pg 81
n/a
midostaurin
Novartis
Pg 81
Tasigna
nilotinib
US
Status
EU
Status
FDA APP
CIO
FDA APP
ASAA, ITP,
thrombocytop
enia in Hep C
Other
Country
Status
Event Timeline
15 Mar: FDA APP CIO
EMA APP
ASAA, ITP,
thrombocytopenia
in Hep C
16 Jun: EXTEND analysis presented at EHA
16 Apr: EMA APP for pediatric ITP
15 Sep: EMA APP for ASAA
15 Aug: FDA expanded indication
15 Jul: EMA CHMP for ASAA
15 Jun: FDA APP for ITP
15 Feb: EMA sMAA for pediatric ITP
14 Dec: FDA sNDA for pediatric chronic ITP
14 Aug: FDA APP for ASAA
14 Jun: PETIT2 study presented at EHA
14 Feb: FDA sNDA for ASAA
14 Feb: FDA BTD for ASAA
13 Sep: EMA APP in thrombocytopenia in Hep C
FDA BTD
16 Jun: FDA BTD for ALM
16 Jun: Ph 2 study published in NEJM
15 Dec: RATIFY presented at ASH
FDA APP
PH+ CML
16 Jun: ENESTop & ENESTfreedom presented at
ASCO
15 Jun: ENEST1st study presented at EHA
14 Dec: 6-yr follow-up of ENEST study
presented at ASH
13 Dec: ENESTend, ENESTcmr, and LASOR
studies presented at ASH
EMA APP
PH+ CML
TOPLINE DATA
Company
Brand
Generic
Name
30
Novartis
Pgs 82-83
Novartis
Pg 84
Arzerra
ofatumumab
Farydak
panobinostat
US
Status
FDA APP
CLL
FDA APP
MM
EU
Status
EMA APP
CLL
EMA APP
US: MM
Other
Country
Status
CA: APP
CLL
Event Timeline
16 Jun: EMA CHMP negative opinion
maintenance CLL
16 May: FDA PRD for CLL sBLA
16 Mar: FDA sBLA for relapsed CLL
16 Mar: EMA sMAA for relapsed CLL
16 Jan: FDA APP for extended use in CLL
15 Jun: COMPLEMENT 2 presented at EHA
15 Apr: Topline data from COMPLEMENT 2
released
15 Apr: COMPLIMENT 1 published in Lancet
14 Jul: Topline data from PROLONG released
14 Jul: EMA APP for CLL 1st line
14 Jul: (see Abbvie: ibrutinib) RESONATE study
published in NEJM
14 Jun: Topline Ph 3 study data released
14 May: EMA CHMP for CLL
14 May: FDA APP for CLL
14 May: ORCHARD study terminated
14 May: (see AbbVie: ibrutinib) RESONATE
study published in NEJM
14 Apr: FDA APP for CLL
13 Dec: FDA PRD for CLL 1st line
13 Oct: EMA MAA and FDA NDA CLL 1st line
13 Sep: FDA BTD for previously untreated CLL
16 Jul Ph 2 study published in Blood
15 Sep: EMA APP for MM
15 Jun: EMA CHMP for MM
15 Jun: PANORAMA-1 presented at EHA
15 Feb: FDA APP for 2nd line MM
14 Nov: FDA extends review period
14 Nov: FDA Advisory Committee does not
recommend for MM
14 Sep: PANORAMA-1 published in Lancet
14 Jun: PANORAMA-1 presented at ASCO
13 Dec: Topline data from the PANORAMA-1
study released
TOPLINE DATA
Company
Brand
Generic
Name
31
Novartis
Pg 84
Brand
Generic
Name
US
Status
n/a
pegfilgrastim
biosimilar
EU
Status
Other
Country
Status
Event Timeline
16 Feb: EMA MAA chemotherapy induced
neutropenia
EMA MAA
16 Jun: FDA BTD for acute GVHD
16 Jun: RESPONSE-2 presented at EHA
16 Jun: 5-year follow-up of COMFORT-I
presented at ASCO
15 Dec: Topline RESPONSE-2 data released
15 Dec: 5-yr follow-up data from COMFORT-II
presented at ASH
15 Jun: RESPONSE update presented at EHA
15 Mar: EMA APP for PCV
15 Jan: RESPONSE published in NEJM
15 Jan: EMA CHMP for PVC
14 Dec: FDA APP for PCV
14 Dec: JUMP study presented at ASH
14 Jun: RESPONSE study presented at ASCO
14 Mar: RESPONSE study topline data released
13 Dec: COMFORT-I and COMFORT-II
presented at ASH
Novartis
Pg 85-86
Jakafi
Jakavi
ruxolitinib
Oryzon
ORY-1001
SEE ROCHE
Otsuka
Sativex
nabiximols
SEE GW PHARMA
Oxigene
Pg 86
OXi4503
FDA ODD
EMA COMP
Pfizer
Pg 86
BeneFIX
rFIX
FDA APP
hem B
EMA APP
hem B
Pfizer
Pg 86
Bosulif
bosutinib
FDA APP
PH+ CML
EMA APP
PH+ CML
FDA APP
PCV
EMA APP
PCV, MF
CA: APP
PCV, MF
TOPLINE DATA
Company
15 Dec: EMA COMP for AML
16 Mar: A Ph 3 study published in Haemophilia
14 Jul: Topline Ph 3 prophylaxis study data
released
CA: APP
PH+ CML
14 Dec: Long-term follow-up data presented at
ASH
14 Mar: Approved in Canada for PH+ CML
32
Pfizer
Pg 87
Pfizer
Pg 87
Brand
Generic
Name
Mylotarg
gemtuzumab+
ozogamicin
n/a
inotuzumab+
ozogamicin
Pfizer/Spark
Therapeutics
Pg 88
SPK-9001
Prothena
Pg 89
n/a
NEOD001
Rigel
Pg 89
n/a
fostamatinib
Roche
Pg 89
n/a
ACE910
emicizumab
US
Status
FDA NDA
withdrawn
EU
Status
Other
Country
Status
Event Timeline
14 Dec: AML-9 and ALFA-0701 presented at
ASH
FDA BTD
16 Jun: INO-VATE ALL was presented at EHA
and published in NEJM
15 Oct: FDA BTD for ALL
15 Apr: Topline data for INO-VATE ALL
released
14 Dec: Ph 2 study data presented at ASH
FDA BTD
16 Jul: Updated Ph 1/2 data released
16 Jul: FDA BTD
16 Jun: Ph 1/2 data presented at EHA
TOPLINE DATA
Company
16 Jul: Ph 1/2 study data presented at ISA
16 Feb: Interim Ph 1/2 study data published in JCO
15 May: Ph 1/2 safety data presented at ASCO and
EHA
FDA ODD
FDA BTD
15 Sep: FDA ODD for chronic ITP
16 May: Ph 1 study published in NEJM
15 Sep: FDA BTD for hem A
15 Jun: ACE002JP presented at ISTH
14 Dec: Ph 1 study presented at ASH
33
Brand
Generic
Name
Roche
Pgs 90-91
Gazyva
Gazyvaro
obinutuzumab
Roche
Pg 91
n/a
ORY-1001
Roche
Pg 91
MabThera
rituximab
Roche
n/a
venetoclax
Sandoz
Pg 92
n/a
rituximab
biosimilar
US
Status
FDA APP
CLL, NHL
(FL)
EU
Status
EMA APP
CLL
NHL (FL)
Other
Country
Status
16 Jul: GOYA did not meet its primary endpoint
16 Jun: EMA APP for FL
16 May: Topline data from GALLIUM released
16 Apr: EMA CHMP for NHL (FL)
16 Feb: FDA APP for NHL (FL)
15 Dec: Follow-up data from GADOLIN
presented at ASH
15 Dec: Updated data from CLL11 presented at ASH
15 Jun: GADOLIN presented at ASCO and EHA
15 Feb: GADOLIN study stopped early
14 Nov: Approved in Canada for CLL
14 Jul: EMA APP for 1st line CLL
14 May: EMA CHMP for 1st line CLL
14 Mar: Ph 3 study data published in NEJM
13 Dec: Updated CLL11 presented at ASH
13 Nov: FDA APP for CLL
13 Jul: Topline data from CLL11 released
CA: APP
CLL
13 Jul: EMA COMP for AML
EMA COMP
FDA APP
NHL (FL),
CLL, DLBCL
EMA APP
NHL (FL),
CLL, DLBCL
Event Timeline
TOPLINE DATA
Company
CA: APP
NHL (FL),
CLL, DLBCL
16 Jul: (see Roche: obinutuzumab) GOYA failed to
meet its primary endpoint
16 Jun: Ph 3 study published in Lancet
16 May: Ph 1/2 CNS lymphoma study published in
JCO
16 May: (see Roche: obinutuzumab) topline data from
GALLIUM released
15 Nov: (see Celgene: lenalidomide) Ph 2 study
published in NEJM
SEE ABBVIE
EMA MAA
16 Jun: Ph 2 study presented at EULAR
16 May: EMA MAA for NHL, CLL, DLBL, FL
34
US
Status
EU
Status
Other
Country
Status
Event Timeline
13 May: Topline data from JAKARTA released
Sanofi
Pg 92
n/a
SAR302503
Seattle
Genetics
Adcertis
brentuximab+
vedotin
Seattle
Genetics
Pg 93
n/a
vadastuximab
talirine
FDA ODD
Spectrum
Pg 93
Beleodaq
belinostat
FDA APP
PTCL
15 Dec: Ph 1 study data presented at ASH
15 Jun: BELIEF study published in JCO
14 Jul: FDA APP for relapsed/refractory PTCL
13 Dec: FDA NDA for relapsed/refractory PTCL
FDA APP
MM
16 Apr: FDA ODD for 7 year exclusivity
16 Mar: FDA APP for MM
15 Oct: FDA CRL requiring more data
15 Sep: Ph 2b study published in BBMT
15 Feb: Ph 2 study presented at BMT
14 Dec: FDA NDA for stem cell transplantation
in MM
14 Apr: Topline Ph 2 study data released
Spectrum
Pg 94
Evomela
melphalan
Spectrum
Pg 94
n/a
SPI-2012
Spectrum
Pg 94
Marqibo
vincristine
liposome
SEE TAKEDA/MILLENIUM
16 Jun: Ph 1 study presented at EHA
TOPLINE DATA
Company
Brand
Generic
Name
EMA COMP
14 Sep: Topline Ph 2 study data released
FDA APP
PH-ALL
13 Dec: Ph 2 study presented at ASH
35
Brand
Generic
Name
Syros
Pg 95
SY-1425
Takeda/
Millenium
Pg 95
n/a
alisertib
Takeda/
Millenium
Pg 95
Takeda/
Millenium
Pg 97
Velcade
bortezomib
Adcetris
brentuximab+
vedotin
US
Status
EU
Status
Other
Country
Status
JP: APP
(Amnolake)
APL
FDA IND
Event Timeline
16 May: FDA IND for AML and MDS
15 May: Ph 3 PTCL study terminated
FDA APP
MM, MCL
FDA APP
ALCL, HL
EMA APP
MM, MCL
EMA APP
ALCL, HL
CA: APP
MM, MCL
16 Jun: The Ph 2 IFM study published in JCO
16 Jun: (see Janssen: daratumumab) CASTOR
study presented at ACO
16 Apr: (see Janssen: daratumumab) CASTOR
study
16 May: IFM2013-04 study published in Blood
15 Mar: LYM-3002 published in NEJM
14 Oct: FDA APP for previously untreated MCL
14 Aug: FDA APP for relapsed MM
13 Dec: Retrospective of VISTA presented at
ASH
13 Aug: EMA APP for induction therapy in MM
CA: APP
Hl, ALCL
16 Jul: ADCETRIS published in Blood
16 Jul: EMA APP for post-transplant HL
16 May: EMA CHMP for HL following ASCT
16 Jan: EMA APP for retreatment of adult rrHL
and rrsALCL
15 Dec: Follow-up data from ADCETRIS study
presented at ASH
15 Oct: EMA CHMP for retreatment of adult
rrHL and rrsALCL
15 May: ATHERA study published in Lancet
14 Dec: ATHERA study presented at ASH
14 Dec: Follow-up data from ADCETRIS study
presented at ASH
14 Sep: Topline data from AETHERA released
14 Jan: Approved in Japan for HL and ALCL
13 Dec: Two Ph 2 studies presented at ASH
TOPLINE DATA
Company
36
Brand
Generic
Name
Takeda/
Millenium
Pg 98
Ninlaro
ixazomib
Takeda/
Millenium
Pg 98
n/a
MLN9708
Tesaro
Pg 99
Triphase
Pg 100
Varubi
rolapitant IV
n/a
marizomib
US
Status
FDA APP
MM
EU
Status
EMA CHMP
Other
Country
Status
JA: NDA
MM
Event Timeline
16 Jul: Japan NDA for MM
16 May: EMA CHMP negative opinion on MM
16 Apr: TOURMALINE-MM1 published in
NEJM
15 Dec: TOURMALINE-MM1 presented at ASH
15 Nov: FDA APP for 2nd line MM
15 Sep: FDA PRD for MM
15 Jul: EMA MAA for MM
15 Jul: FDA NDA for MM
15 Feb: Interim analysis of TOURMALINEMM1 released
14 Dec: Ph 2 study data presented at ASH
14 Dec: FDA BTD for AL
TOPLINE DATA
Company
13 Dec: A Ph 1/2 study was presented at ASH
FDA APP
CINV
FDA ODD
EMA MAA
16 Mar: EMA MAA for CINV
16 Mar: FDA sNDA for IV formulation
15 Sep: FDA APP for CINV
15 May: Topline data released
14 Sep: FDA NDA for CINV
14 Jun: Three Ph 3 studies presented at ASCO
14 May: Topline data from a Ph 3 study released
13 Dec: Topline data from 2 Ph 3 studies released
EMA COMP
16 Jun: Ph 1 study presented at ASCO
16 Jun: Ph 1/2 NPI-0052-101 study published in
Blood
15 Nov: FDA ODD for malignant glioma
15 Sep: Ph 1 study presented at IMS
14 Jun: EMA COMP for MM
14 Feb: FDA ODD for MM
37
Research and Regulatory Timeline Detail
AbbVie
duvelisib
Product Description: Duvelisib is a dual inhibitor of phosphoinositide-3-kinase-delta and gamma (PI3K). PI3K delta and
gamma are proteins that are known to support the growth and survival of malignant B-cells. They are also believed to play a
role in the formation and maintenance of the supportive tumor microenvironment.
Class: PI3K inhibitor Treatments (approved or in clinical trial): non-Hodgkin’s lymphoma, chronic lymphocytic
leukemia (CLL), small lymphocytic lymphoma
2016 Jun: Topline data from the Phase 2 DYNAMO study was released. DYNAMO evaluated the safety and efficacy of
duvelisib as monotherapy in patients with follicular lymphoma, small lymphocytic lymphoma, or marginal zone lymphoma
whose disease had progressed and who were refractory to rituximab and to either chemotherapy or radio-immunotherapy. The
primary endpoint of the study was overall response rate (ORR). ORR was 46% (all of which were partial response) and 41%
for the follicular lymphoma cohort, 68% for the small lymphocytic lymphoma cohort, and 33% for the marginal zone
lymphoma cohort.
2015 May: A Phase 1 study was presented at ASCO. The study evaluated the safety and efficacy of duvelisib in patients with
treatment-naïve CLL. The study achieved an 88% overall response rate.
PRODUCT DETAIL – ABBVIE
Product
38
Product
Continued on
next page
Product Description: Ibrutinib (Imbruvica) is a once-daily oral Bruton’s tyrosine kinase inhibitor (BTK). BTK is a signaling
molecule in the B-cell receptors that plays a key role in the spread of malignant B-cells. Ibrutinib blocks the signal telling
malignant cells to multiply.
Class: BTK inhibitor Treatments (approved or in clinical trial): chronic lymphocytic leukemia (CLL), non-Hodgkin's
follicular lymphoma (NHFL), mantel cell lymphoma (MCL), small lymphocytic lymphoma (SLL), Waldenström’s
macroglobulinenia (WM), chronic graft-versus-host-disease (cGVHD)
2016 Jun: FDA ODD received for the treatment of cGVHD.
2016 Jun: Longer-term follow-up data from the Phase 3 RESONATE, RESONATE-2, and HELIOS studies was presented
at ASCO. The updates from RESONATE and RESONATE-2 showed that ibrutinib was associated with favorable
progression-free survival (PFS) and overall survival in both treatment-naïve and treatment-experienced CLL/SLL patients.
Updates from HELIOS showed that ibrutinib plus bendamustine and rituximab continued to demonstrate superiority versus
bendamustine and rituximab alone in refractory/relapsed CLL/SLL patients.
2016 May: EMA APP received for use as 1st line treatment (treatment-naive) in CLL patients.
2016 May: FDA updates US prescribing information to include data from HELIOS and RESONATE studies and approves
ibrutinib for SLL
2016 Apr: EMA CHMP positive opinion received for use as 1st line therapy in CLL (the average length of time from CHMP
positive opinion to approval in the EU for hematology products listed in PDD is 3 months).
2016 Mar: FDA APP received for use as 1st line therapy in CLL
2015 Dec: The Phase 3 RAY study was published in the Lancet. The study compared ibrutinib with temsirolimus in patients
with relapsed/refractory MCL. The study met its primary endpoint of PFS with a 57% reduction of progression or death at 20
months.
2015 Dec: A Phase 2 study was presented at ASH. The study evaluated the efficacy of ibrutinib in treatment-naïve NHFL. The
study evaluated ibrutinib plus rituximab and achieved an overall response rate of 82%.
2015 Dec: The Phase 1/2b PCYC-1119 study was presented at ASH. The study suggested that the combination of ibrutinib
plus carfilzomib, with or without dexamethasone, achieved a 62% overall response rate in patients with relapsed/refractory
MM.
2015 Dec: The Phase 3 RESONATE-2 study was published in NEJM. The study compared ibrutinib with chlorambucil in
treatment-naïve CLL patients aged ≥65 years. The study met its primary-endpoint of PFS, reducing the risk of progression or
death by 84%.
2015 Nov: FDA sNDA submitted for relapsed/refractory CLL or SLL in combination with bendamustine and rituximab.
2015 Sep: FDA sNDA submitted for potential 5th indication for treatment-naïve CLL based on data from the RESONATE-2.
2015 Jul: EMA APP for the treatment of WM in patients who have received at least one prior therapy.
2015 Jun: AbbVie released results of the Phase 3 RESONATE study. RESONATE compared ibrutinib with chlorambucil in
treatment-naïve CLL/SLL patients ≥65 years. Ibrutinib achieved significant improvement in progression-free survival, overall
survival, and overall response.
2015 May: EMA CHMP positive opinion received for use in WM patients who have had at least one prior therapy or as1 st line
therapy for patients who are unsuitable for chemo-immunotherapy.
PRODUCT DETAIL – ABBVIE
AbbVie/J&J
Imbruvica
Research and Regulatory Timeline Detail
39
Product
Continued
2015 May: The Phase 3 HELIOS study was presented at ASCO. HELIOS compared ibrutinib add on therapy with placebo
in previously treated CLL or SLL patients who were taking the combination bendamustine+rituximab. The ibrutinib cohort
experienced an 80% reduction in disease progression or death compared with the placebo cohort.
2015 Apr: NEJM published a Phase 2 study. The study evaluated ibrutinib in treatment-experienced WM patients. Ibrutinib
achieved durable responses and MYD88 and CXCR4 mutations affected response.
2015 Mar: The Phase 3 HELIOS study was stopped early because it met its primary endpoint.
2015 Jan: FDA APP for the treatment of WM.
2014 Dec: The Phase 2 RESONATE-17 study was presented at ASH. RESONATE-17 evaluated the efficacy and safety of
ibrutinib in CLL and SLL patients with the del 17 mutation. The primary endpoint was overall rate or response (ORR). The
study achieved its endpoint with an ORR of 82.6%.
2014 Oct: EMA APP for the treatment of relapsed or refractory MCL and CCL in patients who have received at least one
prior therapy or 1st line therapy in patients with 17p depletion or TP53 mutations.
2014 Oct: FDA sNDA for the treatment of WM.
2014 Jul: FDA APP for use as 2nd line treatment in CLL patients with 17p deletion.
2014 Jul: EMA CHMP positive opinion received for the treatment of MCL and CLL.
2014 Jul: The Phase 3 RESONATE study was published in NEJM. RESONATE compared ibrutinib with ofatumumab in
relapsed/refractory CLL/SLL patients who received at least one prior therapy and were not eligible for purine analog
treatment. The study met its primary endpoint significantly increasing progression free survival.
2014 Apr: FDA sNDA for the treatment of relapsed/refractory CLL.
2014 Feb: FDA APP for the treatment of CLL in patients who have received at least one previous therapy.
2014 Jan: The Phase 3 RESONATE study was stopped early because it met its primary endpoint
2013 Dec: A Phase 1b/2 study was published in Lancet Oncology. The study evaluated the safety of ibrutinib in previously
untreated CLL and SLL patients aged ≥65 years. The study met its primary endpoint of safety.
2013 Nov: FDA APP for the treatment of MCL in patients who have received at least one prior therapy.
2013 Oct: EMA MAA for the treatment of relapsed/refractory CLL, relapsed refractory SLL, and relapsed refractory MCL.
2013 Aug: FDA NDA for the treatment of MCL, CLL & SLL.
PRODUCT DETAIL – ABBVIE
AbbVie/J&J
Imbruvica
Research and Regulatory Timeline Detail
40
Product
Product Description: Venetoclax (Venclexta)is a B-cell lymphoma-2 (BCL-2) inhibitor. Venetoclax selectively inhibits the
BCL-2 protein. The BCL-2 protein prevents programed cell death (apoptosis) in some cells, including lymphocytes.
Class: BCL-2 inhibitor Treatments (approved or in clinical trial): acute myeloid leukemia (AML), chronic graftversus-host-disease (cGVHD), chronic lymphocytic leukemia (CLL), multiple myeloma (MM)
2016 Apr: FDA APP for 2nd line CLL in patients with 17p deletion.
2016 Feb: EMA COMP received for the treatment of AML.
2016 Jan: FDA BTD granted, for use in combination with hypomethylating agents for patients with treatment-naïve AML
who are ineligible to receive standard induction therapy.
2016 Jan: FDA BTD granted for use in combination with rituximab for relapsed/refractory CLL.
2016 Jan: EMA MAA for use in CLL patients with 17p deletion or TP53 mutation (the average length of time from
submission to approval in the EU for hematology products listed in PDD is 16 months).
2016 Jan: FDA NDA & PRD for use in CLL patients who have received at least one prior therapy, including patients with
17p deletion.
2015 Dec: A Phase 1 study was published in NEJM. The study showed a 79% overall response rate (ORR) in
relapsed/refractory CLL patients treated with venetoclax with 20% experiencing a complete response.
2015 Dec: A Phase 2 study was presented at ASH. The study evaluated venetoclax in patients with relapsed/refractory CLL
and 17p depletion. The study met its primary endpoint with a 79.4% ORR.
2015 Aug: The results of the Phase 2 M13-982 study were released. Venetoclax met its primary endpoint of ORR in patients
with relapsed/refractory or previously untreated CLL in patients with 17p deletion.
2015 Jun: Update results from a Phase 1b study presented at ASCO showed that venetoclax, in combination with rituximab
had on overall response rate of 84%.
2015 May: FDA BTD status received for relapsed/refractory CLL in previously treated patients with 17p deletion genetic
mutation.
2015 May: Results from two Phase 1b studies were presented at ASCO. The 1st study showed that venetoclax, combined with
bortezomib and dexamethasone achieved an 83% ORR in bortezomib-naïve patients with relapsed/refractory MM. The 2nd
Phase 1b/II study suggested that ibrutinib may be effective in patients with cGVHD. cGVHD is a common complication after
stem cell or bone marrow transplantation.
PRODUCT DETAIL – ABBVIE
AbbVie:
Venclexta
Research and Regulatory Timeline Detail
41
Product
Product Description: Caplacizumab is a selective bivalent von Willebrand factor inhibitor (VWF). Caplacizumab inhibits the
interaction between VWF and platelets by targeting the A1 domain of VWF, thus inhibiting the ULVWF mediated platelet
interactions and the formation of string-like clots in patients with TTP.
Class: anti VWF Treatments (approved or in clinical trial): acquired thrombotic thrombocytopenic purpura (aTTP)
2016 May: A post-hoc analysis of the Phase 2 TITAN study was presented at European Society of Hematology (ESH). The
study compared caplacizumab with placebo in patients with aTTP. The post-hoc analysis assessed caplacizumab on a
composite endpoint of major thromboembolic events and TTP-related mortality. The results demonstrated that the
caplacizumab cohort achieved significant improvement in in thromboembolic events or death (11.4% vs 43.2% for placebo).
2016 Feb: NEJM published the Phase 2 TITAN study. The study compared caplacizumab with placebo in patients with aTTP.
The primary endpoint was time to response, defined as confirmed normalization of platelet count. The study met its primary
endpoint with a 39% reduction in the time to response in the caplacizumab cohort compared with placebo.
2014 Dec: Updated data from the Phase 2 TITAN study was presented at ASH. The primary endpoint was confirmed platelet
normalization leading to a stop in daily plasma exchanges (standard therapy for aTTP). Only 8% of the caplacizumab cohort
experienced exacerbations compared with 28% for the placebo cohort.
