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Why Sarepta’s Gene Therapy Trial Enrollment Has Been More Successful Than Its Other DMD Therapies

 
• By Derrick Gingery

The EMBARK trial’s availability to more patients likely contributed to the much quicker enrollment than confirmatory trials for Exondys 51, Vyondys 53 and Amondys 45, which also saw enrollment targets and eligibility ages change over the years.

Duchenne muscular dystrophy
DMD advocates hope Sarepta's gene therapy candidate can push development in the disease forward. • Source: Shutterstock

The clinical trial testing Sarepta Therapeutics, Inc.’s gene therapy candidate in Duchenne muscular dystrophy enrolled remarkably faster than the company’s studies looking at three drugs that treat the rare disease, a likely function of its potential and a concern for the others’ futures.

SRP-9001 (delandistrogene moxeparvovec) is under priority review at the US Food and Drug Administration for accelerated approval. The EMBARK trial was first listed as recruiting on clinicaltrials.gov in October 2021,...

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