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DisEMBARK? FDA Questions Accelerated Approval Impact On Sarepta’s Fully Enrolled Clinical Trial

 
• By Sue Sutter

Advisory committee will consider how approval of SRP-9001 now might affect completion of the 52-week endpoint in EMBARK, a fully enrolled trial with two-thirds of the subjects coming from the US. Sarepta says all study subjects will have guaranteed access to the DMD gene therapy by end of September, making the risk of dropout ‘extremely low.’

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US FDA questions whether US study subjects will leave the EMBARK trial if SRP-9001 receives accelerated approval. • Source: Shutterstock

The potential impact of an accelerated approval for Sarepta Therapeutics, Inc.’s Duchenne muscular dystrophy gene therapy SRP-9001 (delandistrogene moxeparvovec) on completion of the randomized, Phase III EMBARK trial is among the key issues on which the US Food and Drug Administration seeks advisory committee input.

At a 12 May meeting, the Cellular, Tissue and Gene Therapies Advisory Committee will be asked to discuss the impact of accelerated approval on completion of the 52-week first part of the multinational study “without missing the collection of critical data,” the FDA’s briefing

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