2014 Jun: Topline data from the Phase 2 TITAN study was released. The caplacizumab cohort achieved platelet
normalization at twice the rate of the placebo cohort.
PRODUCT DETAIL – ABLYNX
Ablynx:
caplacizumab
Research and Regulatory Timeline Detail
42
Research and Regulatory Timeline Detail
Alexion
Soliris
Product Description: Eculizumab (Soliris) is a terminal complement inhibitor that specifically binds to the terminal
Complement component 5 (C5). The complement system plays a role in immunity as a protective mechanism. Its
dysregulation can result in a variety of life-threatening complications in a broad range of diseases. The C5 component
inhibits terminal complement-mediated intravascular hemolysis in paroxysmal nocturnal hemoglobinuria, complementmediated thrombotic microangiopathy (TMA) in patients with atypical hemolytic uremic syndrome, and may be responsible
for the muscle weakness associated with myasthenia gravis.
Class: C5 inhibitor Treatments (approved or in clinical trial): atypical hemolytic uremic syndrome (aHUS),
paroxysmal nocturnal hemoglobinuria (PNH), myasthenia gravis (MG), acute antibody-mediated rejection (AMR)
2014 Dec: Two post-hoc sub-analyses of studies C10-003 (pediatric) and C10-004 (adults) were presented at ASH. The 26week analysis evaluated the efficacy and safety of eculizumab in patients with aHUS with or without identified genetic
mutations. Platelet count normalization was achieved in 100% of patients with mutations in both studies; for patients without
mutations achievement rates were 91% for children and 95% for adults.
2014 Nov: UK NICE recommends that all aHUS patients be treated with eculizumab.
2014 Nov: A one-year update of a prospective study was presented at ASN Kidney Week. The data showed that eculizumab
inhibited TMA and improved renal outcomes.
2014 Jun: A sub-analysis from a prospective study was presented at ERA-EDTA. The analysis demonstrated that eculizumab
therapy resulted significant improvements in TMA, reversal of kidney damage, significant improvement in life expectancy,
and complement blockade inhibited endothelial damage, inflammation, thrombotic risk and renal damage.
2014 May: FDA sBLA for the treatment of aHUS reflecting research showing longer term benefit of chronic and sustained
eculizumab therapy in aHUS patients.
2014 Mar: UK NICE positive assessment for the treatment of aHUS.
2013 Nov: Two Phase 2 prospective studies were presented at ASN Kidney Week. The studies evaluated the efficacy of
eculizumab in pediatric and adult patients with aHUS. Eculizumab inhibited TMA and improved renal function in both
children and adults.
2013 Sep: Japan APP for the treatment of pediatric and adult patients with aHUS.
PRODUCT DETAIL – ALEXION
Product
43
Research and Regulatory Timeline Detail
Alnylam/
Genzyme
ALN-CC5
Product Description: ALN-CC5 is a subcutaneous RNAi therapeutic targeting the C5 component of the complement
pathway. The complement system plays a role in immunity as a protective mechanism. Its dysregulation can result in a variety
of life-threatening complications in a broad range of diseases. Complement component C5 is predominantly expressed in the
liver. Anti-C5 monoclonal antibody therapy has demonstrated clinical activity and tolerability in a number of complementmediated disease (CMD). ALN-CC5 is a RNAi therapeutic that silences C5. It can be dosed subcutaneously and provides
increased potency and durability compared with monoclonal antibody therapy.
Class: RNAi C5 inhibitor
Treatments (approved or in clinical trial): paroxysmal nocturnal hemoglobinuria (PNH),
atypical hemolytic-uremic syndrome (AHUS), mysathenia gravis, neuromyelitis optica, membranous nephropathy
2016 Jun: Part C of a Phase1/2 study was presented at the European Society of Hematology (ESH). The study is an ongoing
study conducted in three parts. Part C is an open-label, multi-dose study designed to assess safety, tolerability, and clinical
activity of ALN-CC5 administered as monotherapy or as an adjunct to eculizumab for 16 weeks in patients with PNH. Part of
the study included an exploratory evaluation of ALN-CC5 effects on levels of lactate dehydrogenase (LDH – a biomarker for
red blood cell hemolysis). Preliminary results demonstrated that ALN-CC5 achieved clamped knockdown of serum C5 and
lowered LDH. Study results indicated that ALN-CC5 could potentially reduce dose and frequency of administration of
eculizumab.
2015 Dec: A Phase 1/2 study was presented at ASH. The study evaluated the efficacy of ALN-CC5 for the treatment of
CMDs. Results showed a 99% knockdown of serum C5 and up to 98% inhibition of serum hemolytic activity.
Alnylam/
Genzyme
fitusiran
ALN-AT3
Product Description: Fitusiran (ALN-AT3) is a subcutaneous RNAi therapeutic targeting antithrombin (AT) for the treatment
of hemophilia. AT is a protein expressed in the liver that acts as an anti-coagulant by inactivating factor Xa and thrombin.
RNA interference (RNAi) is a new biological therapeutic approach. RNAi is a natural process of gene silencing. RNAi
silences specific mRNAs thus preventing disease-causing proteins from being made.
Class: RNAi AT
Treatments (approved or in clinical trial): hemophilia A (hem A) hemophilia B (hem B)
2015 Dec: A Phase 1 study was presented at ASH. The study showed fitusiran reduced AT by 88% and resulted in an 85%
reduction in median estimated annualized bleeding rates.
2015 Jun: Data from a Phase 1 study was presented at ISTH. The study results showed that ALN-AT3 achieved knockdown
of AT of up to 86% in hem A and B patients. The knockdown was highly durable, lasting up to two months after the last dose.
2015 Jan: Data from a Phase 1 study was presented at GCC. The study results showed up to a 70% knockdown of AT and
resulted in a thrombin increase of 334% with a marked improvement in blood clotting.
2014 Dec: Data from a Phase 1 study was presented at ASH. The study showed up to a 57% knockdown of AT in hemophilia
patients with the effects lasting for about 60 days.
2014 Aug: EMA COMP received for hem A and B.
2014 May: Topline data from a Phase 1 study was presented at WFH. The data showed that a single dose of ALN-AT3
resulted in a 28% to 32% knockdown of AT levels, which was statistically significant compared with placebo.
2013 Aug: FDA ODD for hem A and B.
PRODUCT DETAIL – ALNYLAM
Product
44
Research and Regulatory Timeline Detail
Amgen
Blincyto
Product Description: Blinatumomab (Blincyto) is a monoclonal antibody designed to direct T-cells and helper T-cells
against target cells expressing either the CD3 complex or the CD-19 proteins found on the surface of B-cell derived leukemias
and lymphomas.
Class: anti-CD19/CD3
Treatments (approved or in clinical trial): acute lymphoblastic leukemia (ALL), chronic
lymphocytic leukemia (CLL), hairy cell leukemia (HCL), mantle cell leukemia (MCL), prolymphocytic leukemia (PL),
indolent B-cell lymphoma (IBCL), pediatric relapsed/refractory ALL, relapsed/refractory Philadelphia positive (PH+) Bprecursor ALL, Philadelphia negative (PH-) relapsed/refractory B-precursor ALL, minimal residual disease positive
(MRD+) B-precursor ALL, relapsed/refractory non-Hodgkin’s lymphoma (NHL), relapsed/refractory diffuse large B-cell
lymphoma (DLBCL),
2016 Jun: The Phase 3 TOWER study was presented at European Society of Hematology (ESH). The TOWER study
compared blinatumomab with standard of care chemotherapy (SOC) in adults with PH- relapsed or refractory B-precursor
ALL. The study met its primary endpoint of overall survival (7.7 months in the blinatumomab cohort versus 4 months for the
SOC cohort).
2016 May: FDA PRD received for the treatment of pediatric PH- ALL.
2016 Apr: The Journal of Clinical Oncology (JCO) published a Phase 1 dose escalation study. The study evaluated the
tolerability of blinatumomab in patients with relapsed/refractory B-cell NHL. The maximum tolerated dosage was 60 μg/m2 per
day and blinatumomab showed anti-lymphoma activity as a single agent.
2016 Mar: FDA sBLA filed for the treatment of pediatric and adolescent PH- relapsed/refractory B-cell precursor ALL (the
average length of time from FDA BLA submission to approval in the US for hematology products listed in PDD is 7 months).
2016 Feb: The Phase 3 TOWER study was stopped early. The study met its primary endpoint of overall survival. The study
was ended early for efficacy.
2015 Dec: Data from three Phase 2 studies was presented at ASH. All three studies evaluated the efficacy and safety of
blinatumomab in adult patients with ALL. Blinatumomab achieved significant improvement in relapse-free survival in patients
with B-cell precursor ALL. Patients who achieved minimal residual disease with blinatumomab had longer overall survival and
relapse-free survival, and 36% of patients with poor prognosis achieved complete remission (CR) and partial hematological
recovery.
2015 Nov: EMA APP. The EMA has granted conditional approval for relapsed/refractory B-precursor PH- ALL.
2015 Sep: EMA CHMP positive opinion received for the treatment PH- relapsed or refractory B-precursor ALL
2015 Jul: Results from a Phase 2 efficacy and safety study showed that blinatumomab achieved CR or CR with partial
hematological recovery in a significant number of relapsed/refractory PH+ B-cell precursor ALL patients.
2014 Dec: The Phase 2 211 study was presented at ASH. Study results showed that 40% of patients relapsed/refractory B-cell
precursor ALL achieved CR or CR with partial hematologic recovery and were able to proceed to stem cell transplantation.
2014 Dec: FDA APP received for the treatment of PH- relapsed/refractory B-cell precursor ALL (PH- ALL).
2014 Oct: FDA PRD for relapsed/refractory B-precursor acute PH- ALL.
2014 Sep: FDA BLA for the treatment of adults with relapsed/ refractory PH- ALL a rapidly progressing blood and bone
marrow cancer.
PRODUCT DETAIL – AMGEN
Product
45
Product
Continued
on next page
Product Description: Carfilzomib (Kyprolis) is a 20S proteasome inhibitor. Proteasome inhibitors break down proteins in cells
that are no longer needed. Inhibition of this process may lead to cell cycle arrest, apoptosis and inhibition of tumor growth.
Carfilzomib is approved in the US for multiple myeloma (MM) in patients who have received at least two other therapies and
have experienced disease progression with 60 days of last treatment.
Class: proteasome inhibitor
Treatments (approved or in clinical trial): multiple myeloma (MM)
2016 Jul: EMA APP received for use in combination with dexamethasone, for patients with MM who have received at least
one prior therapy.
2016 Jun: Blood published the Phase 1/2 CHAMPION-1 study. The study evaluated the safety and efficacy of carfilzomib plus
dexamethasone in patients with relapsed/refractory MM. The primary endpoint of the Phase 1 portion of the study was to
determine the optimum dosage. The primary endpoint of the Phase 2 portion of the study was overall response rate (ORR). A
30-minute IV infusion of 20-70 mg/m2 was well-tolerated, and active with a ORR of 70%.
2016 Jun: A post-hoc analysis of the Phase 3 ASPIRE study and a sub-analysis of the Phase 3 ENDEAVOR study were
presented at European Society of Hematology (ESH). Results from ASPIRE showed a cumulative rate of complete response
increased over time in the carfilzomib plus dexamethasone cohort, most quickly in the first 15 months of treatment. The subanalysis of ENDEAVOR showed that the carfilzomib plus dexamethasone cohort achieved higher progression-free survival
(PFS) in relapsed/refractory MM patients compared with the bortezomib plus dexamethasone cohort.
2016 Jun: A secondary analysis of the Phase 3 ASPIRE study was presented at ASCO. The study compared carfilzomib plus
dexamethasone and cyclophosphamide with dexamethasone and cyclophosphamide alone in patients with relapsed MM. The
primary endpoint was PFS. PFS was 24.5 months in the carfilzomib cohort versus 12.5 months in the dexamethasone and
cyclophosphamide alone cohort.
2016 May: EMA CHMP positive opinion received for carfilzomib in combination with dexamethasone in treatmentexperienced adult patients with MM (the average length of time from EMA CHMP recommendation to approval in the EU for
hematology products listed in PDD is 3 months).
2016 May: The Kyprolis Global Economic Model (K-GEM) study was published in the Journal of Medical Economics (JME).
The study showed that carfilzomib plus lenalidomide and dexamethasone was cost-effective compared with lenalidomide plus
dexamethasone alone in patients with relapsed/refractory MM.
2016 Jan: FDA APP received for the treatment of relapsed/refractory MM in combination with dexamethasone or lenalidomide.
2015 Dec: New data from the Phase 3 ENDEAVOR study was published in Lancet Oncology. The study compared carfilzomib
plus dexamethasone with bortezomib plus dexamethasone in patients with relapsed MM. The study met its endpoint achieving
significant improvement in PFS (18.7 months compared with 9.4 months).
2015 Dec: EMA sMAA for relapsed MM in combination with dexamethasone in patients who have received at least one prior
therapy (the average length of time from submission to approval in the EU for hematology products listed in PDD is 16 months).
2015 Nov: EMA APP received for the treatment of MM in combination with lenalidomide and dexamethasone for treatment of
adults with MM who have received at least one prior therapy.
2015 Sep: EMA CHMP positive opinion received for MM in combination with lenalidomide and dexamethasone in patients
who have received at least one prior therapy.
2015 Sep: FDA sNDA for relapsed MM. The FDA has also agreed to a priority review.
PRODUCT DETAIL – AMGEN
Amgen
Kyprolis
Research and Regulatory Timeline Detail
46
Product
Continued
2015 Jul: FDA APP received for the sNDA of carfilzomib combined with lenalidomide and dexamethasone for MM
patients who have received 1 to 3 prior lines of therapy.
2015 Mar: The results of the ENDEAVOR study were released. The study showed that carfilzomib+low-dose
dexamethasone met its primary endpoint of PFS compared with bortezomib+low-dose dexamethasone.
2015 Jan: FDA sNDA and EMA MAA or patients with relapsed MM who have received at least one prior therapy
2014 Dec: The Phase 3 ASPIRE study was published in NEJM. The study showed that carfilzomib combined with
lenalidomide+dexamethasone increased PFS by 8.7 months compared with lenalidomide+dexamethasone alone.
2014 Aug: Topline results of the Phase 3 FOCUS study were released. FOCUS compared single therapy carfilzomib
with dexamethasone+cyclophosphamide in patients with relapsed advanced refractory MM. Carfilzomib did not meet its
primary endpoint.
Amgen
Aranesp
Product Description: Darbepoetin alfa (Aranesp) is a recombinant analog of the endogenous cytokine erythropoietin, an
erythropoiesis-stimulating agent (ESA). Two carbohydrate chains have been added to darbepoetin tripling its half-life
compared with erythropoietin. Darbepoetin binds to and activates erythropoietin receptors thereby stimulating endothelial
cell proliferation, B-cell proliferation and immunoglobulin. It is indicated in both Europe and the United States for renal
failure and chemotherapy induced anemia.
Class: ESA Treatment (approved or in clinical trial): renal failure induced anemia, chemotherapy induced
anemia, anemia in myelodysplastic syndrome (MDS)
2016 Feb: Topline data from the Phase 3 ARCADE study was released. ARCADE compared darbepoetin with placebo
in patients with low or intermediate-1 risk MDS who had not previously taken an ESA. The study met its primary
endpoint of reducing the incidence of red blood cell transfusions in anemic patients with low or intermediate-1 risk MDS.
Amgen
Xgeva
Product Description: Denosumab (Xgeva) is a monoclonal antibody receptor activator of nuclear factor kappa-B ligand
(RANKL) inhibitor. It is a subclass of tumor necrosis factor (TNF) superfamily. Denosumab is approved in both the US
and Europe for bone metastases and giant cell tumor of bone (GCTB). The FDA approved denosumab for hypercalcemia
of malignancy (HCM). HCM is a serious complication in both solid and hematologic cancers.
Class: RANKL
Treatments (approved or in clinical trial): bone metastases, GCTB, HCM
2014 Dec: FDA APP received for the treatment of HCM refractory to bisphosphonate therapy.
Amgen
Nplate
Product Description: Romiplostim (Nplate) is a recombinant protein with megakaryopoiesis stimulating activity.
Romiplostim mimics endogenous thrombopoietin (TPO), directly binding to and activating the platelet thrombopoietin
receptor (TpoR, Mpl, or CD110 antigen), a cytokine receptor belonging to the hematopoietin receptor superfamily.
Activation of TpoR stimulates an increase in the production of blood platelets.
Class: thrombopoietin (THPO) Treatments (approved or in clinical trial): immune thrombocytopenia (ITP)
2016 Jul: Lancet published a Phase 3 study that compared romiplostim with placebo in children (aged 1-17 years) with
ITP and a mean platelet count of ≤30x109/L. The study met its primary endpoint of durable platelet response
(achievement of weekly platelet response - platelet counts ≥50 × 109/L without rescue drug use in the preceding 4 weeks
in 6 or more of the final 8 weeks of the study). Durable platelet response was observed in 52% of the romiplostim cohort
versus 10% in the placebo cohort.
PRODUCT DETAIL – AMGEN
Amgen
Kyprolis
Research and Regulatory Timeline Detail
47
Research and Regulatory Timeline Detail
Ariad
Iclusig
Product Description: Ponatinib hydrochloride (Iclusig) is a tyrosine kinase inhibitor (TKI). The primary target of
ponatinib is the tyrosine kinase BCR-ABL. BCR-ABL is associated with excessive, and unregulated production of white
blood cells in the bone marrow. TK inhibitors stop this process. Ponatinib seems to especially effective in patients with a
T3151 mutation.
Class: TKI
Treatments (approved or in clinical trial): T3151+ chronic myeloid leukemia (CML), Philadelphia+
acute lymphoblastic leukemia (PH+ ALL), gastrointestinal stromal tumors (GIST), chronic phase chronic myeloid
leukemia (CP-CML)
2016 Jun: Long-term follow-up data from the Phase 2 PACE study was presented at the European Society of Hematology
(ESH). The study evaluated the efficacy and safety of ponatinib in CML.The study evaluated the efficacy of ponatinib in
patients with CML and PH+ ALL patients resistant to or intolerant of dasatinib or nilotinib, or with the T3151 mutation. The
follow-up data showed that patients treated with ponatinib continued to demonstrate anti-leukemic activity with a median
follow-up of 4 years for CP-CML patients.
2016 Apr: Blood published the Phase 2 PACE study. Ponatinib was efficacious in PH+ leukemia patients resistant to other
TKIs. CP-CML patients with T3151 mutation had better responses to ponatinib. Those patients with T3151 plus additional
mutations had significantly inferior response.
2016 Jan: Japan NDA submission for the treatment of CML and PH+ ALL.
2015 May: 4 year data from a Phase 1 study was presented at ASCO. The study showed that ponatinib continued to show
antileukemic activity in patients with CP-CML.
2014 Dec: Results from the Phase 2 PACE trial were presented at ASH. The study showed that ponatinib demonstrated
antileukemic activity, at 3 years, in patients with CP-CML.
2014 Oct: The EMA Pharmacovigilance Risk Assessment Committee (EMA PRAC) recommended that ponatinib continue
to be used for approved indications in the EU.
2013 Dec: Sales in the US resumed.
2013 Nov: EMA CHMP positive opinion received to continue to market ponatinib for approved indications.
2013 Oct: Distribution of ponatinib temporarily suspended in the US due to adverse arterial thrombotic (AT) events.
2013 Oct: The Phase 3 EPIC study, focusing on patients with newly diagnosed CML, was discontinued early due to
adverse AT events.
Astellas
ASP2215
Product Description: ASP2215 is an investigational tyrosine kinase inhibitor (TKI) of the FTL3 and AXL receptors, which
are indicated in growth of cancer cells. Mutations in FTL3 are seen in up to 1/3 of acute myeloid leukemia (AML) patients.
Class: TKI Treatments (approved or in clinical trial): AML
2015 May: Preliminary results from a Phase 1/2 study were presented at ASCO. The study showed that ASP2215 achieved
a 57% overall response rate in patients with relapsed or refractory AML.
PRODUCT DETAIL – ARIAD, ASTELLAS
Product
48
Research and Regulatory Timeline Detail
Astellas/
Basilea
Cresemba
Product Description: Isavuconazole (Cresemba) is an azole antifungal. Azole antifungals work by inhibiting the enzyme
lanosterol 14 a-demethylase which is required to convert lanosterol to ergosterol. Ergosterol depletion disrupts the
membrane structure of fungi. Isavuconazole is effective in treating aspergillosis and mucormycosis which are common in
the environment but can be dangerous in immuno-compromised patients.
Class: azole antifungal
Treatments (approved or in clinical trial): aspergillosis, mucormycosis, candidiasis
2015 Oct: EMA APP received for the treatment of invasive aspergillosis and invasive mucormycosis.
2015 Jul: Topline results from the Phase 3 ACTIVE study was released. The study compared isavuconazole with
caspofungin in patients with candidemia and other candida infections. Isavuconazole did not meet its primary endpoint
of non-inferiority to caspofungin.
2015 Jul: EMA CHMP positive opinion received for the treatment of invasive aspergillosis and mucormycosis.
2015 Mar: FDA APP received for the treatment of invasive aspergillosis and invasive mucormycosis.
2015 Jan: FDA ACR received for the treatment of invasive aspergillosis and invasive mucormycosis.
2014 Nov: FDA ODD received for the treatment of invasive candidiasis.
2014 Jul: EMA MAA for the treatment of invasive aspergillosis and mucormycosis.
2014 Jul: FDA NDA for treatment of invasive aspergillosis and invasive mucormycosis and QIDP for the treatment of
invasive candidiasis.
2014 May: Results from the Phase 3 SECURE study were presented at ECCMID. Isavuconazole had statistically fewer
adverse events than voriconazole.
2014 Feb: FDA QIDP for the treatment of invasive mucormycosis.
2013 Dec: FDA QIDP for the treatment of invasive aspergillosis.
2013 Nov: FDA ODD for the treatment of invasive mucormycosis.
2013 Sep: Topline data from the Phase 3 SECURE study was released. The study compared isavuconazole with
voriconazole in the treatment of aspergillosis. Isavuconazole achieved its primary endpoint of statistical non-inferiority to
voriconazole.
AstraZeneca
acalabrutinib
Product Description: Acalabrutinib is a selective, irreversible, second generation Bruton’s tyrosine kinase (BTK)
inhibitor. BTK plays an important role in the maturation of B-cells as well as mast cell activation.
Class: BTK Treatments (approved or in clinical trial): chronic lymphocytic leukemia (CLL), small lymphocytic
lymphoma (SLL), mantle-cell lymphoma (MCL), and lymphoplasmacytic lymphoma (Waldenström's
macroglobulinemia; WM)
2015 Feb: EMA COMP for the treatment of CLL, SLL, MCL, WM.
2015 Dec: A Phase 1/2 study was published in NEJM. The study evaluated the efficacy and safety of acalabrutinib in
patients with relapsed CLL. The study showed that acalabrutinib had a promising safety and efficacy profile including in
patients with chromosome 17p13.1 deletion.
PRODUCT DETAIL – ASTELLAS, ASTRA
Product
49
Research and Regulatory Timeline Detail
AstraZeneca
AZD9150/
ISIS-STAT3
Product Description: AZD9150/ISIS-STAT3 is a signal transducer and activator of transcription 3 (STAT3) inhibitor.
STAT3 mediates the expression of a number of genes and plays an active role in cell growth and apoptosis.
Consequently, STAT3 activation has been shown to be associated with growth, invasiveness, and metastases of a number
of cancers.
Class: STAT3 inhibitor Treatments (approved or in clinical trial): hepatocellular carcinoma, diffuse large B-cell
lymphoma (DLBCL)
2013 Jun: A Phase 1 study was presented at ASCO. The study evaluated the safety and efficacy of AZD9150 in patients
with advanced solid tumors, lymphoma and DLBCL who had relapsed or were refractory to multiple chemotherapy
regimens. The study showed a partial response in DLBCL patients.
ASTX
guadecitabine
Product Description: Guadecitabine (SGI-110) is a small molecule, DNA-hypomethylating agent that targets and
reverses aberrant DNA hypermethylation, a characteristic of many cancer cells. It restores the expression of tumor
suppressor genes and cancer testis antigens. It may also sensitize tumor cells to other anticancer agents.
Class: DNMT inhibitor Treatments (approved or in clinical trial): Acute myeloid leukemia (AML),
myelodysplastic syndromes (MDS)
2015 Oct: FDA ODD received for the treatment of AML.
2015 Aug: A Phase 1 study was published in Lancet Oncology. The study evaluated the safety, pharmacokinetics, and
dosage of guadecitabine is heavily pre-treated AML and MDS patients. The study showed a dose-related DNA
demethylation response.
2014 Jun: A Phase 2 study was presented at European Society of Hematology (ESH). The study evaluated the safety and
efficacy of SGI-110 in elderly treatment-naïve AML patients not suitable for intensive chemotherapy. The primary
endpoint was overall complete remission (CR) or CR with incomplete normal count recover (CRi). The study met its
endpoint with 55% of patients achieving CR or CRi.
Baxalta
Advate
Product Description: ADVATE is a recombinant antihemophilic factor plasma/albumin-free method derived from the
FVIII gene. It is to control and prevent bleeding in adults and children with hemophilia A (hem A). It is also indicated for
routine prophylaxis to prevent or reduce bleeding episodes in adults and children with hem A.
