ANNUAL RARE DISEASE SCIENTIFIC WORKSHOP
Improving the Clinical Development Process
Workshop 6: Rationalizing Safety Testing to Enable Clinical Studies &
Approval in the US for Rare Disease Treatments
Hosted By:
EveryLife Foundation for Rare Diseases
Sofitel Hotel Washington, D.C. Lafayette Square
September 16, 2014
AGENDA
Workshop Overall Goal: The goal of workshop 6 is to continue the discussions from previous workshops
concerning issues most relevant to the development process of innovative medicines to treat rare diseases. This
workshop will focus on the regulatory aspects of safety requirements during the drug development process, and
current trends, issues, and perspectives on safety and toxicology. The goal of the workshop is to ensure all
stakeholders have a chance to discuss and comment on the important scientific elements regarding the toxicity
and safety regulatory requirements.
Morning Session
8:30 – 9:00 AM
REGISTRATION AND BREAKFAST
Introduction
9:00 AM
Welcome
Emil Kakkis, MD, PhD
President & Founder, EveryLife Foundation for Rare Diseases
President &CEO, Ultragenyx
Session A: Safety & Toxicology Perspectives from Health Authority Agencies
9:30 AM
9:50 AM
10:00 AM
10:20 AM
10:30 AM
10:50 AM
IND-Enabling Safety Studies in Rare Diseases
Timothy McGovern, PhD
ODE Associate Director for Pharmacology/Toxicology
Office of New Drugs, CDER, FDA
Questions & Discussion
An EU Regulatory View on Non-Clinical Support for First in Human Trials in Rare Disease
David R. Jones
Expert Scientific Assessor, Licensing Division, MHRA
Questions & Discussion
Safety Assessment for Rare and Orphan Diseases in Clinical Trials
Andrew Mulberg, MD, FAAP, CPI
Division Deputy Director, Division of Gastroenterology and Inborn Error Products
ODE III,CDER, FDA
Questions & Discussion
1
September 16, 2014
EveryLife Foundation Annual Scientific Workshop
Session A (cont)
11:00 – 11:15 AM
11:15 AM
11:35 AM
11:45 AM
BREAK
Immune Tolerance Induction in Enzyme Replacement Therapy for Lysosomal Storage
Diseases: Optimizing Therapeutic Outcome with Preventive Strategies
Amy S. Rosenberg, MD
Director, Division of Therapeutic Proteins, CDER, FDA
Questions & Discussion
12:05 PM
An Introduction to Biosafe and Overview of Activities in the Rare Disease Area
Timothy MacLachlan, PhD, DABT
Chair, Biosafe Leadership Committee
Executive Director, Preclinical Safety, Novartis
Questions & Discussion
12:15 – 1:00 PM
LUNCH
Afternoon Session
Session B: Case Studies in PreClinical Safety and Toxicology
1:00 PM
1:20 PM
1:30 PM
Preclinical Experience with rhASM for Neimann-Pick Disease
Gerald Cox, MD
Vice President, Clinical Development, Rare Disease Group, Genzyme
Questions & Discussion
1:50 PM
Nonclinical Development of Enzyme Replacement Therapies for Severely Affected
Patients of Orphan Diseases: Assessment of Animal Model and Normal Animal
Toxicology Data
Charles O’Neill, PhD
Vice President, Pharmacological Sciences, Biomarin
Questions & Discussion
2:00 – 2:15 PM
BREAK
Session C: Safety and Toxicology Issues for Genetic Platform Technologies
2:15 PM
2:35 PM
OSWG’s Role in Shaping Nonclinical Regulatory Strategy in the Development of
Oligonucleotide Therapeutics
David Schubert, PhD
Vice President, Regulatory & Quality, Stealth Peptides
Questions & Discussion
2
September 16, 2014
EveryLife Foundation Annual Scientific Workshop
Session C (cont)
2:45 PM
3:05 PM
3:15 PM
3:35 PM
Preclinical Safety Assessment of Phosphorodiamidate Morpholino Oligomers (PMO)
Peter Sazani, PhD
Executive Director, Medical Affairs, Sarepta
Questions & Discussion
Gene Therapy Vector Platforms share common safety and efficacy issues when deployed
across groups of similar orphan diseases
James Wilson, MD, PhD
Professor of Pathology and Laboratory Medicine
Research Director, Center for Orphan Disease Research and Therapy
Director, Gene Therapy Program, University of Pennsylvania
Questions & Discussion
3:45 PM
Summary and Final Discussion, Future Directions and Closing Remarks
Emil Kakkis, MD, PhD
President & Founder, EveryLife Foundation for Rare Diseases
President & CEO, Ultragenyx
4:15 PM
End of Workshop
Thank you to our Scientific Workshop Sponsors:
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