Sarepta Therapeutics Inc’s SRPT efficacy supplement to the Biologics License Application (BLA) for Elevidys was accepted and filed by the FDA on Friday.

The purpose of the Efficacy Supplement is twofold:

• To expand the labeled indication for Elevidys for the treatment of Duchenne muscular dystrophy (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, 2024. Furthermore, the FDA confirmed they are not planning to hold an advisory committee meeting to discuss the supplement.

According to Doug Ingram, president and chief executive officer of Sarepta Therapeutics, “We are pleased to announce that FDA has accepted and filed Sarepta’s Efficacy Supplement to evaluate broadening the approved indication of Elevidys by removing age and ambulation restrictions and converting the approval from accelerated to traditional.”

Sarepta Therapeutics is working with Roche Holdings AG through a collaboration agreement signed in 2019. Sarepta is responsible for regulatory approval and commercializing Elevidys in the U.S. and manufacturing, while Roche is responsible for regulatory approvals and bringing Elevidys to patients across the rest of the world.

Analysts at William Blair write in summary that the accumulated data strongly indicates the effectiveness of Elevidys for its current approved age range and suggests potential benefits for older boys. However, extending the label to non-ambulatory patients might face challenges, and it wouldn’t be unexpected if these patients are excluded from the expanded approval.

The approval of Elevidys is seen as a transformative milestone for Duchenne patients. The broader application, especially with the inclusion of older age groups, could represent a significant yearly opportunity amounting to $1 billion for Sarepta.

Price Action: SRPT shares are up 9.7% at $137.09 on the last check Friday.

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Image created using artificial intelligence via MidJourney.