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.