Sarepta announces FDA acceptance of efficacy supplement to Elevidys BLA

Sarepta announced the FDA has accepted and filed the company’s efficacy supplement to the biologics license application, or BLA, for Elevidys. The goals of the Efficacy Supplement are twofold: To expand the labeled indication for Elevidys as follows: “(ELEVIDYS is indicated for) the treatment of Duchenne muscular dystrophy, or DMD, patients with a confirmed mutation in the DMD gene.” To convert the Elevidys accelerated approval to a traditional approval. The FDA has granted the Efficacy Supplement a priority review with a review goal date of June 21. The agency has also confirmed they are not planning to hold an advisory committee meeting to discuss the supplement.