FDA Fast-Tracks Review Of Sarepta's Groundbreaking Muscular Dystrophy Treatment With $1B Market In Sight

Friday, the FDA accepted and filed Sarepta Therapeutics Inc’s SRPT efficacy supplement to the Biologics License Application (BLA) for Elevidys (delandistrogene moxeparvovec-rokl).

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 (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. The FDA also confirmed they are not planning to hold an advisory committee meeting to discuss the supplement.

“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,” said Doug Ingram, president and chief executive officer, Sarepta Therapeutics. 

As part of a collaboration agreement signed in 2019, Sarepta Therapeutics is working with Roche Holdings AG RHHBY. 

Sarepta is responsible for regulatory approval and commercializing Elevidys in the U.S. and manufacturing. 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.

Despite the need for flexibility in the data submission to the FDA, 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.