PPMD is excited to learn that the U.S. Food and Drug Administration (FDA) has accepted and filed Sarepta Therapeutics’ efficacy supplement to the Biologics License Application (BLA) for ELEVIDYS.

The efficacy supplement aims to ​​expand the labeled indication for ELEVIDYS to state: “[ELEVIDYS is indicated for] the treatment of Duchenne muscular dystrophy (DMD) patients with a confirmed mutation in the DMD gene,” removing age and ambulation restrictions previously approved and filed with the FDA. Additionally, the FDA will be evaluating the efficacy supplement 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, 2024. The agency has confirmed they are not planning to hold an advisory committee meeting to discuss the supplement.

PPMD is pleased to learn this news and reinforce our shared commitment to advocate for a broader labeling of approved therapies to strive for access for all eligible individuals with Duchenne. PPMD, along with community partners, sent a letter to the FDA in December 2023 urging a timely and transparent review of EMBARK trial data that supports Sarepta’s efficacy supplement.

Read Sarepta’s press release here.