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