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Sarepta’s DMD Gene Therapy Adcomm Likely To Focus On Dystrophin As A Surrogate Endpoint

 
• By Sue Sutter and Derrick Gingery

Cellular, Tissue and Gene Therapies Advisory Committee will weigh whether the increase in dystrophin production seen with SRP-9001 is reasonably likely to predict clinical benefit in Duchenne muscular dystrophy, Sarepta says; agency had previously told the company a panel meeting would not be needed, but it reversed course late in the review.

U turn sign
FDA reversed course and decided a panel review was needed for Sarepta's gene therapy. • Source: Shutterstock

A US Food and Drug Administration advisory committee review of Sarepta Therapeutics, Inc.’s SRP-9001 (delandistrogene moxeparvovec) is expected to focus on whether the increase in dystrophin production seen with the gene therapy is reasonably likely to predict clinical benefit in Duchenne muscular dystrophy, the company said.

The decision to convene the public meeting also appears to reflect the agency’s interest in exploring the use of surrogate endpoints, biomarkers and

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