Class: rFVIII Treatments (approved or in clinical trial): hem A
2014 Apr: FDA APP received for the new BAXJECT III reconstitution system for Advate.
2014 Feb: The AHEAD study results were presented at EAHAD. AHEAD is a four-year outcomes study which showed
that more than half of the patients using Advate prophylaxis therapy had fewer than two bleeding episodes per year.
PRODUCT DETAIL – ASTRAZENECA, ASTX, BAXALTA
Product
50
Research and Regulatory Timeline Detail
Baxalta
EU: Adynovi
US: Adynovate
Product Description: Adynovate is built on the full-length Advate molecule. Advate is a recombinant antihemophilic
factor plasma/albumin-free method derived from the factor VIII (FVIII) gene. Adynovate combines Advate with
PEGylation technology designed to extend the amount of FVIII available for use by the body.
Class: rFVIII Treatment (approved or in clinical trial): hemophilia A (hem A)
2016 Apr: Approved in Japan for the treatment of hem A.
2016 Mar: EMA MAA for the treatment of pediatric hem A, adult hem A, and for use during surgery (the average
length of time from EMA MAA submission to approval in the EU for hematology products listed in PDD is 16 months).
2016 Feb: FDA sBLA for use in children aged <12 years with hem A and for use in surgical settings (the average length
of time from FDA BLA submission to approval in the US for hematology products listed in PDD is 7 months).
2015 Dec: Topline data from an uncontrolled Phase 3 study was released. The study was designed to measure the safety
and efficacy of Adynovate in previously treated patients with severe hem A aged <12 years as prophylaxis and for the
treatment of bleeding episodes. The study met its primary endpoints as no patients developed antibodies to Adynovate
and no treatment-related adverse events were reported.
2015 Nov: FDA APP received for the treatment of hem A.
2015 Jul: A Phase 2/3 study was published in Blood. The study compared twice weekly prophylaxis treatment with ondemand treatment in previously treated patients aged 12-65 years with hem A. The study met its primary endpoint of
prevention of bleeding episodes achieving a 95% reduction in annualized bleed rate (ABR).
2015 Feb: A Phase 3 study was presented at EHAD. The study evaluated the efficacy of BAX 855 as prophylaxis for
bleeding in treatment-experienced hem A patients aged >12 years. The prophylaxis cohort achieved a 95% reduction in
annualized bleed rate (ABR) compared with on-demand therapy.
2014 Dec: FDA BLA for for treatment of hem A.
2014 Aug: Topline results from a Phase 3 study showed that BAX 855 achieved a 95% reduction in ABR when
administered as a twice-weekly prophylaxis compared with on-demand treatment.
2013 Nov: Baxter completes enrollment of a Phase 3 trial to measure ABR for Bax 855 in combination with Advate vs
Advate alone.
Baxalta
Bax 817
Product Description: BAX 817 is an investigational recombinant factor VIIa (rFVIIa) treatment for people with
hemophilia A (hem A) or hemophilia B (hem B). Factor VIIa is one of the several proteins that causes blood to clot.
Class: rFVIII
Treatments (approved or in clinical trial): hem A & B
2015 Mar: Baxter announced positive results from a prospective open-label Phase 3 study. The study was designed to
assess the safety and efficacy of Bax 817 in adult male patients with hem A or hem B and who have developed inhibitors.
The trial met its primary endpoint of successful resolution of acute bleeding of on-demand treatment at 12 hours.
PRODUCT DETAIL – BAXALTA
Product
51
Research and Regulatory Timeline Detail
Baxalta
BAX 930
Product Description: BAX 930 is a recombinant von Willebrand factor-cleaving protease also called ADAMTS13.
ADAMTS13 is a zinc-containing metalloprotease enzyme that cleaves to von Willebrand factor (vWf). vWf is a protein
involved in the blood clotting process. It is secreted in blood and degrades large vWf multimers, decreasing their activity.
Unusually large von Willebrand factor (ULvWF) is present in the blood plasma of patients with hereditary thrombotic
thrombocytopenic purpura (hTTP). BAX 930 may be effective against hTTP due to the fact that it degrades ULVWF.
Class: ADAMTS13
Treatments (approved or in clinical trial): hTTP
2016 May: A Phase 1 study was presented at the International Society on Thrombosis and Haemostatis Scientific and
Standardization Committee meeting (ISTH). The study was a dose escalation safety and pharmacokinetic (PK) profile
study in patients with severe hTTP. The PK profile had a consistent half-life and linear dose response. There were no
serious adverse events.
Baxalta
FEIBA NF
Product Description: FEIBA NF is an anti-inhibitor coagulant complex (AICC) designed to counteract the inhibitor
process. Up to 1/3 of hemophilia patients eventually develop inhibitors in response to rFVIII therapy, neutralizing the
effect of rVIII and preventing the patient’s blood from clotting. It is indicated in the US for control of spontaneous
bleeding episodes or surgical interventions in both hemophilia A & B (hem A and hem B).
Class: AICC
Treatments (approved or in clinical trial): hem A & B
2013 Dec: FDA APP received for use as routine prophylaxis to reduce the frequency of bleeding in hem A & B who
have developed inhibitors.
Baxalta
HyQvia
Product Description: HyQvia is a combination product consisting of human normal immunoglobulin (IGSC) and
recombinant human hyaluronidase. IGSC provides the therapeutic effect for persons suffering primary immunodeficiency
(PI) and some secondary immunodeficiencies (SI). Primary deficiencies are genetically derived while secondary
deficiencies are caused by a variety of conditions, particularly malignancies of the hematopoietic and lymphoreticular
systems.
Class: IGSC Treatments (approved or in clinical trial): Primary immunodeficiency (PI), secondary
immunodeficiencies (SI) caused by myeloma, chronic and lymphocytic leukemia
2016 May: EMA CHMP positive opinion received for the treatment of pediatric PI and SI (the average length of time
from EMA CHMP to approval in the EU for hematology products listed in PDD is 3 months).
2014 Jul: FDA ACR received for the treatment of PI.
2014 May: The FDA announced that it was extending the review period for the BLA.
2013 Dec: Baxter submitted an amended FDA BLA for the treatment of PI in adults.
PRODUCT DETAIL – BAXALTA
Product
52
Research and Regulatory Timeline Detail
Baxalta
Obizur
Product Description: OBI-1 (Obizur) is a porcine factor VIII (pdFVIII) which is effective in achieving hemostatic VIII levels
in patients who have developed inhibitors to human recombinant factor VIII (rFVIII). Patients do not appear to develop
inhibitors to pdFVIII and it appears to be effective in acquired hemophilia A (hem A).
Class: pfFVIII
Treatments (approved or in clinical trial): acquired hem A
2015 Nov: EMA APP received for the treatment of bleeding episodes in adults with acquired hem A caused by antibodies to
FVIII.
2015 Oct: Canadian APP received for the treatment of bleeding episodes in adults with acquired hem A caused by antibodies
to FVIII.
2015 Jul: EMA CHMP positive opinion received for the treatment of bleeding episodes in adults with acquired hem A caused
by antibodies to FVIII.
2014 Oct: FDA APP received for the treatment of bleeding episodes in adults with acquired hem A.
2013 Dec: FDA BLA for acquired hem A.
2013 Dec: A Phase 2/3 study was presented at ASH. The study showed that OBI-1 was effective in treating bleeding in
patients with acquired hem A.
Baxalta
Oncaspar
Product Description: Pegaspargase (Oncaspar) is a pegylated form of the enzyme asparaginase. Asparaginase acts by
enzymatic degradation of the amino acid asparagine. Asparagine depletion in the blood leads to apoptosis of cells highly
dependent on asparagine, especially leukemic blasts. Pegylation of asparaginase prolongs half-life and reduces
immunogenicity making pegaspargase more effective in destroying cancer cells.
Class: asparagine inhibitor Treatments (approved or in clinical trial): acute lymphoblastic leukemia (ALL)
2016 Jan: EMA APP received for use in combination therapy for pediatric and adult patients with ALL.
2015 Nov: EMA CHMP positive opinion received for use in combination therapy for pediatric and adult patients with ALL.
Baxalta
Rixubis
Product Description: Rixubis is a recombinant factor IX (rFIX) protein used for prophylaxis and control of bleeding episodes
in adults with hemophilia B (hem B). Factor IX is also called the “Christmas” factor and is a serine proteases of the
coagulation system. hem B patients have a genetic mutation in the IX gene leading to deficiency in factor IX. Rixubis replaces
this factor.
Class: rFIX
Treatments (approved or in clinical trial): hem B
2014 Dec: EMA MAA for the treatment of hem B in children and adults (the average length of time from submission to
approval in the EU for hematology products listed in PDD is 16 months)..
2014 Sep: FDA APP received for use as routine prophylactic treatment, control, and prevention in children with hem B.
2013 Dec: FDA sNDA for hem B in children.
2013 Oct: EMA MAA for the treatment and prophylaxis of bleeding in adults and children with hem B.
PRODUCT DETAIL – BAXALTA
Product
53
Research and Regulatory Timeline Detail
Baxalta
Vonvendi
Bax 111
Product Description: Vonvendi (Bax 111) is a recombinant von Willebrand factor (rVWF) under investigation for the
treatment of bleeding episodes in patients with the most common bleeding disorder, von Willebrand disease (VWD).
VWD patients either produce too little VWF or their VWF is defective, resulting in problems of clot formation
particularly in the GI system. Bax 111 has orphan drug status in both the EU and US.
Class: rVWF
Treatments (approved or in clinical trial): VWD
2015 Dec: FDA APP received for the treatment of VWD in adults.
2015 Aug: A Phase 3 study was published in Blood. The study evaluated the efficacy, safety, and pharmacokinetics of
Vonvendi in patients with severe VWD. The study achieved its primary endpoint of patients experiencing successful
treatment of bleeding episodes on a 4 point scale. Vonvendi achieved a score of 2.5 or greater in 100% of events.
2015 Apr: Baxter presented data on a Phase 3 study at HTRS. The study met its primary endpoint which was defined as
the number of patients who achieved successful control of bleeding episodes (100% of patients).
2014 Dec: FDA BLA for treatment for patients with VWD.
2014 Apr: Baxter released topline results from a Phase 3 study evaluating the safety, efficacy and pharmacokinetics of
Bax 111. Bax 111 met its primary efficacy endpoint as 100% of patients in the study achieved pre-specified success in
on-demand treatment for bleeding events.
Baxter
EU: Vivia
US: Amia
Product Description: Vivia/Amia is a high dose (HD) dialysis machine used in patients with end-stage kidney disease
(ESKD). ESKD patients generally receive conventional hemodialysis (CHD) which is usually performed in a clinic three
times per week for three to five hours at a time. Vivia offers a HD therapy which is performed more frequently; short
daily treatments five days per week for session of less than four hours. Vivia is designed to be operated by the patient and
therefore can be done at home. HD therapy is associated with improved survival and quality of life improvements over
CHD.
Class: hemodialysis
Treatments (approved or in clinical trial): ESKD
2014 Oct: FDA APP received for the treatment of ESKD.
2014 Jun: Data from two Phase 3 studies was presented at ERA-EDTA. Both studies showed that Vivia demonstrated
acceptable clearance or uremic toxins and an overall similar safety profile compared with conventional hemodialysis.
2013 Dec: EMA CE received for use with ESKD patients.
PRODUCT DETAIL – BAXALTA, BAXTER
Product
54
Research and Regulatory Timeline Detail
Bayer
Kovaltry
Product Description: BAY 81-8973 (Kovaltry) is a full-length recombinant factor VIII (rFVIII) compound for the
treatment of hemophilia A (hem A)in both children and adults. BAY 81-8973 has demonstrated efficacy as a
prophylaxis 2 to 3 times a week.
Class: rFVIII
Treatments (approved or in clinical trial): hem A
2016 Mar: Approved in Japan for the treatment of hem A.
2016 Mar: FDA APP received for the treatment of hem A in children and adults.
2016 Feb: EMA APP received for use as prophylaxis in children and adults with hem A.
2016 Feb: Approved in Canada for the treatment of hem A.
2015 Dec: EMA CHMP positive opinion received for treatment and prophylaxis in children and adults with hem A (the
average length of time from EMA CHMP to approval in the EU for hematology products listed in PDD is 3 months).
2015 Jun: Bayer submitted an application for marketing authorization for the treatment of hem A in Japan.
2014 Dec: FDA BLA submission for hem A prophylaxis in adults and children.
2014 Dec: EMA MAA for use as prophylaxis of hem A in children and adults.
Bayer
damoctocog
BAY 94-9027
Product Description: Damoctocog is a site-specific PEGylated recombinant factor VIII (rFVIII). It is intended to slow
down the clearance of factor VIII from the blood stream, thus extending its plasma half-life.
Class: rFVIII
Treatments (approved or in clinical trial): hem A
2014 May: The Phase 3 PROJECT study was presented at WFH. PROJECT studied previously treated adults and
adolescents with severe hem A. The study met its primary endpoint of protection from bleeds with fewer infusions.
Bayer
EU: Kogenate
US: Kogenate FS
Product Description: Octocog (Kogenate) alpha is a recombinant factor VIII (rVIII) compound. It is approved for
prophylaxis of bleeding in adults and children with hem A. It is approved in over 90 countries including the US and the
EU for prophylaxis of bleeding.
Class: rFVIII
Treatments (approved or in clinical trial): hem A
2014 Dec: The Phase 3 SPINART study was published in Blood. The SPINART study looked at the efficacy of
secondary prophylaxis vs on-demand therapy with octocog in patients with severe hem a. The study showed that the
prophylaxis cohort had less pain, were more active, and had better treatment outcomes than the on-demand cohort.
2014 May: FDA APP received for use as routine prophylaxis in adults with hem A.
2013 Dec: EMA CHMP determined that there is no evidence to indicate an increased risk of FVIII inhibitors in patients
who take Kogenate. The CHMP ruled the benefits of Kogenate outweigh the risks.
PRODUCT DETAIL – BAYER
Product
55
Research and Regulatory Timeline Detail
Biogen
Alprolix
Product Description: Alprolix, an rFIXFc, is a long-acting recombinant clotting factor IX developed using Biogen’s
new monomeric Fc fusion technology. The new technology makes use of a naturally occurring pathway that delays the
destruction of factor IX consequently leading to a longer circulating half-life. This should reduce the treatment burden for
hem B patients.
Class: rFIX Treatments (approved or in clinical trial): hem B
2016 May: EMA APP received for on-demand and prophylaxis treatment of hem B.
2016 Feb: EMA CHMP positive opinion received for the treatment of hem B (the average length of time from EMA
CHMP to approval in the EU for hematology products listed in PDD is 3 months).
2015 Dec: A post-hoc analysis of the B-LONG study was presented at ASH. The analysis showed that patients taking
Alprolix prophylactically had reduced frequency of bleeding episodes overall and in target joints.
2015 Aug: Results from the B-YOND extension study were presented at the NHF meeting. The study showed that
people with severe hem B who were treated with Alprolix with one to two prophylaxis regimens experienced low
bleeding rates for up to two years.
2015 Jun: EMA MAA for the treatment of hem B (the average length of time from submission to approval in the EU for
hematology products listed in PDD is 16 months).
2015 Feb: Topline results for the Phase 3 Kids B-LONG study were released. The study focused on children under 12
with severe hem B who were given an once-weekly prophylaxis dosing of Alprolix. The study showed that Alprolix was
well-tolerated, achieved low bleeding rates, and no inhibitors were detected.
2014 Mar: FDA APP received for use in the control and prevention of bleeding, perioperative management and routine
prophylaxis in adults and children with hem B.
2014 Mar: Approved in Canada for use as control and prophylaxis in adolescents and adult patients with hem B.
2013 Dec: The FDA asked for more data and extended the review date by 3 months.
PRODUCT DETAIL – BIOGEN
Product
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Research and Regulatory Timeline Detail
Biogen
EU: Elocta
US: Eloctate
Product Description: rFVIIIFc (Elocta/Eloctate) is a long-acting recombinant clotting factor VIII developed using Biogen’s
new monomeric Fc fusion technology. The new technology makes use of a naturally occurring pathway that delays the
destruction of factor VIII consequently leading to a longer circulating half-life. This should reduce the treatment burden for
hem A patients.
Class: rFVIII
Treatments (approved or in clinical trial): hem A
2015 Dec: A post-hoc analysis of the ASPIRE study was presented at ASH. The analysis showed that patients taking Eloctate
prophylactically had fewer annualized bleeding rates overall and in target joints.
2015 Nov: EMA APP received for the treatment of hem A.
2015 Sep: EMA CHMP positive opinion issued for the treatment of hem A.
2015 Aug: The Phase 3 ASPIRE study was published in Haemophilia. The study showed that people on extended-interval
prophylaxis regimens of Eloctate experienced low bleeding rates and no inhibitors.
2014 Oct: EMA MAA for the treatment of hem A.
2014 Jun: FDA APP received for the control and prevention of bleeding, perioperative management, and routine prophylaxis
in adults and children with hem A.
2014 Apr: Topline data from the Phase 3 Kids A-LONG study was released. The data showed that the administration of
twice-weekly Eloctate as prophylaxis in children with severe hem A was well-tolerated, no inhibitors were detected, and a low
bleeding rate was maintained.
2013 Nov: Results from the Phase 3 A-LONG was study published in Blood.
PRODUCT DETAIL – BIOGEN
Product
57
Research and Regulatory Timeline Detail
BioMarin
BMN270
Product Description: BMN270 is a gene therapy AAV5 factor VIII vector, designed to restore factor VIII in hemophilia A.
Gene therapy works by inserting a functional gene, containing a DNA sequence, which acts as a delivery mechanism
providing a functional gene to the cells. The cells then use the functional gene to produce functional factor VIII.
Class: AAV5 FVIII Treatments (approved or in clinical trial): hem A
2016 Mar: EMA COMP for the treatment of severe hem A.
2016 Mar: FDA ODD received for the treatment of hem A.
Boehringer
Ingelheim
volasertib
Product Description: Volasertib is a dihydropteridine Polo-like kinase 1 (Plk1) inhibitor. Plk1 is an enzyme that regulates
cell division (mitosis). Volasertib induces selective G2/M arrest followed by apoptosis in a variety of tumor cells while
causing reversible cell arrest at the G1 and G2 stages without apoptosis in normal cells.
Class: Plk1
Treatments (approved or in clinical trial): acute myeloid leukemia (AML)
2014 Jul: A Phase 2 study was published in Blood. The study results showed that previously untreated AML in patients aged
≥65 years and ineligible for intensive remission induction therapy, treated with volasertib+cytarabine lived significantly
longer than those treated with cytarabine alone.
2014 Apr: EMA COMP and FDA ODD received for the treatment of AML.
2013 Sep: FDA BTD status granted for the treatment of AML.
BMS
Sprycel
Product Description: Dasatinib (Sprycel) is a multi-kinase inhibitor (MKI), including BCR/ABL and Src tyrosine kinase
inhibitor (TKI). TKIs inhibit the protein signaling that causes cell growth. They are therefore effective in stopping or slowing
growth in a variety of tumor types. Dasatinib has been shown to overcome the resistance to imatinib in chronic myeloid
leukemia (CML) cells which have BCR-ABL kinase mutations.
Class: MKI
Treatments (approved or in clinical trial): Philadelphia+ (PH+) acute lymphoblastic leukemia (ALL) ,
+
PH chronic myeloid leukemia (CML), prostate cancer (PC)
2016 Jul: JCO published 5-year follow-up data from the Phase 3 DASISION study. The study compared 1st line therapy
dasatinib with imatinib in patients with PH+ chronic phase myeloid leukemia (CP-CML). Final results continue to support
dasatinib therapy as safe and effective 1st line therapy for long-term CML-CP.
2013 Dec: Four-year follow up data from the Phase 3 DASISION study was presented at ASH. At Year 4, 76% of dasatinib
patients had major molecular response compared with 63% for imatinib.
PRODUCT DETAIL – BIOMARIN, BI, BMS
Product
58
Research and Regulatory Timeline Detail
BMS/AbbVie
Empliciti
Product Description: Elotuzumab (Empliciti) is a monoclonal antibody CS1 antigen agonist. Elotuzumab binds to the
CS1 antigen, triggering an antibody response to cells expressing CS1. CS1 is a glycoprotein and is highly expressed by
multiple myeloma cells but minimally expressed by normal cells. Elotuzumab therefore causes an immunological
response to the presence of multiple myeloma cells leading to their destruction.
Class: CS1 agonist
Treatments (approved or in clinical trial): multiple myeloma (MM)
2016 Jun: Blood published a Phase 2 proof-of-concept study that compared elotuzumab plus bortezomib and
dexamethasone (Ebd) with bortezomib and dexamethasone (Bd) in patients with relapsed/refractory MM. The primary
endpoint was progression-free survival (PFS). Median PFS was longer in the Ebd cohort 9.7 months versus the Bd cohort
6.9 months. There was also a difference in PFS for the Ebd cohort between patients with homozygous for the highaffinity FcyRIIIa allele (22.3 months) versus 9.8 months for Ebd patients homozygous for the low-affinity allele.
2016 May: EMA APP received for use in combination with lenalidomide and dexamethasone, for the treatment of MM
in patients who have received at least one prior therapy (2nd line).
2016 Feb: EMA CHMP positive opinion received for use in combination with lenalidomide and dexamethasone in MM
patients who have received at least one prior therapy (the average length of time from EMA CHMP to approval in the
EU for hematology products listed in PDD is 3 months).
2015 Dec: Extended follow-up data from ELOQUENT-2 was presented at ASH. The analysis demonstrated a 44%
relative improvement in progression-free survival at 3 years.
2015 Nov: FDA APP received for the treatment of MM in combination with lenalidomide and dexamethasone in patients
who have received one to three prior therapies.
2015 Sep: FDA PRD status received for use as combination therapy in MM patients who have received one or more prior
therapies.
2015 Jul: EMA MAA for use as combination therapy in MM patients who have received one or more prior therapies.
2015 Jun: The Phase 3 ELOQUENT-2 study was published in NEJM. The study evaluated elotuzumab, in combination
with lenalidomide and dexamethasone versus lenalidomide+dexamethasone alone for relapsed refractory MM. The study
met its co-primary endpoints of progression-free-survival and overall response rate.
2014 May: FDA BTD status received for use in combination with lenalidomide and dexamethasone for the treatment of
MM in patients who received one or more prior therapies.
PRODUCT DETAIL – BRISTOL-MYERS SQUIBB
Product
59
Research and Regulatory Timeline Detail
BMS
Yervoy
Product Description: Ipilimumab (Yervoy) is a monoclonal antibody that assists the immune system in fighting certain types of
cancers by targeting the CTLA-4 protein receptor on T-cells. CTLA-4 acts as a down regulator to T-cell response. Ipilimumab
binds to the CTLA-4 expressed by T-cells, interfering with the downregulation of T-cell response to cancer cells. US approval
for late-stage melanoma that cannot be removed by surgery. EU Approval for 2nd line metastatic melanoma.
Class: CTLA-4 inhibitor Treatments (approved or in clinical trial): non-small cell lung cancer (NSCLC) , small-cell lung
cancer (SCLC), bladder cancer, prostate cancer (PC), melanoma, renal cell carcinoma (RCC), acute myeloid leukemia (AML)
2016 Jul: NEJM published an independently funded Phase 1/1b study that evaluated the safety and efficacy of ipilimumab in
patients with relapsed hematologic cancer after allogenic hematopoietic stem-cell transplantation (HSCT). Durable responses
were observed in several subtypes of hematological cancers including extramedullary AML.
BMS
Opdivo
Product Description: Nivolumab (Opdivo) is an immunomodulator which blocks the ligand activation of program cell death 1
(PD-1) receptor on T-cells. Nivolumab blocks a regulator of T-cells (PD-1). PD-1 inactivates T-cells as a preventive measure to
avoid overreaction of the immune system. However, this action inhibits immune response to cancer cells. Nivolumab blocks
PD-1 thus allowing the immune system to better attack certain types of tumors.
Class: PD-1 inhibitor
Treatments (approved or in clinical trial): metastatic melanoma (mM), metastatic non-small
cell lung cancer (NSCLC), Hodgkin’s lymphoma (HL), renal cell carcinoma (RCC)
2016 Jun: The Phase 2 CHECKMATE-205 study was presented at European Society of Hematology (ESH). CHECKMATE205 was a single-arm registry study evaluating the safety and efficacy of nivolumab in patients with classic HL. Results from
Cohort B included patients who had relapsed or progressed after autologous hematopoietic stem cell transplantation (autoHSTC) and post-transplantation brentuximab vedotin therapy. The study met its primary endpoint with an objective response
rate of 66.3%.
2016 May: FDA APP received for the treatment of patients with classic HL who have relapsed or progressed after autologous
hematopoietic stem cell transplantation.
2016 Apr: FDA sBLA for 2nd line classical HL (the average length of time from FDA sBLA to approval in the US for
hematology products listed in PDD is 7 months).
2016 Apr: EMA MAA for use as 2nd line therapy in classical HL (the average length of time from EMA MAA to approval in the
EU for hematology products listed in PDD is 15 months).
2014 Dec: The Phase 1b CHECKMATE-039 study was published in NEJM. The study showed a high level of response in
patients with relapsed or refractory classical HL with an overall response rate of 87%.
