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Wednesday, June 21 • 4:00pm - 5:15pm
#370: Lessons Learned from the Sarepta Exondys 51 Approval

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Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-17-531-L04-P; CME 1.25; IACET 1.25; RN 1.25

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 Objectives

Describe circumstances surrounding the development and ultimate approval of eteplirsen; Identify how to apply lessons learned to other drug development efforts.

Chair

Shamim Ruff, MSc

Speaker

A Challenging Path: Bridging Patient Needs With Regulatory Approval Requirements
Shamim Ruff, MSc

The Role of the Patient Perspective in the Approval of Exondys 51
Christine McSherry, RN

The Evolving Role of Orphan Drug and Subpart H Flexibility in Drug Approval
Alexander Varond, JD


Moderators
SR

Shamim Ruff

Senior Vice President of Regulatory Affairs and Quality, Sarepta Therapeutics

Speakers
avatar for Christine McSherry

Christine McSherry

Executive Director, Jett Foundation
Christine McSherry is the executive director of Jett Foundation, a non-profit dedicated to Duchenne muscular dystrophy, and the co-founder of Casimir Trials, a CRO that designs patient, caregiver, and observer reported outcomes that support regulatory approval and reimbursement. Christine... Read More →
avatar for Alexander Varond

Alexander Varond

Associate, Goodwin Procter LLP
Alexander J. Varond works on drug development and medical devices, advertising and promotion, and enforcement issues. He counsels clients on drug development matters such as clinical trial design, REMS, orphan drug designation, formal dispute resolution, and patent and exclusivity... Read More →

Wednesday June 21, 2017 4:00pm - 5:15pm CDT
S404bc McCormick Place 2301 South Martin Luther King Jr. Drive, Gate 4 Chicago, IL 60616
  05: Regulatory, Forum