CSL Behring Submits Marketing Authorization Application to
the European Medicines Agency for rVIII-SingleChain for
Patients with Hemophilia A
rVIII-SingleChain demonstrates CSL Behring’s promise to develop and deliver
innovative biotherapies that improve the well-being of patients with serious medical
conditions
MARBURG, Germany – December 22, 2015 — Global biotherapeutics leader CSL
Behring announced today that the company has submitted its Marketing Authorization
Application (MAA) to the European Medicines Agency (EMA) for its novel
investigational recombinant factor VIII single-chain (rVIII-SingleChain) for the treatment
of hemophilia A. In the pivotal clinical trial, rVIII-SingleChain met all primary endpoints.
Hemophilia A is a congenital bleeding disorder characterized by deficient or defective
factor VIII; nearly all affected patients are male. People with hemophilia A may
experience prolonged or spontaneous bleeding, especially into the muscles, joints, or
internal organs. According to the World Federation of Hemophilia, about 1 in 10,000
people are born with hemophilia, the majority of whom have hemophilia A.
“Our focused research and development and strong relationship with the bleeding
disorders community led us to seek further advancements in the care and treatment of
hemophilia,” said Dr. Andrew Cuthbertson, Chief Scientific Officer and Director of R&D,
CSL Limited. “Today, we have the only recombinant single-chain factor VIII product in
late-stage development for the management of hemophilia A. We look forward to, upon
approval, providing this innovative specialty biotherapy to patients in the EU and
European Economic Area.”
The MAA is based on the AFFINITY clinical development program, which includes a
phase I/III open-label, multi-center trial examining safety and efficacy. The
pharmacokinetics of rVIII-SingleChain compared with recombinant human
Page 1 of 3
antihemophilic factor VIII (octocog alfa) was also studied. Study design details for rVIIISingleChain are available at clinicaltrials.gov (NCT01486927).
Results from the phase I/III study presented earlier this year at the International Society
on Thrombosis and Haemostasis (ISTH) congress in Toronto showed that patients
treating prophylactically had a median annualized bleeding rate (ABR) of 1.14 and a
median annualized spontaneous bleeding rate (AsBR) of 0.00. The data also showed
that, of 848 bleeds treated in the study, 94 percent were controlled with no more than
two infusions of rVIII-SingleChain, with 81 percent controlled by one infusion.
Moreover, hemostatic control of a bleeding event treated with rVIII-SingleChain was
assessed by the investigator as excellent or good 94 percent of the time (835 assessed
bleeding events).
The results presented at ISTH included data on more than 14,000 exposure days in
146 patients on prophylaxis and 27 patients treating on demand for a bleeding event.
In total, 120 patients were treated for more than 50 days of exposure; 52 had more
than 100 days of exposure. In the prophylaxis group, 32 percent of patients were dosed
twice weekly and 54 percent received treatment three times per week; the regimen was
determined by the investigator. The most common adverse events were nasopharyngitis, arthralgia, and headache. No inhibitors were reported.
About rVIII-SingleChain
Specifically designed for greater molecular stability, rVIII-SingleChain is the first and
only recombinant single-chain factor VIII (FVIII) product in late-stage development for
the treatment of hemophilia A. rVIII-SingleChain has a strong affinity for von Willebrand
factor, leading to greater stability and integrity of factor VIII in circulation. The stability
of rVIII-SingleChain leads to a longer-lasting therapeutic effect with reduced dosing
frequency. In July 2015, the U.S. Food and Drug Administration accepted for review
CSL Behring’s Biologics License Application for rVIII-SingleChain.
For more information about CSL Behring’s recombinant products in development to
treat hemophilia, visit http://www.cslbehring.com/products/bleeding-disorders/novelrecombinant-hemophilia-treatments.
Page 2 of 3
About CSL
CSL (ASX:CSL) is a leading global biotherapeutics company with a dynamic portfolio of
life-saving innovations, including those that treat hemophilia and immune deficiencies,
as well as vaccines to prevent influenza. Since our start in 1916, we have been driven
by our promise to save lives using the latest technologies. Today, CSL — including our
two businesses CSL Behring and Seqirus — operates in over 30 countries with more
than 14,000 employees. Our unique combination of commercial strength, R&D focus
and operational excellence enables us to identify, develop and deliver innovations so
our patients can live life to the fullest.
For more information about CSL Behring visit www.CSLBehring.se (Nordic Region),
www.CSLBehring.com or follow us at www.Twitter.com/CSLBehring.
###
Media:
Greg Healy
CSL Behring
Office: 610-878-4841
Mobile: 610-906-4564
Email: Greg.Healy@cslbehring.com
Contacts Nordic Region (from January 7th):
Stefan Grass
Medical Affairs Manager
CSL Behring Nordic Region
Office: 08- 544 96 685
Mobile: 070- 725 24 48
Email: stefan.grass@cslbehring.com
Tove Forsberg
Product Manager Coagulation & Critical Care
CSL Behring Nordic Region
Office: 08- 544 96 675
Mobile: 070- 518 45 62
Email: tove.forsberg@cslbehring.com
Page 3 of 3