2014 May: FDA BTD for HL after failure of autologous stem cell transplant and brentuximab.
PRODUCT DETAIL – BMS
Product
60
Research and Regulatory Timeline Detail
Cellectar
CLR-131
Product Description: CLR-131 is a small-molecule, broad-spectrum, cancer-targeted radiopharmaceutical. It is engineered to
combine an “intracellular radiation” mechanism with targeted delivery to a wide range of solid and hematological cancers. It also
selectively uptakes in cancer stem cells, potentially offering longer lasting cancer remission.
Class: radiopharmaceutical Treatments (approved or in clinical trial): multiple myeloma (MM)
2015 Jan: Topline data from a Phase 1 dosing study was released. The study evaluated the safety, tolerability, and efficacy of
CLR-131 in patients with relapsed/refractory MM. The study showed that CLR-131 was well safety, well-tolerated, and
demonstrated some efficacy in relapsed/refractory MM.
Celgene
Vidaza
Product Description: Azacitidine CC-486 (Vidaza) is an epigenetic agent used to treat MDS. The mechanism of action is not
fully understood but it is a chemical analogue of the cytosine nucleoside used in DNA and RNA. It is thought to induce
antineoplastic activity by two mechanisms; 1) inhibition of DNA methyltransferase (at low doses) thereby blocking DNA
methylation (which may activate tumor suppressor genes silenced by hyper methylation), and 2) direct cytotoxicity in abnormal
hematopoietic cells in bone marrow by direct incorporation into both DNA and RNA (at high doses) causing cell death.
Class: epigenetic Treatments (approved or in clinical trial): myelodysplastic syndrome (MDS), acute myeloid leukemia
(AML), chronic myelomonocytic leukemia (CMML), refractory anemia (RA)
2015 Oct: EMA APP received for the treatment of AML in patients ≥65 years who are not eligible for hematopoietic stem cell
transplantation.
2015 Sep: EMA CHMP positive opinion received for an expanded indication. The expanded indication includes the treatment of
patients ≥65 years with AML who are not eligible for hematopoietic stem cell transplantation.
2013 Dec: A Phase 1/2 study was presented at ASH. The study evaluated patients with high-risk MDS. There was a 48%
overall response rate to azacitidine with 17% of patients achieving complete remission resulting in a lower risk of acute AML.
PRODUCT DETAIL – CELLECTAR, CELGENE
Product
61
Celgene
Revlimid
Continued
on next page
Research and Regulatory Timeline Detail
Product Description: Lenalidomide (Revlimid) is a thalidomide derivative product. It has multiple modes of action with three
modes of activity; 1) direct anti-tumor effect, 2) inhibition of angiogenesis, and 3) it is an immunomodulator. It directly induces
tumor cell apoptosis as well as prohibiting angiogenesis, thus starving tumor cells of blood.
Class: IMiDs
Treatments (approved or in clinical trial): multiple myeloma (MM), myelodysplastic syndromes (MDS),
non-Hodgkin's lymphoma (nHL), chronic lymphocytic leukemia (CLL), pancreatic cancer (PAN), mantle-cell lymphoma
(MCL), follicular lymphoma (FL), diffuse large b-cell lymphoma (DLBCL), smoldering multiple myeloma (SMM)
2016 Jul: EMA APP received for the treatment of relapsed/refractory MCL.
2016 Jun: An independent meta-analysis was presented at ASCO. The meta-analysis was of three studies of multiple myeloma
patients treated with lenalidomide after high-dose melphalan and autologous stem cell transplant, At a median follow-up of 80
months median overall survival had not yet been reached in patients receiving lenalidomide maintenance therapy, representing an
estimated 2.5 year benefit over the control cohorts.
2016 May: (see BMS: elotuzumab) EMA APP received for elotuzumab in combination with lenalidomide.
2016 Jan: Lenalidomide is included in a new approved sNDA for carfilzomib (see Amgen: carfilzomib).
2015 Dec: Approved in Japan in for use in combination with dexamethasone as 1st line therapy in MM.
2015 Nov: NEJM published a Phase 2 study. The study evaluated the efficacy and safety of lenalidomide plus rituximab in
treatment-naïve MCL patients. The primary endpoint was overall response rate (ORR). The study met its primary endpoint with
an ORR of 92% at 30 months.
2015 Jun: Updated data from the SPRINT study was presented at EHA. The study compared lenalidomide with investigator’s
choice in patients with relapsed/refractory MCL. The study’s primary endpoint, progression-free survival (PFS), was 8.7 months
in the lenalidomide cohort.
2015 Jun: Updated data from the FIRST study was presented at EHA. Updated PFS for continuous
lenalidomide+dexamethasone was 26 months.
2015 Feb: EMA APP received for continuous oral treatment with lenalidomide in adults with previously untreated MM who are
not eligible for stem cell transplantation.
2015 Feb: FDA APP received for an expanded indication to include newly diagnosed MM in combination with dexamethasone.
2014 Dec: (see Amgen: carfilzomib) ASPIRE STUDY published in NEJM.
2014 Dec: EMA CHMP positive opinion received for use as continuous oral treatment with lenalidomide in adults with
previously untreated MM who are not eligible for stem cell transplantation.
2014 Dec: The Phase 2 SPRINT study was presented at ASH. The study met its primary endpoint with PFS of 8.7 months for
lenalidomide compared with 5.2 months for investigator’s choice.
2014 Dec: Results from a Phase 3 study were presented at ASH. The study compared lenalidomide with placebo in patients with
red-blood cell transfusion dependent low-risk MDS who were unresponsive or refractory to erythropoietin stimulating agents and
did not have a deletion 5q cytogenic abnormality. The study met its primary endpoint of transfusion independence with a median
transfusion independence of 8.2 months.
PRODUCT DETAIL – CELGENE
Product
62
Celgene
Revlimid
Continued
Research and Regulatory Timeline Detail
2014 Sep: The Phase 3 FIRST study was published in NEJM. The study evaluated three different regimens; 1) continuous
lenalidomide+low-dose dexamethasone, 2) a fixed course of lenalidomide+low-dose dexamethasone, and 3) melphalan,
prednisone and thalidomide in patients with newly diagnosed MM. The continuous dose of lenalidomide+low-dose
dexamethasone met its primary endpoint with PFS of 25.5 months vs 20.7 months in the second cohort and 21.2 months in the
third cohort.
2014 Sep: NEJM published a Phase 3 study. The study investigated the efficacy of high-dose consolidation therapy in
treatment-naïve MM patients aged <65 years compared with MPR therapy. The study also evaluated maintenance therapy with
lenalidomide after consolidation therapy. The study showed that lenalidomide maintenance therapy significantly improved
PFS.
2014 Aug: A Phase 2 study was published in Journal of Clinical Oncology . The study evaluated the combination of
lenalidomide with R-CHOP therapy in patients with untreated DBBCL. 98% of patients had an overall response rate and 80%
had a complete response rate.
2013 Dec: A retrospective analysis of patients with IPSS low- or intermediate-1-risk MDS with del(5q) treated with
lenalidomide was presented at ASH. The analysis showed that that 57% of patients achieved complete and partial response.
2013 Aug: The Phase 3 PETHEMA/GEM study was published in NEJM. The study compared lenalidomide+dexamethasone
followed by maintenance therapy with lenalidomide compared with placebo in patients with SMM. The median time to
progression to symptomatic disease was significantly longer in the treatment cohort.
PRODUCT DETAIL – CELGENE
Product
63
Celgene/
Acceleron
luspatercept
Continued
on next page
Research and Regulatory Timeline Detail
Product Description: Luspatercept (ACE-536) is a modified type IIb activin receptor. It acts as a ligand trap for members in
the Transforming Growth Factor-Beta (TGF-β) superfamily involved in the late-stages of red blood cell production
(erythropoiesis). ACE-536 promotes late-stage erythrocyte precursor cell differentiation which promotes red blood cell
production.
Class: TGF-β
Treatments (approved or in clinical trial): myelodysplastic syndromes (MDS), beta-thalassemia (BTH)
2016 Jun: Data from two Phase 2 studies was presented at the European Society of Hematology (ESH). The first study was a
3-month dose escalation study. The second was a long-term extension study. Both studies evaluated the efficacy and safety of
luspatercept in patients with high transfusion burden and low transfusion burden MDS. The primary endpoint for the 3-month
study was the proportion of patients who had an erythroid response (hemoglobin increase ≥1.5 g/dL from baseline for ≥14 days
in non-transfusion dependent patients or a reduction of either ≥4 units or ≥50% of units of RBCs transfused compared with
pretreatment in transfusion dependent patients). The primary endpoint for the long-term extension study was long-term safety
and tolerability in low or intermediate risk MDS patients who were previously enrolled in the 3-month study. Results showed
that 51% of patients with lower risk MDS achieved increased hemoglobin levels and 35% of patients achieved transfusion
independence in the 3-month study. In the extension study, 81% of patients had increased hemoglobin levels and of the patients
eligible for transfusion independence (TI), 50% achieved TI.
2016 Jun: Data from two Phase 2 studies was presented at ESH. The first study was a base dose escalation study and the
second was an ongoing long-term extension study evaluating the efficacy of luspatercept in transfusion dependent and nontransfusion dependent BTH patients. The primary endpoint in the base study was the proportion of patients who had a
erythroid response (hemoglobin increase ≥1.5 g/dL from baseline for ≥14 days in non-transfusion dependent patients or a
≥20% reduction in RBC transfusion burden compared with pretreatment in transfusion dependent patients). The primary
endpoint in the long-term study was safety and tolerability. Results showed that 80% of patients achieved at least a 20%
reduction in transfusion burden and 36% of patients achieved a hemoglobin increase of at least 1.5 g/dL in the dose escalation
study. In the long-term study 96% of patients achieved at least a 20% reduction in transfusion burden and 56% of patients
achieved a hemoglobin increase of at least 1.5 g/dL.
2015 Dec: A Phase 2 study was presented at ASH. The study evaluated the efficacy of luspatercept in patients with BTH. The
study showed that luspatercept increased hemoglobin levels, reduced transfusion burden, improved health-related quality of life
measures, and had beneficial effects on liver iron concentration.
2015 Dec: FDA FTD received for the treatment of anemia in patients with MDS.
2015 Dec: A Phase 2 long-term study was presented at ASH. The study evaluated the efficacy of luspatercept in patients with
lower risk MDS. The study showed that patients treated with luspatercept maintained increased hemoglobin and transfusion
independence.
2015 Jun: Two Phase 2 studies were presented at EHA. The first study evaluated luspatercept in patients with lower risk MDS
and showed that luspatercept produced sustained hemoglobin increases and transfusion independence for more than six months
in some patients. The second study evaluated luspatercept in patients with non-transfusion and transfusion dependent BTH.
Luspatercept demonstrated increased hemoglobin levels, reduced transfusion burden, and reduced iron load.
2015 May: FDA FTD received for transfusion dependent and non-transfusion dependent BTH.
PRODUCT DETAIL – CELGENE
Product
64
Research and Regulatory Timeline Detail
Celgene/
Acceleron
Luspatercept
Continued…
2015 May: Preliminary data from the Phase 2 PACE-MDS study were presented at ISMDS. The study evaluated luspatercept
in patients with lower-risk MDS. 54% of patients achieved the IWG hematologic improvement-erythroid threshold for efficacy
and 36% of patients who received red blood transfusions prior to treatment achieved transfusion independence.
2014 Dec: Interim data from a Phase 2 study was presented at ASH. The study showed that luspatercept improved anemia and
transfusion burden, and reduced iron overload in patients with BTH.
Celgene
EU: Imnovid
US:
Pomalyst
Product Description: Pomalidomide (Imnovid/Pomalyst) is a thalidomide derivative product. It is an immunomodulator
(IMiDs) which is a class of drugs that include thalidomide and its derivative analogues. It’s exact mode of action is not fully
understood. However, it has antiangiogenic properties that inhibit tumor angiogenesis and promotes cell cycle arrest in
susceptible tumor cells.
Class: IMiDs
Treatments (approved or in clinical trial): multiple myeloma (MM)
2015 May: Blood published the results of a Phase 2 study. The study compared PomDex (pomalidomide plus low-dose
dexamethasone) with PomCyDex (pomalidomide plus cyclophosphamide and low-dose dexamethasone) therapy in patients
with lenalidomide refractory MM. The study met its primary endpoint of overall response rate (ORR) with the PomDex cohort
achieving an ORR of 38.9% and the PomCyDex cohort achieving an ORR of 64.7%.
2014 Feb: Approved in Canada for the treatment of MM when previous therapy with lenalidomide and bortezomib has failed.
2013 Dec: A retrospective analysis of the MM-033 study was presented at ASH. The retrospective analysis maintained results
published earlier in the year.
2013 Sep: The Phase 3 MM-003 study was published in Lancet Oncology. The study compared pomalidomide+low-dose
dexamethasone with pomalidomide+high-dose dexamethasone in patients with relapsed and refractory multiple myeloma
(rrMM). The low-dose combination met its primary endpoint of progression-free survival (PFS).
2013 Aug: EMA APP received for the use of pomalidomide in combination with dexamethasone for the treatment of rrMM in
adults who have received at least two prior therapies and have demonstrated disease progression with the last therapy.
PRODUCT DETAIL – CELGENE
Product
65
Research and Regulatory Timeline Detail
Celgene/
Acceleron
sotatercept
Product Description: Sotatercept is a type IIa activin receptor. That acts as a ligand trap for members in the Transforming
Growth Factor-Beta (TGF-β) superfamily involved in the late-stages of red blood cell production (erythropoiesis). Sotatercept
promotes late-stage erythrocyte precursor cell differentiation which promotes red blood cell production.
Class: TGF-β
Treatments (approved or in clinical trial): myelodysplastic syndromes (MDS), beta-thalassemia
(BTH), end-stage kidney disease (ESKD)
2014 Nov: Interim results from a Phase 2 study was presented at CAN. The study demonstrated encouraging effects of
sotatercept on vascular calcification, bone mineral density, and hemoglobin levels in patients with ESKD.
2014 Apr: Interim date from a Phase 2 study was presented at the NKF meeting. Sotatercept demonstrated a dose dependent
increase in hemoglobin in ESKD patients on hemodialysis.
2013 Dec: Interim data from a Phase 2 study was presented at ASH. The data demonstrated dose dependent increases in
hemoglobin in non-transfusion dependent BTH patients.
Coherus
pegfilgrastim
biosimilar
CH-1701
Product Description: CH-1701 is a pegfilgrastim biosimilar version of Amgen Neulasta. Pegfilgrastim is a pegylated form of
granulocyte colony-stimulating factor (GCSF) analog filgrastim. Pegfilgrastim stimulates bone marrow to produce more white
blood cells.
Class: GCSF Treatments (approved or in clinical trial): chemotherapy induced neutropenia
2016 Jul: Topline data from a follow-on pharmacokinetic and pharmacodynamic clinical study was released. The study
compared CH-1701 with Neulasta (pegfilgrastim). The study met its primary endpoint of non-inferiority with Neulasta.
Collegium
Xtampza ER
Product Description: Oxycodone extended release capsule (Xtampza ER) is an opioid agonist designed for management of
severe pain requiring around-the-clock long-term opioid treatment. It uses a unique formulation which deters abusive use of the
product.
Class: opioid Treatments (approved or in clinical trial): severe pain
2016 Apr: FDA APP received for the management of severe pain requiring daily long-term treatment where non-opioid
alternatives are inadequate.
2015 Nov: FDA APP tentative approval was granted subject to review of a patent infringement case.
2015 Sep: FDA ACR for severe pain.
2015 Aug: Pain published a Phase 3 study. The study compared Xtampza ER to placebo in patients with moderate-to-severe
lower back pain. The primary endpoint was average pain intensity at Week 12. The Xtampza ER cohort achieved significant
improvement in average pain intensity.
2015 Jun: Pain Medicine published a Phase 3 study. The study compared Xtampza ER to OxyContin in healthy individuals.
The primary endpoint was increase in the Abuse Quotient (AQ) from crushing the capsule. Xtampza demonstrated a significant
improvement in AQ with no significant in increase in AQ when the capsule was crushed compared to a four fold increase for
OxyContin.
2015 Dec: FDA NDA for daily long-term use in severe pain.
PRODUCT DETAIL – CELGENE, COHERUS, COLLEGIUM
Product
66
Research and Regulatory Timeline Detail
CSL
Behring
Afstyla
Product Description: Recombinant factor VIII (Afstyla) single-chain is a recombinant Factor VIII. The stability of rFVIII singlechain provides a longer half-life and reduces frequency of dosing. The rVIII single-chain also has an affinity for von Willebrand
factor which plays a role in the drug’s stability and integrity in the blood stream.
Class: rFVIII Treatments (approved or in clinical trial): hemophilia A (hem A)
2016 May: FDA APP for use as routine prophylaxis in adult and pediatric hem A patients.
2015 Dec: EMA MAA for the treatment of hem A (the average length of time from EMA submission to approval in the EU for
hematology products listed in PDD is 15 months).
2015 Jul: FDA BLA for the treatment of hem A (the average length of time from FDA submission to approval in the US for
hematology products listed in PDD is 6 months).
2015 Jun: The Phase 3 AFFINITY study was presented at ISTH. The study compared rFVIII single-chain with octocog alpha as
a prophylaxis in hem A patients. rFVIII single-chain had improved pharmacokinetic parameters compared with octocog and lead
to a low annualized spontaneous bleeding rate.
CSL
Behring
Idelvion
Product Description: Coagulation factor IX recombinant, albumin fusion protein (Idelvion) is a long-acting recombinant factor
IX with recombinant albumin with an extended half-life. The addition of albumin maintains levels of factor IX above 5% for
prolonged periods of time, providing improved bleeding control.
Class: rFIX Treatments (approved or in clinical trial): hemophilia B (hem B)
2016 May: EMA APP for on demand and prophylaxis in hem B.
2016 May: FDA grants 7 years of market exclusivity.
2016 Apr: Blood published the Phase 3 PROLONG-9FP study that compared on-demand Idelvion with routine prophylaxis in
previously treated hem B patients. The median annualized spontaneous bleeding rate (AsBR) for the prophylaxis cohort was zero.
2016 Mar: FDA APP received for use as prophylaxis and on-demand control of hem B in adolescents and adults (aged ≥12
years) and perioperative management of bleeding.
2016 Feb: EMA CHMP positive opinion received for the treatment of hem B (the average length of time from EMA CHMP to
approval in the EU for hematology products listed in PDD is 3 months).
2015 Dec: The Phase 3 extension study PROLONG-9FP was presented at ASH. The median AsBR for the prophylaxis cohort
was zero.
2015 Jun: A Phase 3 study was presented at ISTH. The study compared on-demand treatment with prophylaxis therapy in
previously-treated adolescents and adults. The prophylaxis cohort achieved 100% AsBR in both patients who began with ondemand and switched to prophylaxis and those who began with prophylaxis.
2015 Mar: EMA MAA for the treatment of hem B (the average length of time from EMA submission to approval in the EU for
hematology products listed in PDD is 15 months).
2015 Feb: FDA BLA for the treatment of hem B (the average length of time from FDA submission to approval in the US for
hematology products listed in PDD is 6 months).
2014 May: Interim data from two Phase 3 studies was presented at the WFH congress. The studies demonstrated improved hem B
treatment with a prolonged routine prophylaxis treatment interval of 14 days compared with the current standard.
PRODUCT DETAIL – CSL BEHRING
Product
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Research and Regulatory Timeline Detail
CTI
Biopharma
pacritinib
Product Description: Pacritinib is a multikinase inhibitor with specificity for JAK2 and FLT3. Mutations in these kinases
have been shown to be directly related to the development of a variety of blood-related cancers, including myeloproliferative
neoplasms, leukemia, and lymphoma.
Class: multikinase inhibitor (MKI) Treatments (approved or in clinical trial): myelofibrosis (MF), acute myeloid
leukemia
2016 Jun: Updated data from the Phase 3 PERSIST-1 study were presented at ASCO. The update focused on efficacy and
safety data. The main adverse events were gastrointestinal. A 72-week analysis of efficacy showed that pacritinib led to durable
reductions in spleen volume and symptom burden in patients with MF, including patients with low platelet count.
2016 Feb: FDA places full clinical hold on all clinical studies. These restrictions were put in place due to excess mortality of
pacritinib-treated patients in PERSIST-1. CTI withdrew its NDA until it can analyze data from PERSIST-2.
2016 Jan: FDA NDA filing completed for the treatment of patients with intermediate and high risk myelofibrosis with low
platelet counts (NDA withdrawn).
2015 Dec: A posy-hoc analysis of the PERSIST-1study was presented at ASH. The analysis showed that pacritinib achieved
consistent rates of spleen volume reduction and control of disease-related systems across all intermediate and high-risk
myelofibrosis subgroups.
2015 Nov: FDA NDA for intermediate and high-risk myelofibrosis patients with low platelet counts.
2015 May: The Phase 3 PERSIST-1 study results were presented at ASCO. The study met its primary endpoint of spleen
volume reduction, with statistically significant reductions (≥35%) observed from baseline to Week 24.
2015 Mar: Phase two study results published in Blood showed that pacritinib is active in patients with myelofibrosis, resulting
in spleen volume reduction, while producing substantial and prolonged improvement in disease-related symptoms without
causing clinically significant myelosuppression.
2015 Mar: Positive topline results for the Phase 3 PERSIST-1 study were released showing the study met its primary endpoint
in the intent-to-treat population with statistically significant activity observed in patients irrespective of their initial platelet
count, including patients with very low platelet counts at study entry.
2014 Dec: Results from a Phase 3 study presented at ASH showed that pacritinib preferentially killed AML cells with FLT3
mutations, overcame stromal protection, and suppressed leukemic outgrowth from stroma adherent AML cells in both and
long-term assays.
2014 Dec: Results from the Phase 3 PERSIST-2 study presented at ASH showed a unique kinome profile for pacritinib among
agents in for myelofibrosis and suggests potential therapeutic benefit across a spectrum of blood-related cancers.
2014 June: FDA FTD received for MF.
2013 Dec: An integrated analysis of data from two completed Phase 2 trials was presented at ASH. Pacritinib demonstrated
clinically meaningful spleen size in patients with low platelets.
PRODUCT DETAIL – CTI BIOPHARMA
Product
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Research and Regulatory Timeline Detail
CTI
Biopharma
Pixuvri
Product Description: Pixantrone (Pixuvri) is an anthracycline medication that works by targeting and killing the cancer cells that
are dividing. It interferes with the DNA in the cells, preventing them from making more copies of DNA and producing proteins.
Ultimately when the cells cannot divide, they die.
Class: anthracycline
Treatments (approved or in clinical trial): non-Hodgkin’s lymphoma (NHL)
2014 Jan: Pixantrone received a positive final appraisal from UK NICE which recommended funding the treatment as a
monotherapy for adult patients with multiply relapsed or refractory aggressive B-cell non-Hodgkin’s lymphoma.
2013 Oct: UK NICE rejected funding for pixantrone for the second time.
CTI
Biopharma
tosedostat
Product Description: Tosedostat is an aminopeptidase (APD) inhibitor. It has particular efficacy inhibiting the aminopeptidases
puromycin-sensitive aminopeptidase (PuSA), and leukotriene hydrolase (LTA4). Inhibition of these aminopeptidases may result in
amino acid deprivation. Amino acids are required to make proteins necessary for tumor cell survival. Deprivation of these amino
acids can lead to cell death in certain tumor cells.
Class: APD-inhibitor
Treatments (approved or in clinical trial): acute myeloid leukemia (AML), myelodysplastic
syndrome (MDS)
2015 Dec: A Phase 2 study was presented at ASH. The study evaluated the efficacy of tosedostat in elderly patients with primary
or secondary AML. Tosedostat plus cytarabine achieved a complete response of 48.5%.
2015 Jun: Results from a Phase 2 study were presented at EHA. Results showed that a combination of tosedostat with low dose
cytarabine achieved an overall response rate of 54% in elderly patients who had primary AML or AML evolved from MDS.
2014 Jan: The FDA partial hold order was lifted, allowing all tosedostat studies to continue.
2013 Dec: Two Phase 2 studies were presented at ASH. The studies measured the effectiveness of tosedostat in combination with
cytarabine or decitabine in newly diagnosed patients with AML or high-risk MDS. Tosedostat met its primary objective in both
studies.
PRODUCT DETAIL – CTI BIOPHARMA
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Research and Regulatory Timeline Detail
Curis
CUDC-907
Product Description: CUDC-907 is a dual histone deacetylase (HDAC) and phosphoinositide 2-kinase (PI3K) inhibitor. Histone
wraps around DNA and DNA expression is regulated by acetylation and de-acetylation. Chronic activation of PI3K may
contribute to metastatic cell growth. By inhibiting these two enzymes CUDC-907 may slow down cell division and growth
thereby slowing the growth of tumors and metastatic disease.
Class: HDAC/PI3K
Treatments (approved or in clinical trial): breast cancer (BC), diffuse large B-cell lymphoma
(DLBCL), Hodgkin’s lymphoma (HL), multiple myeloma (MM), NUT midline carcinoma (NMC)
2016 Apr: Lancet Oncology published a Phase 1 study. The study evaluated the efficacy and tolerability of CUDC-907 in
relapsed/refractory DLBCL. CUDC-907 achieved complete and partial responses in heavily pretreated patients.
2015 Dec: A Phase 1 study was presented at ASH. The study evaluated the safety and efficacy of CUDC-907 in patients with
relapsed/refractory DLBCL. The study showed that CUDC-907 achieved complete and partial responses in heavily pretreated
patients.
