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Lessons Learned from the Sarepta Exondys 51 Approval
Session Chair(s)
Shamim Ruff, MSc
Chief Regulatory Officer and Senior Vice President Quality
Stoke Therapeutics, United States
FDA’s approval amidst controversy of Sarepta’s Duchenne muscular dystrophy drug Exondys 51 (eteplirsen) was unprecedented in terms of the level of flexibility used to support accelerated approval in a rare disease. This session explores the unique conditions surrounding the drug’s patient-focused drug development program and its approval.
Learning Objective : Describe circumstances surrounding the development and ultimate approval of eteplirsen; Identify how to apply lessons learned to other drug development efforts.
Speaker(s)
A Challenging Path: Bridging Patient Needs With Regulatory Approval Requirements
Shamim Ruff, MSc
Stoke Therapeutics, United States
Chief Regulatory Officer and Senior Vice President Quality
The Role of the Patient Perspective in the Approval of Exondys 51
Christine McSherry, BSN, RN
Casimir LLC, United States
Chief Executive Officer
The Evolving Role of Orphan Drug and Subpart H Flexibility in Drug Approval
Alexander Varond, JD
Goodwin Procter LLP, United States
Partner
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