Sarepta Therapeutics, Inc. today announced the submission of an efficacy supplement to the Biologics License Application (BLA) for ELEVIDYS to expand its labeled indication. The company also shared that it completed the Phase 3 EMBARK study postmarketing requirement (PMR) and submitted the PMR to the U.S. Food and Drug Administration (FDA) requesting conversion from accelerated approval to traditional approval.

Sarepta is requesting the label indication be expanded to “[ELEVIDYS is indicated for] the treatment of Duchenne muscular dystrophy patients with a confirmed mutation in the DMD gene,” which would remove age and ambulation restrictions previously approved and filed with the FDA. The efficacy supplement is supported by results from EMBARK, as well as data from the open label ENDEAVOR study. Sarepta has requested the FDA’s Priority Review.

PPMD is pleased to learn this news and reinforce our shared commitment to advocate for a broader labeling of approved therapies to ensure access for all individuals with Duchenne. PPMD, along with community partners, recently sent a letter to the FDA urging a timely and transparent review of EMBARK trial data. 

For additional details, you can read Sarepta’s press release here.