2015 May: A Phase 1 study was presented at ASCO. The study evaluated dose escalation for CUDC-907 in refractory/relapsed
DLBCL and HL patients. The study demonstrated efficacy for CUDC-907 in both DLBCL and HL patients.
2015 Apr: FDA ODD for DLBCL.
2014 Apr: Preliminary data from a Phase 1 study was presented at AACR. Data suggest a possible correlation between plasma
TARC levels (a chemokine involved in the stimulation of helper T-cells required for the survival of certain malignant blood cells)
and tumor response to CUDC-907.
PRODUCT DETAIL – CURIS
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Research and Regulatory Timeline Detail
Daiichi/
Ambit
quizartinib
Product Description: Quizartinib is a FMS-like tyrosine kinase (FLT3) inhibitor. It selectively inhibits FLT3, colonystimulating factor 1 receptors, stem cell factor receptors, and platelet growth factor receptors which result in the inhibition of
ligand-independent leukemic cell proliferation leading to cell death.
Class: FLT3 inhibitor
Treatments (approved or in clinical trial): acute myeloid leukemia (AML)
2014 Jun: Results from a Phase 2 study was presented at ASCO and EHA. The study evaluated quizartinib in patients with
FLT3-ITD positive relapsed or refractory AML. The composite complete response rate was 47% with the rate of hematopoietic
stem cell transplantation (HSCT) after quizartinib therapy 34%. The median overall survival was 20.9 weeks with the 30 mg
dose and 25.4 weeks with the 60 mg dose.
2013 Dec: A Phase 2b study was presented at ASH. The study showed that both 30 mg and 60 mg doses of quizartinib showed
significant activity in composite complete response rate and bridge to HSCT with improved overall survival.
Egalet
Arymo
Product Description: Egalet-001 (Arymo) is an abuse-deterrent, extended-release, oral morphine formulation.
Class: analgesic Treatments (approved or in clinical trial): moderate to severe pain including lower back pain, arthritis,
headache, and face and jaw pain
2016 May: A Phase 2/3 HAP study was presented at AAPS. The study compared the pharmacokinetic (PK) profile and abuse
potential of Arymo with MS Contin. The study showed that Arymo ER required a higher level of effort to manipulate than MS
Contin, the overall “drug liking” rate was lower (51 on the VAS scale compared to 71 for MS Contin), and the abuse quotient
(AQ) was lower 2.3 – 9.2 for Arymo ER versus 37.2 for MS Contin.
2015 Dec: FDA NDA for the management of pain severe enough to require daily, around-the-clock opioid treatment and for
which alternative treatments are inadequate.
2015 Jun: Topline results from study 067-EG-001, a pivotal pharmacokinetic study, demonstrated the bioequivalence of
Egalet-001 60 mg to MS Contin 60 mg.
2014 Feb: FDA FTD for the treatment of moderate to severe pain.
PRODUCT DETAIL – DAIICHI, EGALET
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Research and Regulatory Timeline Detail
Egalet
Egalet-002
Product Description: Egalet-002 is an abuse-deterrent, extended-release, oral oxycodone formulation.
Class: analgesic Treatments (approved or in clinical trial): moderate to severe pain
2016 Mar: Positive topline results from a study using the ALERRT instrument (a tool to standardize how to quantify the level
of effort required to manipulate an abuse-deterrent product candidate) were announced. Egalet-002 was rated as moderately to
extremely hard to physically manipulate.
2015 Jun: Topline results from an alcohol-interaction study was released. The study compared three alcohol arms plus
Egalet-002 with water plus Egalet-002 in healthy moderate drinkers. There was no evidence of alcohol dose dumping.
2014 Feb: FDA FTD for the treatment of moderate to severe pain.
Eli Lilly
galcanezumab
Product Description: Galcanezumab is a calcitonin gene-related peptide (CGRP) antibody. CGRP is a sensory neuropeptide
with vascular and pro-inflammatory effects, two processes that have been implicated in migraine headaches. Galcanezumab
binds to and inhibits the activity of CGRP, which is released during activation of sensory neurons involved in pain signaling.
Class: CGRP antibody
Treatments (approved or in clinical trial): migraine headache, cluster headache, osteoarthritis
pain, cancer pain, chronic lower back pain
Gilead
entospletinib
Product Description: Entospletinib is a spleen tyrosine kinase (Syk) inhibitor. Entospletinib inhibits B-cell receptor (BCR)
signaling and leads to an inhibition of tumor cell activation, migration, adhesion, and proliferation. Syk, a non-receptor
cytoplasmic, BCR-associated tyrosine kinase, is expressed in hematopoietic tissues and is often overexpressed in
hematopoietic malignancies.
Class: SYK inhibitor Treatments (approved or in clinical trial): non-Hodgkin lymphoma (NHL), chronic lymphocytic
leukemia (CLL), follicular lymphoma (FL)
2016 May: Blood published a Phase 2 study that evaluated the safety and efficacy of idelalisib plus entospletinib in patients
with relapsed/refractory CLL or NHL. 60% and 26% of patients with CLL or FL, achieved the primary endpoint of objective
response rate (ORR). However, the study was terminated early due to increased risk of treatment-emergent pneumonitis.
2015 May: Blood published a Phase 2 study that evaluated the safety and efficacy of entospletinib in patients with
relapsed/refractory CLL. The study met its primary endpoint with 70.1% of patients achieving progression-free survival (PFS)
at 26 weeks. Median PFS was 13.8 months.
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Product Description: Idelalisib (Zydelig) is a phosphoinositide 3-kinase delta (PI3K) inhibitor. PI3K is important for the
activation and proliferation of B lymphocytes. PI3K delta signaling is especially hyperactive in many B-cell leukemias and
lymphomas.
Class: PI3K inhibitor
Treatments (approved or in clinical trial): chronic lymphocytic leukemia (CLL), indolent nonHodgkin’s lymphoma (iNHL), mantle cell lymphoma (MCL), follicular B-cell non-Hodgkins lymphoma (FL), small
lymphocytic lymphoma (SLL)
2016 Jul: Blood published a Phase 2 study. The study evaluated the safety of idelalisib as up front therapy in patients with
relapsed/refractory CLL. Patients were given idelalisib for 56 days, followed by 6 months of idelalisib plus ofatumumab,
followed by idelalisib alone indefinitely. The study found that upfront treatment with idelalisib lead to hepatotoxicity in 54% of
patients.
2015 Dec: An interim analysis of the Phase 3 STUDY 115 was presented at ASH. STUDY 115 compared idelalisib plus
bendamustine+rituximab with bendamustine+rituximab alone in patients with previously treated CLL. The study met its
primary endpoint with a 67% reduction in the risk of disease progression or death.
2015 Nov: The Phase 3 STUDY 115 was ended early due to highly significant efficacy. The tri-therapy cohort demonstrated
significant improvement in progression-free survival (PFS).
2015 May: The Phase 3 STUDY 119 was presented at ASCO. STUDY 119 evaluated idelalisib in combination with
in previously-treated patients with CLL. The combination cohort achieved a 73% reduction in the risk of disease progression
or death compared with ofatumumab alone.
2014 Dec: Follow-up data from STUDY 101-09 and STUDY 116 was presented at ASH. STUDY 101-09 showed a 58%
response rate with a median duration of response of 12.5 months. STUDY 116 showed median PFS in the combination cohort
of 19.4 months.
2014 Sep: EMA APP received for the treatment of CLL in combination with rituximab in patients who have received at least
one prior treatment or as 1st line therapy in patients with 17p deletion or TP53 mutation, and FL in patients who are refractory
to two prior lines of treatment.
2014 Jul: EMA CHMP positive opinion received for the treatment of CLL in combination with rituximab in patients who have
received at least one prior treatment or as 1st line therapy in patients with 17p deletion or TP53 mutation, and FL in patients
who are refractory to two prior lines of treatment.
2014 Jul: FDA APP received for the treatment of relapsed CLL in combination with rituximab, for relapsed FL, and for SLL
in patients who have received at least two prior systemic therapies.
2013 Mar: STUDY 116 & STUDY 101-09 results were published in NEJM.
2013 Dec: FDA NDA submission for the treatment of CLL in combination with rituximab in patients who have received at
least one prior therapy.
2013 Dec: Phase 2 STUDY 101-09 results were presented at ASH. The study evaluated idelalisib in patients with iNHL that is
refractory to rituximab and to alkylating-agent-containing chemotherapy. Idelalisib achieved an overall response rate (ORR) of
57% with a median duration of response of 12.5 months.
2013 Dec: An interim analysis of STUDY 116 was presented at ASH.
PRODUCT DETAIL – GILEAD
Gilead
Zydelig
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Research and Regulatory Timeline Detail
Gilead
Zydelig
continued
2013 Oct: Gilead stopped STUDY 116 early due to the fact that an interim analysis showed highly significant efficacy. The
Phase 3 STUDY 116 compared idelalisib+rituximab with rituximab alone in previously-treated CLL patients who were not fit for
chemotherapy. The combination therapy achieved highly significant efficacy for the primary endpoint of PFS.
2013 Sep: FDA NDA submitted for the treatment of iNHL that is refractory to rituximab and to alkylating-agent-containing
chemotherapy.
GSK
Strimvelis
Product Description: GSK2696273 (Strimvelis) is a hematopoietic stem cell therapy that uses the patient’s own bone marrow.
The patient’s bone marrow is removed and a vector is used to insert a copy of the ADA gene into the stem cells in a procedure
called transduction. The gene-corrected cells are then re-introduced int the patient
Class: hematopoietic Treatments (approved or in clinical trial): severe combined immunodeficiency due to adenosine
deaminase deficiency (ADA-SCID)
2016 Apr: EMA CHMP positive opinion received for the treatment of ADA-SCID (the average length of time from EMA
CHMP to approval in the EU for hematology products listed in PDD is 3 months).
GW Pharma
Sativex
Product Description: Nabiximols (Sativex) is a cannabinoid extracted from the cannabis plant. Its two active ingredients are
tetrahydrocannabinol (THC) and cannabidiol (CBD). Both chemicals act as ligands, binding to specific receptors THC has a
weak affinity to binds to cannabinoid type 1 (CB1) receptors in the brain while CBD has a weak affinity to bind to cannabinoid
type 2 (CB2) receptors in immune tissues. This binding action modulates pain and spasticity.
Class: cannabinoid Treatments (approved or in clinical trial): MS induced spasticity, pain in advanced cancer,
glioblastoma multiforme (GBM)
2015 Oct: Topline data from two Phase 3 studies was released. The studies compared Sativex with placebo in combination with
opioids in patients with advanced cancer whose pain was not well controlled with opioids alone. The study’s primary endpoint
was reduction in pain measured on a 10 point scale. Both studies failed to meet their primary endpoint.
2015 Jan: Topline data from a Phase 3 study was released. The study compared Sativex with placebo in combination with
opioids in patients with advanced cancer whose pain was not well controlled with opioids alone. The study’s primary endpoint
was reduction in pain measured on a 10 point scale. The study failed to meet its primary endpoint.
2014 Apr: FDA FTD received for the treatment of pain in patients with advanced cancer, who do not respond adequately to
opioid therapy.
Helsinn
anamorelin
Product Description: Anamorelin is a small-molecule mimetic that stimulates appetite. Anamorelin binds and stimulates the
growth hormone secretagogue receptor; which mimics the appetite and growth hormone releasing effects of grhelin. It is currently
under investigation for treating cachexia in non-small cell lung cancer (NSCLC)
Class: GR agonist
Treatments (approved or in clinical trial): cachexia
2014 Nov: Helsinn released data from the Phase 3 ROMANA 1 and ROMANA 2 studies. The studies compared anamorelin
with placebo in patients with unresectable stage III/IV NSCLC and cachexia. The co-primary endpoints were lean body mass
(LBM) and handgrip strength. Anamorelin met the first primary endpoint of increased LBM.
PRODUCT DETAIL – GILEAD, GSK, GW PHARMA, HELSINN
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Helsinn
Akynzeo
Product Description: Netupitant+palonosetron (Akynzeo) is a combination of neupitant, a selective neurokinin 1 (NK1)
receptor antagonist and palonosetron, a selective serotonin (5-hydroxytryptamine) receptor subtype 3 (5-HT3) antagonist. This
combination provides anti-emetic activity. Palonosetron blocks 5-HT3 receptors on the vagal nerve in the chemoreceptor
trigger. Neupitant completely blocks the NK1 receptors in the central nervous system, which inhibits delayed emesis,
preventing chemotherapy-induced nausea and vomiting (CINV).
Class: antiemetic
Treatments (approved or in clinical trial): CINV
2015 Jun: EMA APP received for the treatment of CINV.
2014 Oct: FDA APP received for the treatment of CINV.
2014 Jan: EMA MAA for the treatment of CINV.
2013 Dec: FDA NDA for the treatment of CINV.
Helsinn
Aloxi
Product Description: Palonosetron (Aloxi) is a selective serotonin (5-hydroxytryptamine) receptor subtype 3 (5-HT3)
antagonist. This combination provides antiemetic activity. Palonosetron blocks 5-HT3 receptors on the vagal nerve in the
chemoreceptor trigger.
Class: antiemetic
Treatments (approved or in clinical trial): CINV
2015 May: EMA APP received for use in children aged ≥1 month.
2015 Jan: EMA CHMP positive opinion received for use in infants and children aged ≥1 month.
2014 May: FDA APP received for use in children aged ≥1 month.
INSYS
dronabinol
Product Description: Dronabinol is an orally administered liquid formulation of cannabinoid dronabinol, a synthetic version
of tetrahydrocannabinol. It has a faster absorption rate than merinol, maintains more consistent blood levels , and can by
titrated.
Class: cannabinoid Treatments (approved or in clinical trial): anorexia, weight loss in AIDS, chemotherapy
induced nausea and vomiting (CINV)
2016 Jul: FDA APP received for the treatment of CINV.
2015 Jun: FDA NDA resubmitted for the treatment of anorexia, weight loss in AIDS, and CINV (the average length of time
from FDA NDA to approval in the US for hematology products listed in PDD is 7 months).
2014 Oct: FDA CRL issued due to an incomplete pediatric study plan.
2014 Aug: FDA NDA for the treatment of anorexia, weight loss in AIDS, and CINV.
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Jazz
Defitelio
J&J Janssen
Darzalex
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Research and Regulatory Timeline Detail
Product Description: Defibrotide (Defitelio) is a deoxyribonucleic acid derived from cow lung or porcine mucosa. It is a mild
anticoagulant that prevents the formation of blood clots and inhibits platelet aggregation. It is used to treat severe veno-occlusive
disease (VOD) in patients undergoing hematopoietic stem cell transplantation. VOD is a condition that blocks blood vessels in the
liver, leading to liver dysfunction and failure.
Class: anticoagulant Treatments (approved or in clinical trial): VOD, multiple myeloma
2016 Mar: FDA APP received for the treatment of VOD in adults and children with renal or pulmonary dysfunction following
hematopoietic stem-cell transplantation.
2016 Feb: Blood published a Phase 3 study. The Phase 3 study compared defibrotide with historical controls in patients with VOD,
multiple organ failure post-hematopoietic stem-cell transplantation. The primary endpoint was Day +100 survival. The study met its
primary endpoint with a significant 23% improvement in Day 100+ survival.
2015 Sep: FDA PRD received for the treatment of VOD.
2014 Dec: FDA NDA submitted & FTD received for the treatment of severe VOD.
2013 Oct: EMA APP received for the treatment of severe VOD.
Product Description: Daratumumab (Darzalex) is a human monoclonal antibody which binds to the glycoprotein CD380. CD38 is
highly expressed on the surface of multiple myeloma (MM) cancer cells. By binding to the surface of the MM cells, daratumumab
enhances the immunological response to destroying cancer cells.
Class: CD38 agonist
Treatments (approved or in clinical trial): multiple myeloma (MM)
2016 Jul: FDA BTD for use in combination with standard of care regimens for multiple myeloma patients who have received at least
one prior line of therapy.
2016 Jul: Blood published pooled data from the GEN501 and SIRIUS studies. The pooled data showed no new safety issues,
achieved an overall response of 31%, and demonstrated a median overall survival of 20.1 months.
2016 Jun: The Phase 3 POLLUX study was presented at the European Hematology Association (EHA). The study compared
daratumumab plus lenalidomide and dexamethasone with lenalidomide and dexamethasone alone in patients with relapsed/refractory
MM. The primary endpoint was progression-free survival (PFS). The study achieved its endpoint with a reduction in PFS of 63%. The
median PFS in the daratumumab cohort has not yet been reached.
2016 Jun: The Phase 3 CASTOR study that compared daratumumab plus bortezomib and dexamethasone with bortezomib and
dexamethasone in patients with relapsed or refractory MM was presented at ASCO. The primary endpoint was progression free
survival (PFS). The study met its endpoint with a 61% reduction in risk of disease progression compared with bortezomib plus
dexamethasone.
2016 May: EMA APP received for use as 2nd line in the treatment of relapsed/refractory MM patients whose prior therapy included a
PI and an immunomodulatory agent.
2016 May: The Phase 3 POLLUX study was stopped early. The study was ended early because it met its primary endpoint.2016 Apr:
Lancet published the Phase 2 SIRIUS study. The study evaluated the efficacy of daratumumab in patients who had experienced at least
three lines of treatment and/or were refractory to proteasome inhibitors and immunomodulators. The primary endpoint was overall
response rate (ORR). The study met its primary endpoint achieving a 29.2% ORR.
2016 Apr: EMA CHMP positive opinion received for use in patients with relapsed or refractory MM and whose prior therapies
included a PI and an immunomodulatory agent and whose disease has progressed (the average length of time from EMA CHMP to
approval in the EU for hematology products listed in PDD is 3 months).
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2016 Mar: An interim analysis of the Phase 3 CASTOR study was released. The primary endpoint was PFS. The study met its
endpoint and the study was stopped early.
2015 Aug: The Phase 1/2 GEN501 study was published in NEJM. Daratumumab demonstrated a tolerable safety profile and a
36% overall response rate in patients with MM who had relapsed or where the disease was refractory to at least two lines or
more prior lines of therapy including proteasome inhibitors, immunomodulatory agents, chemotherapy, and stem cell
transplantation.
2015 Nov: FDA APP received for the treatment of MM in patients who have received at least three prior lines of therapy,
including a proteasome inhibitor and an immunomodulatory agent, or who are double-refractory to both agents.
2015 Sep: EMA MAA (accelerated assessment) for use in heavily pre-treated MM patients.
2015 Sep: FDA PRD status granted for MM in patients who are refractory to both proteasome inhibitors and
immunomodulatory agents and who have received at least three or more prior lines of therapy.
2015 Jun: FDA BLA for the treatment of MM in patients who have received at least three prior lines of therapy.
2015 May: The Phase 2 MMY2002 study was presented at ASCO. Daratumumab achieved an overall response rate of 29.2%.
J&J
Janssen
imtelestat
Product Description: Imtelestat (GRN163L) is a telomerase inhibitor active against both CD138+ and CD138- cancer stem
cells. In normal cells, absent telomerase, cells become senescent after 50-70 cell divisions and cell division stops. Telomerase
replaces short bits of DNA allowing cell division to continue and the cells become “immortal.“ Many cancer cells are
considered immortal because telomerase allows the cells to divide indefinitely. By stopping this process the cancer can be
slowed or stopped.
Class: telomerase inhibitor Treatments (approved or in clinical trial): myelofibrosis (MF), myelodysplastic syndromes
(MDS), acute myelogenous leukemia (AML), essential thrombocythemia (ET)
2015 Sep: NEJM published two Phase 2 studies. The 1st study evaluated the efficacy and safety of imtelestat in patients with
high-risk or intermediate-2-risk MF. The primary endpoint was overall response rate. There was a 27% response among
patients with a JAK2 mutation and 0% in patients without the mutation, however there was a potential for significant
myelosuppression. The 2nd study evaluated the safety and efficacy of imtelestat in patients with ET. The primary endpoint was
best hematologic response. The study met its endpoint with 89% of patients achieving complete response.
2014 Nov: FDA lifts clinical trial hold.
2014 Jun: FDA lifts partial hold on MF trials.
2014 Mar: The FDA imposed a full clinical hold on all ET and partial hold on MF clinical trials.
J&J
Janssen
Sylvant
Product Description: Siltuximab (Sylvant) is an anti-interleukin-6 (IL-6) chimeric monoclonal antibody. A monoclonal
antibody is a type of protein designed to recognize and attach to a specific antigen. Siltuximab attaches and blocks IL-6 and
thus stops abnormal cell growth.
Class: IL-6 inhibitor Treatment (approved or in clinical trial): multicentric Castleman’s disease (MCD), metastatic
renal cell cancer (mRCC), prostate cancer (PC)
2014 Jun: EMA APP received for the treatment of MCD patients who are HIV or HHV-8 negative
2014 Apr: FDA APP received for the treatment of MCD patients who are HIV or HHV-8 negative.
2014 Mar: EMA CHMP positive opinion received for the treatment of MCD patients who are HIV or HHV-8 negative.
2013 Sep: EMA MAA and FDA BLA for the treatment of MCD who are HIV and human herpes virus-8 (HHV-8) negative.
PRODUCT DETAIL – J&J& JANSSEN
J&J
Janssen
Darzalex
Research and Regulatory Timeline Detail
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MSD
Emend
Product Description: Fosaprepitant (Emend) is a P/neurokinin-1 (NK1) receptor antagonist approved for use in combination
with other antiemetics. It is approved for use in both highly and moderately emetogenic cancer chemotherapy.
Class: NK1 antiemetic
Treatments (approved or in clinical trial): Adult high and moderate emetogenic cancer
chemotherapy, pediatric high and moderate emetogenic cancer chemotherapy
2016 Feb: FDA APP received for single-dose injection in combination with other anti-emetics for the prevention of delayed
nausea after initial and repeat courses of moderately emetogenic chemotherapy (MEC).
2015 Sep: FDA APP received for pediatric use in patients aged ≥12 years.
2015 Jun: A Phase 3 study was presented at MASCC. The study compared fosaprepitant plus an antiemetic with placebo plus
an antiemetic in adult cancer patients receiving moderate emetogenic chemotherapy. Fosaprepitant provided greater protection
from nausea and vomiting following chemotherapy compared with placebo.
2014 Jun: A Phase 3 study was presented at MASCC. The study evaluated the efficacy of aprepitant for the prevention of
chemotherapy-induced nausea and vomiting in pediatric cancer patients aged 6 month to 17 years. The study demonstrated that
aprepitant+ondansetron achieved significant effectiveness compared with placebo+ondansetron in pediatric patients
undergoing very high, high, and moderate emetogenic chemotherapy.
MSD
Keytruda
Product Description: Pembrolizumab MK-3475 (Keytruda) is a monoclonal antibody that blocks the inhibitory ligand of
programmed cell death 1 receptor (PD-1). Certain tumors utilize the PD-1 pathway to prevent the activation of T-cells thus
blocking the immune system’s natural response to cancer cells. Pembrolizumab blocks PD-1 allowing the immune system to
recognize certain cancer cells and destroy them.
Class: PD-1
Treatments (approved or in clinical trial): melanoma, colorectal cancer (CRC), non-small cell lung
cancer (NSCLC), Hodgkins lymphoma (HL), head and neck cancer (HNC), gastric cancer (GC), mesothelioma, urothelial
(bladder) cancer (UC), nasopharyngeal carcinoma (NPC), multiple myeloma (MM)
2016 Jun: The Phase 2 KEYNOTE-087 study was presented at ASCO. The study evaluated the efficacy of pembrolizumab as
monotherapy in patients with relapsed/refractory HL across three cohorts. The primary endpoints were overall safety,
tolerability and overall response rate (ORR). Cohort 1 was comprised of patients whose disease progressed after stem cell
transplantation and treatment with brentuximab vedotin, Cohort 2 contained patients who failed salvage therapy and were
ineligible for transplantation and whole failed brentuximab, and Cohort 3 consisted of transplant patients who did not receive
brentuximab. ORR was 73% in Cohort 1, 83% in Cohort 2, and 73% in Cohort 3.
2016 Apr: FDA BTD received for the treatment of relapsed/refractory classical HL.
2015 Dec: The Phase 1 KEYNOTE-023 study was presented at ASH. The study evaluated the efficacy of pembrolizumab plus
lenalidomide and low-dose dexamethasone in MM patients whose disease had progressed after at least two lines of prior
therapy. The study showed that pembrolizumab achieved an overall response rate of 76%.
2014 Dec: The Phase 1b KEYNOTE-013 study was presented at ASH. The study showed a 66% overall response rate and a
complete remission of 21% in patients in transplant-ineligible and failure patients with relapsed/refractory classical Hl whose
disease progressed on or after treatment with brentuximab.
PRODUCT DETAIL – MSD
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Novartis
CTL019
Product Description: CTL019 is a chimeric antigen receptor (CAR). CARs are engineered to graft monoclonal antibodies
onto T cells. T cells are withdrawn from the patient’s blood and the cells are programmed to hunt cancer cells and express the
protein CD19. The cells are then reintroduced into the patient’s body where they bind to the targeted cells and destroy them.
The protein CD19 is associated with a number of B-cell cancers including acute lymphoblastic leukemia (ALL), chronic
lymphoblastic leukemia (CLL), diffuse large B-cell lymphoma (DLBCL), non-Hodgkin's follicular lymphoma (NHFL), and
mantle cell lymphoma (MCL).
