sBLA for Elevidys Conversion to Traditional Approval

FDA has accepted for priority review a Sarepta Therapeutics supplemental BLA for Elevidys (delandistrogene moxeparvovec-rokl) to convert the earlier accelerated approval to a traditional approval, and to expand the labeled indication for treating Duchenne muscular dystrophy (DMD) patients with a confirmed mutation in the DMD gene. The agency has set a user fee review action target date of 6/21.

Elevidys is a single-dose, adeno-associated virus-based gene transfer therapy for intravenous infusion designed to address the underlying DMD genetic cause. The therapy has been evaluated in four clinical studies: SRP-9001-101, SRP-9001-102, SRP-9001-103 (ENDEAVOR) and SRP-9001-301 (EMBARK), according to the company. “Accelerated approval of Elevidys was primarily based on data from SRP-9001-102 and SRP-9001-103,” it says, adding that the EMBARK study served as the postmarketing confirmatory trial.