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Elevidys’ Age-Restricted Indication Not Negotiable, CBER’s Peter Marks Told Sarepta

 
• By Sue Sutter

FDA team held several internal meetings on the gene therapy after the 12 May advisory committee, culminating in a teleconference with Sarepta in which the CBER director outlined his accelerated approval decision and reiterated recommendations to modify the ongoing EMBARK trial to better ensure confirmation of benefit. Pink Sheet's Drug Review Profile dives into the story behind the Elevidys review.

Drug Review Profile: Elevidys
CBER Director Peter Marks told Sarepta his decision on an age-limited indication was not up for negotiation. • Source: Shutterstock

FDA Center for Biologics Evaluation and Research Director Peter Marks’ conclusion that Sarepta Therapeutics, Inc.’s gene therapy Elevidys (delandistrogene moxeparvovec-rokl) should receive accelerated approval for an age-restricted indication amounted to a take-it-or-leave-it offer for the sponsor, not subject to negotiation.

In sharing his decision with Sarepta, Marks also made clear agency concerns that the ongoing EMBARK confirmatory trial was designed in such a way that it may not be able to confirm clinical benefit in the 4-5 year-old age group of Duchenne muscular dystrophy patients covered by the accelerated

Key Takeaways

  • After the 12 May advisory committee meeting, FDA’s clinical, clinical pharmacology and biostatistics review teams continued to recommend a complete response letter for Elevidys.

  • CBER Director Peter Marks made the decision to move forward with accelerated approval for an age-restricted indication, telling Sarepta the FDA’s position “is not up for negotiation

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