Class: CAR
Treatments (approved or in clinical trial): ALL, CLL, DLBCL, FL, MCL
2015 Dec: A Phase 2a study was presented at ASH. The study evaluated the efficacy of CTL019 in patients with
relapsed/refractory DLBCL and NHFL. CTL019 achieved an overall response rate of 47% at three months in DLBCL and 73%
in NHFL.
2015 Jun: A Phase 2 study was presented at ASCO. The study evaluated the efficacy of CTL019 in patients with
refractory/relapsed DLBCL and refractory/relapsed FL. The overall response rate was 50% in DLBCL patients and 100% in
FL patients.
2014 Dec: Results from two Phase 2 studies presented at ASH. The studies demonstrated 92% complete remission in pediatric
patients with relapsed/refractory ALL.
2014 Oct: Two Phase 2 studies were published in NEJM. The studies showed 90% of relapsed/refractory ALL patients
achieved complete remission. Two year sustained remission with 6 month event-free survival was 67% and overall survival
was 78%.
2014 Jul: FDA BTD status granted for pediatric and adult relapsed/refractory ALL.
2013 Dec: Two Phase 2 studies were presented at ASH. The first study evaluated the efficacy of CTL019 in patients with ALL
and CLL. In the ALL study 86% of pediatric patients and 100% of adult patients experienced complete remission. In the CLL
study 47% of adults with CLL responded to therapy with CTL019.
Novartis
Jadenu
Product Description: Deferasirox (Jadenu) is a new formulation of Exjade/Desirox. Deferasirox is an iron chelator. Chelating
agents remove heavy metals from the body. Patients who undergo long-term blood transfusions tend to develop a chronic iron
overload in the body. Deferasirox binds to iron atoms both in blood and in organ cells. The deferasirox/iron is subsequently
eliminated via fecal excretion.
Class: chelating agent
Treatments (approved or in clinical trial): chronic iron overload (CIO)
2015 Mar: FDA APP received for chronic iron overload due to blood transfusions in patients ≥2 years, and chronic iron
overload in non-transfusion-dependent thalassemia syndromes (NTDT) in patients ≥10 years.
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Novartis
Revolade
Promacta
Product Description: Eltrombopag (Revolade/Promacta) is a thrombopoietin receptor (TPOR). Thrombopoietins stimulate the
production of platelets by attaching to receptors in bone marrow. Eltrombopag stimulates these same receptors thus inducing
the production of platelets, thereby increasing platelet counts in the blood stream.
Class: TPOR
Treatments (approved or in clinical trial): acquired severe aplastic anemia (ASAA), immune
thrombocytopenic purpura (ITP), thrombocytopenia in Hepatitis C
2016 Jun: The EXTEND study was presented at the the European Hematology Association Congress (EHA). EXTEND is an
open-label extension study of four clinical trials. The objectives of the study were to assess the safety and efficacy of long-term
treatment with eltrombopag. The median duration of exposure was 2.4 years and the mean average daily dose was 50.3
mg/day. 25% of the study subjects were treated for four or more years. Most patients were aged <65 years, female, and had
platelet counts >15 x 109/L at baseline. Grade 3 and 4 adverse events occurred 26% and 6%, respectively. Response rates to
eltrombopag (patients achieving ≥50 x 109/L without rescue therapy) were higher in the non-splenectomized patients. Adverse
events (AEs) occurring in ≥4% of patients in the splenectomized and non-splenectomized groups, respectively, The most
common AEs were mouth hemorrhage (4% and 1%), epistaxis (14% and 6%), petechiae (8% and 2%), ecchymosis (2% and
4%), contusion (3% and 4%), and hematuria (2% and 4%). 13% of patients in each group were able to reduce/discontinue
medication.
2016 Apr: EMA APP received for the treatment of pediatric ( ≥1 year) ITP in patients who are refractory to other therapies.
2015 Sep: EMA APP received for use in adults with ASAA who where either refractory to prior immunosuppressive therapy
or heavily pretreated and unsuitable for hematopoietic stem cell transplant.
2015 Aug: FDA expanded indication to include children 1 year and older.
2015 Jul: EMA CHMP positive opinion received for patients with acquired severe aplastic anemia who have insufficient
response to immunosuppressive therapy.
2015 Jun: FDA APP received for the treatment of ITP in children aged ≥6 yeas with chronic ITP who have had insufficient
response to corticosteroids, immunoglobulins or splenectomy
2015 Feb: EMA sMAA for pediatric (age ≥1 year) with chronic ITP who have had an insufficient response to corticosteroids
or immunoglobulins.
2014 Dec: FDA sNDA for pediatric patients aged ≥6 years with chronic ITP who have had an insufficient response to
corticosteroids, immunoglobulins or splenectomy.
2014 Nov: EMA sMAA for additional indication of ASAA who have insufficient response to immunosuppressive therapy.
2014 Aug: FDA APP received for use in ASAA patients who have not adequately responded to immunosuppressive therapy.
2014 Jun: The Phase 3 PETIT2 study was presented at EHA. The study compared eltrombopag with placebo in pediatric
patients with chronic ITP. The study met its primary endpoint of significant improvement in platelet counts for eltrombopag.
2014 Feb: FDA sNDA for cytopenias in patients with ASAA who have insufficient response to immunotherapy.
2014 Feb: FDA BTD for cytopenias in patients with ASAA who have had insufficient response to immunosuppressive
therapy.
2013 Sep: EMA APP received for the treatment of thrombocytopenia in adult patients with Hep C.
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Novartis
midostaurin
Product Description: Midostaurin PKC412 is a multi-kinase inhibitor (MKI). It is known to inhibit tyrosine kinases; kinase C
alpha (PKCα), vascular endothelial growth factor receptor 2 (VEGFR2), platelet-derived growth factor (PDGFR), and FMSlike tyrosine kinase 3 (FLT3). By inhibiting these tyrosine kinases midostaurin disrupts the cell cycle, inhibits proliferation of
cancer cells, induces apoptosis, and inhibits angiogenesis.
Class: MKI
Treatments (approved or in clinical trial): acute myeloid leukemia (AML), mast cell leukemia (MCL),
myelodysplastic syndrome (MDS), systemic mastocytosis (SM)
2016 Jun: FDA BTD received for the treatment of newly diagnosed FLT3-mutated ALM.
2016 Jun: NEJM published a Phase 2 prospective study that evaluated the safety and efficacy of midostaurin in patients with
advanced SM. The primary endpoint was overall survival (OS). Patients continued to receive midostaurin until disease
progression or unacceptable toxicity. Median OS was 28.7 months.
2015 Dec: The Phase 3 RATIFY study was presented at ASH. The study compared midostaurin plus standard induction and
consolidation chemotherapy with standard induction and consolidation chemotherapy alone in patients <60 years with newlydiagnosed FLT3-mutated AML. The study met its primary endpoint of OS with midostaurin improving OS by 23% compared
with placebo.
Novartis
Tasigna
Product Description: Nilotinib (Tasigna) is a small-molecule tyrosine kinase inhibitor (TKI). Nilotinib selectively inhibits the
enzyme Bcr-Abl kinase which is prevalent in Philadelphia-chromosome-positive (PH+) leukemia which causes them to
replicate uncontrollably.
Class: TKI
Treatments (approved or in clinical trial): PH+ chronic myeloid leukemia (CML), BCR-ABL+
CML, Parkinson’s, Alzheimer’s. Huntingdon’s
2016 Jun: The Phase 2 ENESTfreedom and ENESTop studies were presented at ASCO. Both studies were open label studies
designed to evaluate the effect of stopping nilotinib treatment in chronic PH+ CML patients who had achieved a molecular
response (MR) of 4.5 and a deep MR for one year with 1st line nilotinib (freedom) or who had achieved deep MR for one year
with nilotinib following treatment with imatinib (stop). The primary endpoint in both studies was the percentage of patients in
major molecular response (MMR; BCR-ABL1 International Scale ≤ 0.1%) at 48 weeks in the treatment-free remission (TFR)
phase. The median duration of the trial was 3.6 years. The freedom study did not reach its primary endpoint of achieving
MMR at 48 weeks in the TFR stage, with 51.6% (not statistically significant) achieving MMR in the TFR phase. Stop did meet
its endpoint with 57.9% achieving MMR at 48 weeks in the TFR phase.
2015 Jun: The Phase 3b ENEST1st study was presented at EHA. ENEST1st evaluated the efficacy of nilotinib as 1st line
therapy in patients with newly-diagnosed BCR-ABL+ CML. The study showed that nilotinib achieved rapid and high rates of
molecular response and patients had a very low rate of progression to advanced disease.
2014 Dec: Six-year follow up data from the Phase 3 ENEST study was presented at ASH. ENEST compared nilotinib with
imatinib in newly diagnosed chronic phase PH+ CML patients. The nilotinib cohort had significantly fewer patients progress to
advanced stage disease and a deeper sustained molecular response compared with the imatinib cohort.
2013 Dec: Three Phase 3 studies ENESTnd, ENESTcmr, and LASOR were presented at ASH. All three studies compared
nilotinib with imatinib in PH+ CML patients. The studies demonstrated significant superiority to imatinib by achieving deeper
molecular response across a variety of PH+ CML patient groups including newly diagnosed patients, patients with residual
disease who switched from imatinib, and patients who failed to respond to 1st line imatinib.
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Product Description: Ofatumumab (Arzerra) is a human monoclonal antibody designed to recognize and attach to the CD20
protein which is found on early-stage B lymphocytes. When ofatumumab attaches to CD20 it stimulates the body’s immune system
to attack the cancer cells expressing the protein thus helping to control the spread of the cancer.
Class CD20 antibody
Treatments (approved or in clinical trial): chronic lymphocytic leukemia (CLL), follicular nonHodgkin’s lymphoma (FNHL), diffuse large B-cell lymphoma (DLBCL), rheumatoid arthritis, multiple sclerosis
2016 Jun: EMA CHMP negative opinion received for use as maintenance treatment in patients with relapsed CLL.
2016 May: FDA PRD received for the relapsed CLL sBLA.
2016 Mar: FDA sBLA for the treatment of relapsed CLL in combination with fludarabine and cyclophosphamide (the average
length of time from FDA submission to approval in the US for hematology products listed in PDD is 7 months).
2016 Mar: EMA sMAA for the treatment of relapsed CLL in combination with fludarabine and cyclophosphamide (the average
length of time from EMA submission to approval in the EU for hematology products listed in PDD is 15 months).
2016 Jan: FDA APP received for the sBLA for extended treatment of patients who experience complete or partial response after at
least two lines of therapy for recurrent or progressive CLL.
2015 Jun: The Phase 3 COMPLEMENT 2 study was presented at EHA. COMPLEMENT 2 compared ofatumumab plus
fludarabine and cyclophosphamide with fludarabine plus cyclophosphamide in patients with relapsed CLL. The ofatumumab cohort
significantly improved progression-free survival (PFS) by 54% compared with the fludarabine plus cyclophosphamide cohort.
2015 Apr: Topline data from the Phase 3 COMPLEMENT 2 study was released. The ofatumumab cohort met its primary
endpoint of significant improvement of PFS.
2015 Apr: The Phase 3 COMPLEMENT 1 study was published in Lancet. COMPLIMENT 1 compared ofatumumab plus
chlorambucil with chlorambucil alone in treatment naïve CLL patients for whom fludarabine-based therapy was therapeutically
inappropriate. The study showed a significant improvement for the primary endpoint of PFS.
2014 Jul: Topline data from the Phase 3 PROLONG study was released. The study compared ofatumumab maintenance therapy
with no further treatment in patients with relapsed CLL who responded to therapy at relapse. The study showed that maintenance
ofatumumab met its primary endpoint of progression-free survival (PFS).
2014 Jul: EMA APP received for use 1st line therapy of CLL in combination with chlorambucil or bendamustine for patients
ineligible for fludarabine-based therapy.
2014 Jul: (see Abbvie: ibrutinib) The RESONATE study was published in NEJM.
2014 Jun: Topline data from a Phase 3 study was released. The study compared ofatumumab with “physicians choice” in patients
with bulky fludarabine-refractory CLL. The study did not meet its primary endpoint of PFS.
2014 May: EMA CHMP positive opinion received for use as 1st line therapy in combination with chlorambucil in patients for
whom fludarabine-based therapy is considered inappropriate.
2014 May: FDA APP received for the treatment of CLL as 1st line therapy in combination with chlorambucil in patients for whom
fludarabine-based therapy is considered inappropriate.
2014 May: The Phase 3 ORCHARD study was discontinued due to the fact that it did not meet its primary endpoint of PFS.
ORCHARD compared ofatumumab plus chemotherapy with rituximab plus chemotherapy in patients with relapsed DLBCL.
2014 May: (see AbbVie: ibrutinib) RESONATE study published in NEJM.
PRODUCT DETAIL – NOVARTIS
Novartis
Arzerra
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2014 Apr: FDA APP for the treatment of CLL in combination with chlorambucil in previously untreated patients for whom
fludarabine-based therapy is considered inappropriate.
2013 Dec: FDA PRD received for CLL in combination with an alkylator-based as 1st line therapy in patients who are
inappropriate for fludarabine-based therapy.
2013 Oct: EMA MAA and FDA NDA for CLL in combination with an alkylator-based as 1st line therapy in patients who are
inappropriate for fludarabine-based therapy.
2013 Sep: FDA BTD received for previously untreated CLL.
PRODUCT DETAIL – NOVARTIS
Novartis
Arzerra
Research and Regulatory Timeline Detail
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Novartis
Farydak
Product Description: Panobinostat – LBH589 (Farydak) is a hydroxamic acid which acts as a non-selective histone deacetylase
(HDAC) inhibitor. HDACs work by blocking key enzymes that act as on/off switches within cancer cells. The drug acts in
several different ways. It turns “on” genes that suppress the division of cancer cell, thus slowing down tumor growth and it
modulates the expression of angiogenesis-related genes thus impairing endothelial cell growth.
Class: HDAC
Treatments (approved or in clinical trial): multiple myeloma (MM), Hodgkin’s Lymphoma (HL),
cutaneous T cell lymphoma (CTCL), myelodysplastic syndromes (MDS), breast cancer (BC), prostate cancer (PC), chronic
myelomonocytic leukemia (CMML), HIV infection, diffuse large B-Cell lymphoma (DLBLC)
2016 Jul: Blood published a Phase 2 study. The study compared panobinostat plus rituximab with rituximab alone in patients
with relapsed/refractory (de novo and transformed) DLBLC. The study met its primary endpoint of response rate, with 28% of
the combination cohort responding while none of the rituximab cohort responded. Responses can be predicted by mutations in
MEF2B or significant change in ctDNA levels.
2015 Sep: EMA APP received for use in combination with bortezomib and dexamethasone in adult patients with
relapsed/refractory MM who have received at least two prior regimens including bortezomib and an immunomodulatory agent.
2015 Jun: EMA CHMP positive opinion received for use in combination with bortezomib and dexamethasone, in adult patients
with relapsed/refractory MM who have received at least two prior regimens including bortezomib and an immunomodulatory
agent.
2015 Jun: The Phase 3 PANORAMA-1 study was presented at EHA. The study compared panobinostat in combination with
bortezomib and dexamethasone with bortezomib and dexamethasone alone in patients with relapsed/refractory MM. The study
demonstrated a significant improvement in progression-free survival (PFS) of 7.8 months.
2015 Feb: FDA APP received for use in combination with bortezomib and dexamethasone in patients with MM who have
received at least two prior regimens including bortezomib and immunomodulatory therapy.
2014 Nov: FDA extended the priority review period for MM.
2014 Nov: FDA Oncology Advisory committee rejected recommendation for MM.
2014 Sep: The Phase 3 PANORAMA-1 study was published in Lancet Oncology. The panobinostat cohort achieved a
significant improvement in PFS compared with the control group.
2014 Jun: The Phase 3 PANORAMA-1 study was presented at ASCO. The panobinostat cohort met its primary endpoint
improving PFS by 37%.
2013 Dec: Topline data from the Phase 3 PANORAMA-1 study was released. The study met its primary endpoint of PFS.
Novartis
pegfilgrastim
biosimilar
Product Description: Pegfilgrastim biosimilar is a biosimilar version of Amgen Neulasta. Pegfilgrastim is a pegylated form of
granulocyte colony-stimulating factor (GCSF) analog filgrastim. Pegfilgrastim stimulates bone marrow to produce more white
blood cells.
Class: GCSF Treatments (approved or in clinical trial): chemotherapy induced neutropenia
2016 Feb: EMA MAA for chemotherapy induced neutropenia (the average length of time from submission to approval in the EU
for hematology products listed in PDD is 16 months).
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Product Description: Ruxolitinib (Jakavi/Jakafi) is a Janus Kinase inhibitor (JAK). The JAK enzymes are involved in the
production of blood cells. In the conditions of myelofibrosis and polycythaemia vera (PCV) there is too much JAK activity in the
bone marrow and subsequent abnormal production of blood cells which migrate to organs, including the spleen, leading to
enlargement of these organs. Ruxolitinib blocks JAK production and thus reduces symptoms caused by JAK induced disorders.
Class: JAK
Treatments (approved or in clinical trial): myelofibrosis (MF), polycythemia vera PCV, plaque psoriasis,
alopecia areata, pancreatic cancer (PAN), graft-versus-host disease (GVHD)
2016 Jun: FDA BTD received for the treatment of acute GVHD.
2016 Jun: The Phase 3 RESPONSE-2 study was presented at the European Society of Hematology (EHA). RESPONSE-2
compared ruxolitinib with best available therapy (BAT) in patients with PCV who were resistant to or intolerant of hydroxyurea
(HU), dependent on phlebotomy for hematocrit control and who do not have an enlarged spleen. Ruxolitinib was superior to BAT
in maintaining hematocrit control (62.2% versus 18.7%, respectively) without need for phlebotomy.
2016 Jun: Five-year follow-up data from the Phase 3 COMFORT-I study was presented at ASCO. The COMFORT studies
evaluated the efficacy and safety of ruxolitinib compared with placebo (best available therapy) in patients with intermediate or
higher risk primary MF, post-PCV MF, or post-essential thrombocythemia myelofibrosis. The 5 year follow-up data showed a
31% reduction in risk of death (compared with placebo) in patients with intermediate-2 or high risk MF.
2015 Dec: Topline data from the Phase 3 RESPONSE-2 study was released. The study compared ruxolitinib with best available
therapy in patients with inadequately controlled PCV resistant to or intolerant of hydroxyurea who did not have a large spleen.
The study met its primary endpoint.
2015 Dec: Five-year follow-up data from the Phase 3 COMFORT-II study was presented at ASH. Five-year data demonstrated
continued survival benefit in patients taking ruxolitinib.
2015 Jun: The Phase 3 RESPONSE study was presented at EHA. RESPONSE compared ruxolitinib with investigator-selected
standard therapy in PCV patients resistant to or intolerant of hydroxyurea. The study demonstrated that 80% of patients with
inadequately controlled PCV treated with ruxolitinib experienced a durable response of sustaining hemacrit >45% without the use
of phlebotomy and reducing spleen size for at least 1 year.
2015 Mar: EMA APP received for the treatment of PCV in patients resistant to or intolerant of hydroxyurea.
2015 Jan: The Phase 3 RESPONSE study was published in NEJM. The ruxolitinib cohort met its primary endpoint of durable
hemacrit control compared with standard therapy.
2015 Jan: EMA CHMP positive opinion received for the treatment of PCV in patients who are resistant or intolerant of
hydroxyurea.
2014 Dec: FDA APP received for the treatment of PCV.
2014 Dec: Results from the Phase 3b JUMP study were presented at ASH. JUMP compared various doses of ruxolitinib in
patients with myelofibrosis. Patients taking achieved significant reduction in spleen size that was maintained over time.
2014 Jun: The Phase 3 RESPONSE study was presented at ASCO. The study compared the efficacy of ruxolitinib BAT in
patients with PCV resistant to or intolerant of hydroxyurea. The study met its primary endpoint with 77% of the ruxolitinib cohort
achieving hemacrit control versus 20% for BAT.
2014 Mar: Topline data from the Phase 3 RESPONSE study was released. The study met its primary endpoint of maintaining
hemacrit control without need of phlebotomy and reducing spleen size compared with best available therapy.
PRODUCT DETAIL – NOVARTIS
Novartis
Jakavi
Jakafi
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Novartis
Jakavi
Jakafi
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2013 Dec: Results from the Phase 3 COMFORT-I and COMFORT-II studies were presented at ASH. The COMFORT
studies evaluated the efficacy and safety of ruxolitinib compared with placebo (best available therapy; BAT) in patients with
intermediate or higher risk primary myelofibrosis, post-PCV myelofibrosis, or post-essential thrombocythemia myelofibrosis.
The studies showed that myelofibrosis patients taking ruxolitinib lived significantly longer than those on placebo BAT.
Oxigene
OXi4503
Product Description: OXi4503 (combretastatin A1 diphosphate / CA1P) is a dual-mechanism vascular disrupting agent
(VDA). OXi4503 blocks and destroys tumor vasculature, resulting in tumor cell death and necrosis. In addition, OXi4503 is
metabolized by oxidative enzymes, which are elevated in many solid tumors and tumor white blood cell infiltrates, to an
orthoquinone chemical species that has direct cytotoxic effects on tumor cells.
Class: VDA Treatments (approved or in clinical trial): acute myeloid leukemia (AML)
2015 Dec: EMA COMP received for the treatment of AML.
Pfizer
BeneFIX
Product Description: BeneFIX is a recombinant coagulation Factor IX product used for the control, prevention, and
perioperative management of bleeding in adults and children with hemophilia B (hem B).
Class: rFIX
Treatments (approved or in clinical trial): hem B control, prevention, and perioperative management
2016 Mar: A Phase 3 study was published in Haemophilia. The study was a non-randomized, open-label, sequential-period
trial with a six-month period on-demand treatment followed by a 12 month prophylaxis period in patients with moderate-tosevere hem B. The study showed that a once-weekly prophylactic regimen provided a significant reduction in bleeding
compared with on-demand administration.
2014 Jul: Topline data from a Phase 3 study was released. The study compared a prophylaxis regimen of BeneFIX with an ondemand regimen in patients with moderately-severe or severe hem B. The study met its primary endpoint, showing a
significant reduction in annualized bleeding rate for the prophylaxis cohort.
Pfizer
Bosulif
Product Description: Bosutinib (Bosulif) is a tyrosine-kinase inhibitor which acts by blocking the enzymes Src and Bcr-Abl.
These enzymes are found in some receptors on the surface of leukemia cells. They aid in the spread of disease by stimulating
leukemia cells to divide uncontrollably. Bosutinib works by blocking the activity of these enzymes thus controlling leukemia
cell division.
Class: TKI
Treatments (approved or in clinical trial): Philadelphia chromosome-positive (PH+) chronic
myelogenous leukemia (CML)
2014 Dec: Long-term follow-up data was presented at ASH. The data showed durable clinical benefit at five years in patients
with CML.
2014 Mar: Approved in Canada for the treatment of PH+ CML.
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Pfizer
Mylotarg
Product Description: Gemtuzumab+ozogamicin (Mylotarg) is a monoclonal antibody-drug conjugate (ADC) targeting CD33.
The antibody is linked to a cytotoxic agent (ozogamicin). The antibody binds to and is internalized in tumor cells expressing
CD33, which is commonly found on the surface of leukemic blasts. Once the compound is internalized, the cytotoxic agent
binds to DNA, causing DNA breaks thus leading to cell death.
Class: ADC
Treatments (approved or in clinical trial): acute myelogenous leukemia (AML)
2014 Dec: Two Phase 3 studies AML-19 and ALFA-0701 were presented at ASH. AML-19 compared Mylotarg with best
supportive care in elderly patients with AML who were not considered fit for intensive chemotherapy. Mylotarg met its
primary endpoint of overall survival. ALFA-0701 compared Mylotarg with daunorubicin plus cytarabine in adult AML
patients. Mylotarg met its primary endpoints of significant improvement in event-free survival and relapse-free survival.
Pfizer
inotuzumab
ozogamicin
Product Description: Inotuzumab+ozogamicin is a monoclonal antibody-drug conjugate (ADC) targeting CD22 which is
expressed on the surface of approximately 90% of B-cell malignancies. The antibody is linked to a cytotoxic agent
(ozogamicin). Inotuzumab binds to the CD22 antigen on malignant B-cells. It is then internalized into the cell where it delivers
the cytotoxic agent which is released and kills the cell.
Class: ADC
Treatments (approved or in clinical trial): acute lymphoblastic leukemia (ALL), non-Hodgkin’s
lymphoma (NHL)
2016 Jun: The Phase 3 INO-VATE ALL study was published in NEJM and presented at the European Hematology
Association Congress (EHA). The study compared inotuzumab ozogamicin with investigator-choice chemotherapy in adult
patient with relapsed/refractory CD-22+ ALL. The two primary endpoints were complete response (CR) with or without
hematologic remission and overall survival (OS). The study met its first endpoint with a significant improvement in CR (80.7%
for inotuzumab ozogamicin versus 29.4% for chemotherapy). However, the study did not meet the 2nd primary endpoint of
improved OS. Though the study did show improved OS it was not statistically significant (7.7 months versus 6.7 months,
respectively).
2015 Oct: FDA BTD received for ALL.
2015 Apr: Topline results for the Phase 3 INO-VATE ALL study were released. The study compared inotuzumab
ozogamicin with standard care of therapy in patients with relapsed/refractory CD22-positive ALL. The study met its primary
endpoint of higher complete hematologic remission than standard therapy.
2014 Dec: Data from a Phase 2 study was presented at ASH. The study evaluated the efficacy and safety of inotuzumab
ozogamicin in patients with refractory/relapsed ALL. 69% of the subjects achieved complete response or CR without complete
neutrophil count or platelet recovery.
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Pfizer/Spark
Therapeutics
SPK-9001
Product Description: SPK-9001is a novel bio-engineered adeno-associated virus (AAV) capsid expressing a codonoptimized, high-activity human factor IX variant. It is a gene therapy designed to be administered only once that induces
production of factor IX which is lacking in patients with hemophilia B.
Class: factor IX variant
Treatments (approved or in clinical trial): hemophilia B (hem B)
2016 Jul: Updated Phase 1/2 data released show that the low dose cohort of four subjects enrolled in the study continued to
experienced consistent and sustained factor IX activity and no sustained elevation in liver enzyme levels were observed.
2016 Jul: FDA BTD received for the treatment of hem B.
2016 Jun: Data from an open-label Phase 1/2 dose-escalation study was presented at EHA. The primary endpoint was the
number of participants experiencing drug-related adverse events and the secondary endpoint was changes from baseline in
circulating FIX activity. Across the cohort, no sustained elevation in liver enzyme levels and no drop in factor IX levels were
observed. The drug was well-tolerated and no subjects required, or received, immunosuppression. Additionally, initial
participants experienced consistent and sustained factor IX activity levels following a single administration of SPK-9001 at the
initial dose level (5 x 1011 vg/kg) studied in the trial.
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Prothena
NEOD001
Product Description: NEOD001 is a monoclonal antibody that specifically targets the circulating soluble amyloid and
deposited insoluble amyloid that accumulates in both AL and AA forms of amyloidosis.
Class: antibody Treatments (approved or in clinical trial): transthyretin-mediated (ATTR) amyloidosis, systemic AL
amyloidosis
2016 Jul: Data from the cardiac, renal, and peripheral neuropathy cohorts from a Phase 1/2 dose-escalation/expansion study
(published in JCO in Feb 2016) was presented at the International Symposium on Amyloidosis (ISA). The study showed
response rates of 53% and 63% in the cardiac and renal cohort, respectively. The study demonstrated a 35% improvement in in
the mean NIS-LL score in peripheral neuropathy cohort.
2016 Feb: Journal of Clinical Oncology (JCO) published interim data from a Phase 1/2 study. The study evaluated the efficacy
and safety of NEOD001 in patients with AL amyloidosis and persistent organ dysfunction. The results showed that NEOD001
was well tolerated and demonstrated improvements in functional biomarkers predictive of improvement of disease.
2015 May: Phase 1/2 dose escalation data showing that NEOD001 was safe and well tolerated at a dosage of 24 mg/kg per
month was presented at ASCO and European Hematology Association EHA.
Rigel
fostamatinib
Product Description: Fostamatinib is a spleen tyrosine kinase (SYK) inhibitor. Fostamatinib targets the underlying
autoimmune cause of immune thrombocytopenic purpura (ITP) and autoimmune hemolytic anemia (AIHA), rather than
stimulating platelet production (standard form of treatment). IgG type mediates platelet destruction in ITP. Fostamatinib blocks
IgG receptor signaling in both macrophages and B cells vie SYK, thus mediating the effect of the disease and normalizing
platelet function.
Class: SYK inhibitor Treatment (approved or in clinical trial): ITP, AIHA
2015 Sep: FDA ODD received for the treatment of chronic ITP.
Roche
ACE 910
emicizumab
Product Description: Emicizumab (ACE 910) is a bispecific monoclonal antibody designed to bind factors IXa and X, thus
mimicking the function of factor VIII. ACE910 is designed to promote blood coagulation in hemophilia A (hem A) patients. It
acts regardless of whether the patient has developed inhibitors to factor VIII, and is not expected to lead to the formation of
inhibitors.
Class: factor VIII
Treatments (approved or in clinical trial): hem A
2016 May: NEJM published a Phase 1 study that evaluated the efficacy and safety of emicizumab as prophylaxis in Japanese
hem A patients with or without FVIII inhibitors. The study demonstrated a clinically acceptable safety profile and potential for
prevention of bleeding in patients with and without FVIII inhibitors.
2015 Sep: FDA BTD received for prophylactic treatment in hem A patients ≥12 years who have developed factor VIII
inhibitors.
2015 Jun: The Phase 1/2 ACE002JP study was presented at ISTH. The study evaluated the prophylactic efficacy and safety of
ACE 910 in Japanese hem A patients both with and without FVIII inhibitors. ACE 910 demonstrated prophylactic efficacy
regardless of the presence of FVIII inhibitors.
2014 Dec: A Phase 1 study that evaluated the efficacy and safety of emicizumab as prophylaxis in Japanese hem A patients
with or without FVIII inhibitors was presented at ASH. The study demonstrated a clinically acceptable safety profile and
potential for prevention of bleeding in patients with and without FVIII inhibitors.
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Product Development: Obinutuzumab GA101 (Gazyvaro/Gazyva) is a glycoengineered monoclonal antibody which attaches to
the protein CD20. CD20 is found on the surface of all B-lymphocytes but is over expressed on cancerous B-lymphocytes.
Obinutuzumab attaches to the CD20 expressed in the cancerous cells thus killing the cancer cells and allowing the body to
replace those cells with normal cells.
Class: CD20 antibody
Treatments (approved or in clinical trial): chronic lymphocytic leukemia (CLL), nonHodgkin’s lymphoma (NHL)
2016 Jul: The Phase 3 GOYA study failed to meet its endpoint of progression-free survival. GOYA compared obinutuzumab
plus CHOP with rituximab plus CHOP as 1st line therapy for the treatment of DLBCL.
2016 Jun: EMA APP in for use in combination with bendamustine followed by Gazyvaro maintenance in patients with
follicular lymphoma who did not respond or who progressed during or up to six months after treatment with rituximab.
2016 May: Topline data from the Phase 3 GALLIUM study was released. The study compared obinutuzumab plus
chemotherapy followed by obinutuzumab alone with rituximab plus chemotherapy followed by rituximab alone in treatmentnaïve FL or indolent NHL patients. The primary endpoint was progression-free survival (PFS). The study met its primary
endpoint improving PFS compared with rituximab.
2016 Apr: EMA CHMP positive opinion received for use in combination with bendamustine as maintenance therapy in patients
with follicular lymphoma who did not respond or whose disease progressed after taking MabThera (the average length of time
from EMA CHMP to approval in the EU for hematology products listed in PDD is 3 months).
2016 Feb: FDA APP received for use with bendamustine followed by obinutuzumab alone as 2nd line therapy in follicular
lymphoma (FL), the most common type of NHL, in patients who did not respond to rituximab or in patients whose lymphoma
returned after treatment with rituximab.
2015 Dec: Follow-up results from the Phase 3 GADOLIN study were presented at ASH. GADOLIN compared obinutuzumab
plus bendamustine (followed by obinutuzumab alone) with bendamustine alone in patients with indolent NHL who were
refractory to rituximab. A subgroup analysis showed that obinutuzumab provided significantly greater depth of remission in
follicular lymphoma patients.
2015 Dec: Updated data from the Phase 3 CLL11 study was presented at ASH. CLL11 compared obinutuzumab plus
chlorambucil or rituximab plus chlorambucil with chlorambucil alone in patients with CLL. The obinutuzumab cohort
demonstrated a 28.7 month progression-free survival (PFS) versus 15.7 months for rituximab.
2015 Jun: The Phase 3 GADOLIN study was presented at ASCO and EHA. The study met its primary endpoint improving PFS
by 45% compared to bendamustine.
2015 Feb: The Phase 3 GADOLIN study was stopped early due to significant benefit observed in the obinutuzumab cohort.
The study met its primary endpoint of PFS.
2014 Nov: Approved in Canada for the treatment of CLL.
2014 Jul: EMA APP received for use in combination with chlorambucil for 1st line therapy in CLL patients who have
comorbidities making them unsuitable for intensive fludarabine-based therapy.
2014 May: EMA CHMP positive opinion received for use in combination with chlorambucil, for 1st line treatment of CLL in
adults who have comorbidities making them unsuitable for full-dose fludarabine therapy.
2014 Mar: A Phase 3 study was published in NEJM. The study compared obinutuzumab plus chlorambucil with rituximab plus
chlorambucil or placebo plus chlorambucil in treatment-naïve CLL patients with a CIRS score >6. The primary endpoint was
PRODUCT DETAIL – ROCHE
Roche
Gazyvaro
Gazyva
Research and Regulatory Timeline Detail
90
Research and Regulatory Timeline Detail
Roche
EU: Gazyvaro
US: Gazyva
Continued
PFS. The study met its endpoint with the obinutuzumab cohort experiencing significantly improved PFS compared with the
chlorambucil alone and the rituximab plus chlorambucil cohort.
2013 Dec: Data from the second stage of the Phase 3 CLL11 study was presented at ASH. The analysis showed that
obinutuzumab plus chlorambucil significantly reduced the risk of disease worsening or death by 61% compared with
rituximab in previously untreated CLL.
2013 Nov: FDA APP received for use in combination with chlorambucil, for the treatment of previously untreated CLL.
2013 Jul: Topline data from the Phase 3 CLL11 study was released. The study met its primary endpoint showing a significant
improvement in progression-free survival (PFS) in the obinutuzumab cohort compared with the rituximab cohort.
Roche
ORY-1001
Product Description: ORY-1001 is a selective lysine specific demethylase-1 inhibitor (LSD1). Poorer outcomes in some
cancers have been attributed to the expression of LSD1. By inhibiting this gene, it is thought that ORY-1001 may be effective
in treating some cancers by inhibiting colony formation and inducing cell apoptosis.
Class: LSD1 inhibitor
Treatments (approved or in clinical trial): acute lymphoblastic leukemia (ALL), acute myeloid
leukemia (AML)
2013 Jul: EMA COMP for the treatment of AML.
Roche
MabThera
Product Description: Rituximab (MabThera) is a monoclonal CD20 antibody. Rituximab targets the CD20 antigen, which is
present on the surface of B-lymphocytes. Once attached to the antigen it causes cell death. Rituximab is effective against
lymphoma and chronic lymphocytic leukemia (CLL) due to the fact that it destroys cancerous B-lymphocytes. In rheumatoid
arthritis it destroys B-lymphocytes in the joints thus reducing inflammation.
Class: CD20 Treatments (approved or in clinical trial): non-Hodgkin’s lymphoma (NHL) diffuse large B-cell
lymphoma (DLBCL), follicular lymphoma (FL), chronic lymphocytic leukemia (CLL), Bullous pemphigoid
2016 Jul: (see Roche: obinutuzumab) GOYA failed to meet its primary endpoint.
2016 Jun: Lancet published a Phase 3 study that compared rituximab plus chemotherapy with chemotherapy alone in patients
with Burkitt’s lymphoma or leukemia who were HIV negative and had no history of other cancers (except for non-melanoma
skin cancers and Stage 0 cervical carcinoma). Patients were stratified into two groups Group B (no presence of bone marrow
or CNS involvement) and Group C ( presence of bone marrow or CNS involvement). The primary endpoint was 3-year
event-free survival (EFS). The study met its endpoint with 75% of the rituximab combination cohort achieving 3-year EFS
versus 62% for the chemotherapy alone cohort.
2016 May: JCO published Phase 1 data from a Phase 1/2 study that evaluated the safety and efficacy of TMZ therapy plus
rituximab followed by hyperfractionated whole-brain radiotherapy and subsequent TMZ therapy in patients with CNS
lymphoma. The Phase 1 portion increased doses of TMZ plus 375mg/m2 rituximab. The regimen was safe with the best 2year overall survival and progression-free survival in any Radiation Therapy Oncology Group primary CNS lymphoma trial.
2016 May: (see Roche: obinutuzumab) Topline data from GALLIUM was released.
2015 Nov: (see Celgene: lanalidomide) A Phase 2 study was published in NEJM.
PRODUCT DETAIL – ROCHE
Product
91
Research and Regulatory Timeline Detail
Sandoz
rituximab
biosimilar
Product Description: Rituximab biosimilar is a biosimilar to Roche’s monoclonal CD20 antibody MabThera (rituximab).
Rituximab targets the CD20 antigen, which is present on the surface of B-lymphocytes. Once attached to the antigen it causes
cell death. Rituximab is effective against lymphoma and chronic lymphocytic leukemia (CLL) due to the fact that it destroys
cancerous B-lymphocytes. In rheumatoid arthritis it destroys B-lymphocytes in the joints thus reducing inflammation.
Class: CD20 Treatments (approved or in clinical trial): non-Hodgkin’s lymphoma (NHL) diffuse large B-cell lymphoma
(DLBCL), follicular lymphoma (FL), chronic lymphocytic leukemia (CLL), rheumatoid arthritis (RA)
2016 Jun: A Phase 2 study was presented at EULAR. The study compared rituximab biosimilar with MabThera. The study
demonstrated PK bioequivalence and similar PD, safety, efficacy, and immunogenicity between the two products.
2016 May: EMA MAA submitted for all approved for MabThera indications (NHL, DLBL, CLL & RA; the average length
of time from EMA MAA to approval in the EU for hematology products listed in PDD is 14 months).
Sanofi
SAR302503
Product Description: SAR302503 is a Janus kinase 2 inhibitor (JAK-2). The JAK enzymes are involved in the production of
blood cells. In myelofibrosis there is too much JAK activity in the bone marrow and subsequent abnormal production of blood
cells which migrate to organs, including the spleen, leading to enlargement of these organs. SAR302503 blocks JAK
production and thus reduces symptoms caused by JAK induced disorders.
Class: JAK-2
Treatments (approved or in clinical trial): myelofibrosis (MF), polycythemia vera (PCV), essential
thrombocythemia (TC)
2013 May: Topline data from the Phase 3 JAKARTA study was released. JAKARTA compared SAR302503 with placebo in
patients with intermediate-2 or high-risk primary myelofibrosis, post-polycythemia vera, myelofibrosis, or post-essential
thrombocythemia myelofibrosis. The study met its primary endpoint, achieving a ≥35% reduction in spleen volume compared
with placebo.
PRODUCT DETAIL – SANDOZ, SANOFI
Product
92
Research and Regulatory Timeline Detail
Seattle
Genetics
vadastuximab
talirine
Product Description: Vadastuximab talirine (SGN-CD33A; 33A) is an antibody-drug conjugate (ADC) targeting CD33.
CD33 is expressed on most acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) blast cells. The CD33
antibody is attached to Seattle Genetics’ ADC technology binding agent, a pyrrolobenzodiazepine (PBD) dimer. PBD dimers
are more potent than systemic chemotherapeutic drugs and the site-specific conjugation technology allows uniform drugloading of the cell-killing PBD agent to the anti-CD33 antibody. The ADC is designed to be stable in the bloodstream and
only releases the DNA binding agent upon internalization into the CD33-expressing cell.
Class: CD33 agonist Treatments (approved or in clinical trial): AML, MDS
2016 Jun: A Phase 1 study was presented at the European Hematology Association Congress (EHA). The study evaluated the
safety and efficacy of vadastuximab talirine in combination with hypomethylating agents (azacitidine, decitabine) in frontline
AML patients who had declined intensive therapy. The overall response rate was 76% and complete remission or complete
remission with incomplete platelet or neutrophil recovery was observed in 71% of patients.
Spectrum
Beleodaq
Product Description: Belinostat (Beleodaq) is a histone deacetylase inhibitor (HDAC). The exact mechanism of action is
unknown in cancer. However, HDACs are involved in the pathway by which retinoblastoma protein (pRb) suppresses cell
proliferation.
Class: HDAC Treatments (approved or in clinical trial): peripheral T-Cell lymphoma (PTCL), breast cancer (BC),
ovarian cancer (OC)
2015 Dec: A Phase 1 study was presented at ASH. The study evaluated the efficacy and safety of belinostat plus CHOP in
newly diagnosed PTCL patients. The primary endpoint was objective response rate (ORR). The study demonstrated an ORR
of 86%.
2015 Jun: The Journal of Clinical Oncology (JCO) published the Phase 2 BELIEF study. The study evaluated the safety and
efficacy of belinostat in patients with refractory/relapsed PTCL who had received at least 1 prior systemic therapy. The
primary endpoint was ORR. The study met its endpoint achieving a 25.8% ORR.
2014 Jul: FDA APP received for the treatment of relapsed/refractory PTCL.
2013 Dec: FDA NDA for the treatment of relapsed/refractory PTCL.
PRODUCT DETAIL – SEATTLE GENETICS, SPECTRUM
Product
93
Research and Regulatory Timeline Detail
Spectrum
Evomela
Product Description: Melphalan (Evomela) is a propylene glycol-free nitrogen mustard alkylating agent (l-phenylalanine
mustard). Melphalan works by inhibiting DNA and RNS synthesis, functions that are necessary for a cell to survive. These
changes cause cytotoxicity in both dividing and non-dividing tumor cells. Captisol-enabled (CE) melphalan is propylene
glycol-free, thus reducing renal and cardio-toxicity associated with propylene glycol.
Class: l-phenylalanine mustard (L-PAM)
Treatments (approved or in clinical trial): conditioning treatment
prior to hematopoietic progenitor cell transplantation in multiple myeloma (MM), palliative treatment in MM
2016 Apr: FDA ODD received for 7 year exclusivity.
2016 Mar: FDA APP for use in high-dose conditioning treatment prior to stem cell transplantation in MM patients and as
palliative treatment for MM patients for whom oral therapy is not appropriate.
2015 Oct: FDA CRL was issued requiring more data before consideration of approval.
2015 Sep: The Biology of Blood and Transplantation Journal (BBMT) published a Phase 2b study. The study evaluated the
efficacy of CE melphalan in patients with newly diagnosed or refractory/relapsed MM. The primary endpoint was overall
response rate (ORR). The study achieved it endpoint with 95% of patients achieving ORR.
2015 Feb: A Phase 2 study was presented at BMT. The study evaluated the efficacy and safety of CE melphalan in achieving
myeloablation in MM patients undergoing stem cell transplantation. 100% of subjects achieved myeloablation.
2014 Dec: FDA NDA for high-dose conditioning treatment prior to stem cell transplantation in patients with MM.
2014 Apr: Topline data from a Phase 2 study was released. The study evaluated the safety and efficacy of CE melphalan in
MM patients in preparation for autologous stem cell transplantation (ASCT). The study met its primary endpoint of efficacy
and safety.
Spectrum
SPI-2012
Product Description: SPI-2012 is a long-acting granulocyte-colony stimulating factor (GCSF) using a proprietary platform
technology called, LAPSCOVERY. GCSF stimulates proliferation of granulocyte progenitors and subsequent production of
neutrophils in bone marrow.
Class: GCSF
Treatments (approved or in clinical trial): chemotherapy induced neutropenia
2014 Sep: Topline data from a Phase 2 study was released. The study compared SPI-2012 with pegfilgrastim in patients with
breast cancer who has received adjuvant or neoadjuvant chemotherapy. The primary endpoint was mean duration of
neutropenia during Cycle 1. The study met its primary endpoint.
Spectrum
Marqibo
Product Description: Vincristine liposome (Marqibo) is a liposomal formulation of the vinca alkaloid vincristine. Vincristine
binds to the tubulin protein, stopping the cell from separating its chromosomes during the metaphase, leading to cell
apoptosis. Because vincristine targets all rapidly dividing cells (both cancerous and healthy) it has serious side-effects. The
liposomal formulation decreases side effects by directly delivering the drug to tumor cells.
Class: vinca alkaloid
Treatments (approved or in clinical trial): Philadelphia chromosome negative acute
lymphoblastic leukemia (PH- ALL), diffuse large B-cell lymphoma (DLBCL)
2013 Dec: A Phase 2 study was presented at ASH. The study compared vincristine liposome (R-CHMP) to vincristine in RCHOP therapy in treatment-naïve DLBCL patients. The primary endpoint was overall response rate (ORR). The study met its
primary endpoint with an ORR of 95% without increased toxicity.
PRODUCT DETAIL – SPECTRUM
Product
94
Research and Regulatory Timeline Detail
Syros
SY-1425
Product Description: SY-1425 is a selective RARα agonist. Some tumors have a highly specialized regulatory region of noncoding DNA, known as a super-enhancer, which is associated with the RARA gene. Studies have shown that the RARA
biomarker is predictive of response to treatment with SY-1425. SY-1425 inhibits cancer growth and prolongs survival in
patients whose tumors have the RARA marker.
Class: RARα agonist Treatment (approved or in clinical trial): acute myeloid leukemia (AML), myelodysplastic
syndrome (MDS), acute promyelocytic leukemia
2016 May: FDA IND for the treatment of AML and MDS.
Takeda/
Millenium
alisertib
Product description: Alisertib is an aurora A kinase inhibitor (AAK). Aurora A is very important in the mitosis process. Aurora
A dysregulation is associated with cancer cell formation. Over-expression of Aurora A is common in cancer cells and can
prohibit cell division in the mitosis process leading to the cell becoming aneuploidy, meaning the cell is not able to divide after
duplicating its DNA, leading to too many chromosomes within the cell. Aneuploidy is a common trail in cancer cells. Alisertib
inhibits this process.
Class: AAK inhibitor Treatments (approved or in clinical trial): breast cancer (BC), peripheral T-cell lymphoma (PTCL),
pancreatic cancer (PAN), small-cell lung cancer (SCLC)
2015 May: A Phase 3 trial for PTCL was terminated due to lack of efficacy. The study did not meet its primary endpoint of
progression-free survival.
PRODUCT DETAIL – SYROS, TAKEDA
Product
95
Research and Regulatory Timeline Detail
Takeda/
Millenium
Velcade
Product Description: Bortezomib (Velcade) is a proteasome inhibitor. . Proteasome is the system within cells which breaks
down cell proteins when they are no longer needed by the cell. When this process is stopped, and the unneeded protein is no
longer broken down the cell eventually dies.
Class: Proteasome inhibitor
Treatments (approved or in clinical trial): multiple myeloma (MM), mantle cell
lymphoma (MCL), primary plasma cell leukemia (pPCL)
2016 Jun: The Journal of Clinical Oncology (JCO) published the prospective Phase 2 Intergroup Francophone du Myélome
(IFM) study. The study evaluated the efficacy of a regimen that combined standard chemotherapy, a proteasome inhibitor, and
high dose melphalan and autologous stem cell transplantation (HDM/ASCT) followed by either allogeneic transplantation or
bortezomib plus lenalidomide maintenance therapy in patients with pPCL . The primary endpoint was progression-free survival
(PFS). Bortezomib plus dexamethasone and doxorubicin or cyclophosphamide induction followed by transplantation induced
high response rates and significantly improved PFS.
2016 Jun: (see Janssen; daratumumab) The CASTOR study was presented at ASCO.
2016 May: Blood published the independently funded Phase 3 IFM2013-04 study. The study compared VTD (bortezomib plus
thalidomide and dexamethasone) with VCD (bortezomib plus cyclophosphamide and dexamethasone) as induction therapy
before autologous stem cell transplantation in patients with newly diagnosed MM. The study met its primary endpoint with
66.3% of the VTD cohort achieving at least partial response versus 56.2% for the VCS cohort.
2016 Apr: (see Janssen: daratumumab) Interim data from the CASTOR study was released and the study was stopped early.
2015 Mar: NEJM published the Phase 3 LYM-3002 study. The study compared VR-CHOP therapy (R-CHOP regimen,
replacing vincristine with bortezomib) with R-CHOP therapy (rituximab, cyclophosphamide, doxorubicin, vincristine, and
prednisone) in treatment-naïve MCL patients ineligible for stem-cell transplantation. The primary endpoint was progression-free
survival (PFS). The study met its primary endpoint with PFS of 24.7 months for VR-CHOP versus 14.4 months for R-CHOP.
2014 Oct: FDA APP received for use previously untreated patients with MCL.
2014 Aug: FDA APP received for the retreatment of patients with MM who previously responded to bortezomib and relapsed
at least 6 months after prior bortezomib therapy.
2013 Dec: A retrospective analysis of the Phase 3 VISTA study was presented at ASH. Vista compared bortezomib plus
melphalan and prednisone with melphalan plus prednisone as 1st line therapy in MM, The retrospective analysis showed that a
higher cumulative dose of bortezomib suggests improved overall survival.
2013 Aug: EMA APP received for use as induction therapy in combination with dexamethasone or thalidomide plus
dexamethasone in previously-untreated MM patients who are eligible for high-dose chemotherapy and stem cell transplantation.
PRODUCT DETAIL – TAKEDA
Product
96
Research and Regulatory Timeline Detail
Takeda/
Millenium
Adcetris
Product Description: Brentuximab vedotin (Adcetris) is a combination therapy combining the antibody-drug conjugate CD30
and the antimitotic agent monomethyl auristatin E (MMAE). The compound works by the antibody attaching itself to the cellmembrane protein CD30, which is commonly expressed in some cancers but is rarely expressed in healthy cells. Once the drug
has bound to the receptor it delivers the cytotoxic agent (MMAE) into the cell, leading to cell death.
Class: CD30/MMAE
Treatments (approved or in clinical trial): Hodgkin’s lymphoma (HL), systemic anaplastic
large cell lymphoma (ALCL)
2016 Jul: Blood published the Phase 2 ADCETRIS study. The study evaluated the safety and efficacy of Adcetris in
relapsed/refractory ALCL and HL. The study showed that 41% of ALCL patients were alive at 5 years and that the median
overall survival rate was 40.5 months.
2016 Jul: EMA APP received for the treatment of adults with CD30+ HL at increased risk of relapse or progression following
autologous stem cell transplantation (ASCT).
2016 May: EMA CHMP positive opinion received for the treatment of adults with CD30+ HL at increased risk of relapse or
progression following autologous stem cell transplantation (ASCT).
2016 Jan: EMA APP received for the retreatment of adult patients with rrHL and rrsALCL.
2015 Dec: Four-year follow up data from the Phase 2 ADCETRIS study was presented at ASH. The study showed that 41% of
ALCL patients were alive at 5 years and that the median overall survival rate was 40.5 months.
2015 May: Lancet published the Phase 3 AETHERA study. AETHERA compared brentuximab vedotin with placebo in HL
patients who had autologous stem cell therapy and were at risk of relapse. The primary endpoint was progression-free survival
(PFS). The study met its primary endpoint achieving a median survival of 43 months versus 24 months for placebo.
2015 Oct: EMA CHMP positive opinion received for retreatment of adult patients with rrHL and rrsALCL.
2014 Dec: The Phase 3 AETHERA study was presented at ASH. The study met its primary endpoint of progression-free
survival (PFS).
2014 Dec: Four-year follow up data from the Phase 2 ADCETRIS study was presented at ASH. The study evaluated the safety
and efficacy of Adcetris in relapsed/refractory ALCL and HL patients. The study showed that 60% of ALCL patients were alive
at 4 years.
2014 Sep: Topline data from the Phase 3 AETHERA study was released. The study met its primary endpoint of PFS.
2014 Jan: Approved in Japan for the treatment of CD30+ relapsed/refractory HL and relapsed/refractory ALCL.
2013 Dec: Two Phase 2 studies were presented at ASH. In the first study brentuximab vedotin achieved a 40.5 month overall
survival rate among patients with refractory/relapsed HL. In the second study brentuximab vedotin had not yet achieved an
overall survival rate at three years.
PRODUCT DETAIL – TAKEDA
Product
97
Research and Regulatory Timeline Detail
Takeda/
Millenium
Ninlaro
Product Description: Ixazomib (Ninlaro) is a proteasome inhibitor. Ixazomib prevents the process by which proteasomes
breakdown unneeded proteins within cells. By blocking this process in abnormal cells the proteins within the cell do not
breakdown leading to apoptosis.
Class: proteasome inhibitor Treatments (approved or in clinical trial): systemic light-chain amyloidosis (AL),
multiple myeloma (MM)
2016 Jul: Japan NDA for relapsed/refractory MM.
2016 May: EMA CHMP negative opinion was received for the use of ixazomib in the treatment of relapsed/refractory MM.
2016 Apr: NEJM published the Phase 3 TOURMALINE-MM1 study. The study compared ixazomib plus lenalidomide and
dexamethasone with placebo plus lenalidomide plus dexamethasone with relapsed/refractory MM. The primary endpoint was
progression-free survival (PFS). The study met its endpoint with the ixazomib cohort achieving 20.6 month PFS compared
with 14.7 months for placebo.
2015 Dec: The Phase 3 TOURMALINE-MM1 study was presented at ASH. TOURMALINE-MM1 compared ixazomib
plus lenalidomide and dexamethasone with placebo plus lenalidomide and dexamethasone in patients with relapsed/refractory
MM. The study met its primary endpoint improving progression-free survival (PFS) by a significant 35% compared with
placebo.
2015 Nov: FDA APP received for use in combination with lenalidomide and dexamethasone for MM patients who have
received at least one prior therapy.
2015 Sep: FDA PRD received for the treatment of relapsed/refractory MM.
2015 Jul: EMA MAA for the treatment of relapsed/refractory MM, accelerated assessment.
2015 Jul: FDA NDA for the treatment of relapsed/refractory MM.
2015 Feb: An interim analysis of the Phase 3 TOURMALINE-MM1 study was released. The study met its primary endpoint
of improving PFS at the interim analysis.
2014 Dec: A Phase 2 study was presented at ASH. The study evaluated the safety and efficacy of ixazomib as maintenance
therapy in patients with MM. 71% of patients had a complete or partial very good response.
2014 Dec: FDA BTD received for the treatment of relapsed/refractory systemic light-chain AL.
Takeda/
Millenium
MLN9708
Product Description: MLN9708 is a proteasome inhibitor. Proteasome is the system within cells which breaks down cell
proteins when they are no longer needed by the cell. When this process is stopped, and the unneeded protein is no longer
broken down the cell eventually dies.
Class: Proteasome inhibitor
Treatments (approved or in clinical trial): multiple myeloma (MM)
2013 Dec: Preliminary results from a Phase 1/2 study were presented at ASH. The study showed that MLN9708 achieved a
complete/very good partial response rate of 76% and an overall response rate of 94% in patients with newly diagnosed MM.
PRODUCT DETAIL – TAKEDA
Product
98
Research and Regulatory Timeline Detail
Tesaro
rolapitant
Product Description: Rolapitant is a neurokinin-1 antagonist (NK-1). NK-1 receptors are concentrated in the center of the
brain that controls vomiting. Rolapitant prevents the binding of Substance P to the NK-1 receptor in the brain. By blocking
this interaction rolapitant improves the management of nausea and vomiting in chemotherapy patients.
Class: NK-1 antagonist Treatment (approved or in clinical trial): chemotherapy induced nausea and vomiting (CINV)
2016 Mar: EMA MAA for CINV (the average length of time from EMA MAA to approval in the EU for hematology
products listed in PDD is 14 months).
2016 Mar: FDA sNDA for IV formulation.
2015 Sep: FDA APP for CINV in combination with other antiemetics.
2015 May: Topline data from a bioequivalence study was released. The study showed that the IV formulation of rolapitant
was bioequivalent to the oral formulation.
2014 Sep: FDA NDA for prevention of CINV (the average length of time from FDA NDA to approval in the US for
hematology products listed in PDD is 7 months).
2014 Jun: Three Phase 3 studies were presented at ASCO. The primary endpoint in all three studies was complete response
(CR; no vomiting). The first study compared rolapitant with a 5-HT3 antagonist plus dexamethasone in patients with
cisplatin-based highly emetogenic chemotherapy. The study met its endpoint with 70% of the rolapitant cohort achieving CR
compared with 82% for the control group. The second study compared rolapitant with a 5-HT3 receptor antagonist plus
dexamethasone in patients taking highly emetogenic chemotherapy. The study achieved its primary endpoint with 73% of the
rolapitant cohort achieving CR versus 58% for control. The third study compared rolapitant with 5-HT3 antagonist plus
dexamethasone in patients receiving moderately emetogenic chemotherapy. The study met its primary endpoint with the
rolapitant cohort achieving significant improvement in CR during the delayed phase; 71% versus 62% for the control group.
2014 May: Topline data from a Phase 3 study was released. The study compared rolapitant with a 5-HT3 antagonist plus
dexamethasone in patients with cisplatin-based highly emetogenic chemotherapy. The primary endpoint was CR. The study
met its primary endpoint with the rolapitant cohort achieving significant improvement in CR.
2013 Dec: Topline data from two Phase 3 studies released. The first study compared rolapitant with placebo in patients
receiving moderately emetogenic chemotherapy. The primary endpoint was CR. The study met is primary endpoint. The
second study compared rolapitant with a 5-HT3 receptor antagonist plus dexamethasone in patients taking highly emetogenic
chemotherapy. The primary endpoint was CR. The study met its primary endpoint with significant improvement in CR for the
rolapitant compared with the control cohort.
PRODUCT DETAIL – TESARO
Product
99
Research and Regulatory Timeline Detail
Triphase
marizomib
Product Description: Marizomib is a IV/PO proteasome inhibitor that irreversibly binds and inhibits all three proteasome
subunits, translating into longer duration of effect and possible improved clinical activity. Proteasome is the system within
cells which breaks down cell proteins when they are no longer needed by the cell. When this process is stopped, and the
unneeded protein is no longer broken down the cell eventually dies.
Class: IV/PO Proteasome inhibitor Treatments (approved or in clinical trial): multiple myeloma (MM), glioblastoma
2016 Jun: A Phase 1 dose-escalation study was presented at ASCO. The study evaluated three dose regimens of marizomib
plus bevacizumab in patients with WHO Grade IV malignant glioma. The response rate was 45% in efficacy evaluable
patients and 39% in the intent-to-treat population.
2016 Jun: Blood published Phase 1 results from the Phase1/2 NPI-52-101 study. The primary objective was to determine the
maximum tolerated dose and to recommended a Phase 2 dose when administered intravenously to patients with relapsed MM.
Study results indicated that marizomib was well tolerated and suggested clinical activity in treatment-experienced
relapsed/refractory MM patients.
2015 Nov: FDA ODD for malignant glioma.
2015 Sep: A Phase 1 dose escalation study was presented at the International Myeloma Society 15 th International Myeloma
Workshop (IMS). The study evaluated marizomib plus pomalidomide and low dose dexamethasone in patients with
relapsed/refractory MM. The combination therapy decreased myeloma proteins by ≥50% and achieved an overall response
rate of 62%.
2014 Feb: FDA ODD for MM.
PRODUCT DETAIL – TRIPHASE
Product
100
Drug or Device
Class
EU
US
Company
Acute
Lymphoblastic
Leukemia (ALL)
Blincyto (PH-ALL)
Iclusig (PH+ ALL)
Marqibo (PH- ALL)
Oncaspar
Sprycel (PH+ ALL)
CD19/CD3
TKI
vinca alkaloid
asparagine
MKI
Approved
Approved
Not Approved
Approved
Approved
Approved
Approved
Approved
Approved
Approved
Amgen
Ariad
Spectrum
Baxalta
BMS
Acute Myeloid
Leukemia (AML)
pacritinib
quizartinib
tosedostat
Vidaza
JAK inhibitor
FLT3 inhibitor
APD inhibitor
epigenetic
Not Approved
Not Approved
Not Approved
Approved
Not Approved
Not Approved
Not Approved
Not Approved
CTI Biopharma
Daiichi/Ambit
CTI Biopharma
Celgene
Acquired Severe
Aplastic Anemia
(ASAA)
EU: Revolade US: Promacta
TPOR
Approved
Approved
Novartis
Anaplastic Large
Cell Lymphoma
(ALCL)
Adcetris
CD30/MMAE
Approved
Approved
Takeda
Anemia
Aranesp
Injectafer (IDA)
ESA
iron
Approved
Approved
Approved
Approved
Amgen
Daiichi
Aspergillosis
Mucormycosis
Cresemba
antifungal
Approved
Approved
Astellas
Atypical Hemolytic
Uremic Syndrome
(aHUS)
Soliris
C5
Approved
Approved
Alexion
Beta-thalassemia
(BT)
luspatercept
sotatercept
TGF-β
TGF-β
Not Approved
Not Approved
Not Approved
Not Approved
Celgene/Acceleron
Celgene/Acceleron
Bone Metastasis
Xgeva
RANKL
Approved
Approved
Amgen
Cachexia
anamorelin
GR antagonist
Not Approved
Not Approved
Helsinn
DRUG LIST BY INDICATION
Indication
101
Drug or Device
Class
EU
US
Company
ChemotherapyInduced Nausea
and Vomiting
(CINV)
Akynzeo
Aloxi
Emend
Syndros
Varubi
antiemetic
antiemetic
Antiemetic
cannabinoid
antiemetic
Approved
Approved
Approved
Not Approved
Not Approved
Approved
Approved
Approved
Approved
Approved
Helsinn/Chugai/Eisai
Helsinn
MSD
Insys
Tesaro
Chronic Iron
Overload (CIO)
Jadenu
chelator
Not Approved
Approved
Novartis
Chronic
Lymphocytic
Leukemia (CLL)
Arzerra
ASP2215
Gazyvaro/Gazyva
Imbruvica
MabThera
Venclexta
volasertib
Zydelig
CD20
TKI
CD20
BTK
CD20
BCL-2
PlK1
PI3K
Approved
Not Approved
Approved
Approved
Approved
Not Approved
Not Approved
Approved
Approved
Not Approved
Approved
Approved
Approved
Approved
Not Approved
Approved
Novartis
Astellas
Roche
US: AbbVie EU: J&J Janssen
Roche
AbbVie
Boehringer Ingelheim
Gilead Sciences
Chronic Myeloid
Leukemia (CML)
Bosulif
Iclusig (T3151+)
Sprycel (PH+ CML)
Tasigna (PH+ CML)
TKI
TKI
MKI
TKI
Approved
Approved
Approved
Approved
Approved
Approved
Approved
Approved
Pfizer
Ariad
BMS
Novartis
Chronic
Myelomonocytic
Leukemia (CMML)
Vidaza
epigenetic
Approved
Approved
Celgene
Diffuse Large BCell Lymphoma
(DLBCL)
Enzastaurin
MabThera
VGEF inhibitor
CD20
Not Approved
Approved
Not Approved
Approved
Eli Lilly
Roche
End-Stage Kidney
Disease (ESKD)
EU: Vivia US: Amia
dialysis
Approved
Approved
Baxter
DRUG LIST BY INDICATION
Indication
102
Drug or
Device
Class
EU
US
Hemophilia A (hem A)
Advate
Adynovate
Afstyla
ALN-AT3
Bax 187
Bax 855
Kovaltry
damoctocog
EU: Elocta US: Eloctate
FEIBA NF
Kogenate
Obizur (acquired hem A)
rFVIII
rFVIII
rFVIII
RNAi
rFVIII
rFVIII
rFVIII
rFVIII
rFVIII
AICC
rFVIII
pdFVIII
Approved
Not Approved
Not Approved
Not Approved
Not Approved
Not Approved
Approved
Not Approved
Not Approved
Not Approved
Approved
Approved
Approved
Approved
Approved
Not Approved
Not Approved
Not Approved
Approved
Not Approved
Approved
Approved
Approved
Approved
Baxalta
Baxalta
CSL Behring
Alnylam
Baxalta
Baxalta
Bayer
Bayer
Biogen
Bayer
Bayer
Baxter
Hemophilia B (hem B)
Alprolix
ALN-AT3
Bax 187
BeneFIX
FIEBA NF
Idelvion
Rixubis
rFIX
RNAi
rFVIII
rFIX
AICC
rFIX
rFIX
Approved
Not Approved
Not Approved
Approved
Not Approved
Approved
Approved
Approved
Not Approved
Not Approved
Approved
Approved
Approved
Approved
Biogen
Alnylam
Baxalta
Pfizer
Baxalta
CSL Behring
Baxalta
Hodgkin’s Lymphoma
(HL)
Adcetris
Opdivo
CD30/MMAE
PD20 inhibitor
PD-1 inhibitor
Approved
Not Approved
Not Approved
Approved
Approved
Approved
Takeda
Pfizer
BMS
Hypercalcemia of
Malignancy (HCM)
XGEVA Prolia
RANKL
Not Approved
Approved
Amgen
Immune
Thrombocytopenic
Purpura (ITP)
Nplate
Revolade Promacta
THPO
TPOR
Approved
Approved
Approved
Approved
Amgen
Novartis
EU: Gazyvaro US: Gazyva
Company
DRUG LIST BY INDICATION
Indication
103
Drug or Device
Class
EU
US
Company
Mantel Cell
Lymphoma (MCL)
Imbruvica
Revlimid
Velcade
BTK
IMiDs
proteasome
Approved
Not Approved
Approved
Approved
Approved
Approved
US: AbbVie EU: J&J Janssen
Celgene
Takeda/Millenium
Multicentric
Castleman’s Disease
(MCD)
Sylvant
IL-6
Approved
Approved
J&J Janssen
Multiple Myeloma
(MM)
Darzalex
Empliciti
Evomela
Farydak
Kyprolis
EU: Imnovid US: Pomalyst
Ninlaro
Revlimid (rrMM)
Velcade
CD38
CS1
L-PAM
HDAC
proteasome
IMiDs
proteasome
IMiDs
proteasome
Approved
Approved
Not Approved
Approved
Approved
Approved
Not Approved`
Approved
Approved
Approved
Approved
Approved
Approved
Approved
Approved
Approved
Approved
Approved
J&J Janssen
BMS
Spectrum
Novartis
Amgen
Celgene
Takeda/Millenium
Celgene
Takeda/Millenium
Myelodysplastic
Syndromes (MDS)
Revlimid
luspatercept
sotatercept
Tosedostat
Vidaza
IMiDs
TGF-β
TGF-β
APD inhibitor
epigenetic
Approved
Not Approved
Not Approved
Not Approved
Approved
Approved
Not Approved
Not Approved
Not Approved
Approved
Celgene
Celgene/Acceleron
Celgene/Acceleron
CTI Biopharma
Celgene
Myelofibrosis (MF)
Jakavi
JAK
Approved
Not Approved
Novartis
Non-Hodgkin's
Lymphoma (NHL)
Gazyva (FL)
MabThera (FL)
Pixuvri
Zydelig (FL)
CD20
CD20
anthracycline
PI3K
Approved
Approved
Approved
Approved
Approved
Approved
Not Approved
Approved
Roche
Roche
CTI Biopharma
Gilead
Pain
Xtampza (severe)
opioid
Not Approved
Approved
Collegium
Paroxysmal
Nocturnal
Hemoglobinuria
(PNH)
Soliris
C5
Approved
Approved
Alexion
DRUG LIST BY INDICATION
Indication
104
Drug or Device
Class
EU
US
Company
Peripheral T-Cell
Lymphoma (PTCL)
Beleodaq
HDAC
Not Approved
Approved
Spectrum
Polycythemia vera
(PCV)
Jakavi
JAK
Approved
Approved
Novartis
Primary
Immunodeficiency
(PI)
HyQvia
IGSC
Approved
Approved
Baxalta
Refractory Anemia
(RA)
Vidaza
epigenetic
Not Approved
Approved
Celgene
Secondary
Immunodeficiency
(SI)
HyQvia
IGSC
Approved
Not Approved
Baxalta
Small Lymphocytic
Lymphoma (SLL)
Imbruvica
Zydelig
BTK
PI3K
Not Approved
Not Approved
Approved
Approved
AbbVie
Gilead
Thrombocytopenia
in Hep C
EU: Revolade US: Promacta
TPOR
Approved
Approved
Novartis
Veno-occlusive
Disease (VOD)
EU: Defitelio US: Gentium
anticoagulant
Approved
Approved
Jazz
von Willebrand
Disease (VWD)
Vonvendi
rVWF
Not Approved
Approved
Baxalta
Waldenström’s
Macroglobulinenia
(WM)
Inbruvica
BTK
Approved
Approved
US: AbbVie EU: J&J Janssen
DRUG LIST BY INDICATION
Indication
105
2015
Q4
2015
Q3
2015
Q2
71
66
54
235
76
0
532
(US 261)
843
(US 417)
653
(US 421)
3
(US3)
185
(US 80)
27
(US 21)
499
(US 249)
493
(US 239)
479
(US 223)
n/a
n/a
n/a
n/a
n/a
n/a
3
(US3)
194
(US 81)
2
(US2)
193
(US 87)
n/a
n/a
n/a
n/a
1930
(US 794)
2984
(US 1281)
1677
(US 1275)
5
(US5)
216
(US 102)
3
(US 3)
1911
(US 747)
2786
(US 1216)
1616
(US 1228)
3
(US3)
191
(US 87)
1951
(US 900)
2840
(US 1339)
1750
(US 1350)
10
(US10)
752
(US 325)
Darzalex
102
0
0
0
0
0
0
Defitelio
18
18
20
15
71
71
45
Eloctate
108
(US 99)
101
91
74
320
58
0
139
(US 80)
3
(US3)
34
(US 19)
33
(US 25)
343
(US 295)
235
(US 122)
141
(US 88)
134
(US 79)
553
(US 312)
507
(US 293)
n/a
27
(US 20)
n/a
40
(US 19)
n/a
107
(US 97)
154
(US 89)
n/a
49
(US 43)
56
(US 40)
n/a
3
(US 3)
n/a
204
(US 267)
184
(US 98)
536
(US 325)
3
(US3)
128
(US 76)
113
(US 86)
754
(US 659)
689
(US 375)
0
200
(US 145)
0
Alprolix
Aranesp
Baxalta hematology
Baxalta immunology
Beleodaq
BeneFIX
Blincyto (Amgen)
Emend
Empliciti
Gazyva
Gazyvaro
Iclusig
Imbruvica (AbbVie)
Imbruvica (Janssen)
2016
Q1
75
(US 65)
126
28
(US28)
45
34
(US25)
381
(US 325)
261
(US 132)
2015
2014
2013
0
213
(US 97)
0
GLOBAL REVENUE (US$ MIL)
Drug or Device
($mil)
n/a
0
Notes: 1) All revenues are recorded in US Dollars, some companies report their revenues in currencies other than US Dollars, in the event that a company reports in a currency other
than dollars, the reported figures are converted to USD as at the exchange rate on the last day of the reporting period. 2) All figures come from unaudited quarterly reports filed to
stockholders. Therefore, the quarterly totals may not equal the annual total. 3) Green indicates growth (in US Dollars) red indicates contracting sales. 4) If data is not available, it is
indicated by n/a. “0” indicates no sales for the period. 5) Top number represents global sales, numbers in parenthesis “(US66)” represent sales within the United States. If there are no
numbers in parenthesis, than only global data is provided.
106
Jadenu
Jakavi
Keytruda
Kogenate
Kyprolis
Marqibo
Opdivo
Pomalyst
Promacta/Revolade
Revlimid
Solaris
Sprycel
Tasigna
Varubi
Velcade
Xgeva/Prolia
Zydelig
2016
Q1
223
2015
Q4
2015
Q3
2015
Q2
2015
2014
2013
248
213
262
917
926
893
130
214
(US 132)
n/a
159
(US 109)
n/a
110
(US 86)
410
566
(US 392)
279
55
(US 48)
163
309
148
(US 134)
3
(US3)
475
(US 410)
294
(US 170)
334
137
(US 124)
1
(US1)
305
(US 268)
257
(US 150)
323
119
(US 112)
2
(US2)
122
(US 107)
235
(US 144)
1247
512
(US 467)
8
(US8)
942
(US 823)
983
(US 592)
n/a
73
(US 71)
131
n/a
n/a
n/a
415
1574
(US 997)
1561
(US 956)
1454
(US 895)
1444
(US 873)
5801
(US 3535)
1231
331
(US 306)
6
(US6)
6
(US 1)
680
(US 443)
360
(US 142)
2980
(US 2916)
665
689
665
636
2590
2146
1551
407
(US 210)
429
(US 228)
411
(US 215)
405
(US 205)
1620
(US 829)
1493
(US 671)
1280
(US 541)
432
416
412
1632
1529
1266
0
0
0
0
0
0
321
(US 0)
736
(US 501)
329
(US 0)
698
(US 478)
344
(US 0)
671
(US 449)
1333
2717
(US 1843)
1618
2251
(US 1482)
1660
1763
(US 1226)
40
36
30
132
23
0
124
249
340
(US 110)
154
(US 129)
1
(US1)
704
(US 594)
274
(US 171)
382
0.2
(US0.2)
304
(US 0)
730
(US 492)
0
1 (US1)
0
202
(US 132)
309
(US 121)
1323
(US 798)
Notes: 1) All revenues are recorded in US Dollars, some companies report their revenues in currencies other than US Dollars, in the event that a company reports in a currency other
than dollars, the reported figures are converted to USD as at the exchange rate on the last day of the reporting period. 2) All figures come from unaudited quarterly reports filed to
stockholders. Therefore, the quarterly totals may not equal the annual total. 3) Green indicates growth (in US Dollars) red indicates contracting sales. 4) If data is not available, it is
indicated by n/a. “0” indicates no sales for the period. 5) Top number represents global sales, numbers in parenthesis “(US66)” represent sales within the United States. If there are no
numbers in parenthesis, than only global data is provided.
GLOBAL REVENUE (US$ MIL)
Drug or Device
($mil)
107
Bayer
Egalet
Immunocore
Oxigene
Ablynx
Biogen
Eisai
INSYS Therapeutics
Pfizer
Acceleron
BioMarin
Eli Lilly
Ionis Pharma
Prothena
Alexion
Boehringer Ingelheim
Genentech
Jazz Pharma
Rigel
Alnylam
Boston Biomedical
Genmab
J&J Janssen
Roche
Ambit Biosciences
Bristol-Myers Squibb
Geron
MedImunne
Sanofi
Amgen
Celgene
Gilead Sciences
Merck Serono
Seattle Genetics
Ariad
Chiasma
GlaxoSmithKline
MSD
Spark
Astellas
Chugai
GW Pharmaceuticals
Millenium
Spectrum
Pharmaceuticals
AstraZeneca
CSL Behring
Helsinn
Molecular Partners
Syros
Basilea
CTI Biopharma
ImClone
Novartis
Takeda
Baxalta
Curis
Imcyte
Oryzon
Tesaro
Baxter
Daiichi Sankyo
Immatics
Otsuka
Triphase
COMPANIES FOLLOWED BY THIS REPORT
AbbVie
Total number of products covered: 108
108