FDA official announcements or press releases

FDA Expands Approval of Sarepta's Elevidys for Duchenne Muscular Dystrophy, Shares Surge 36%

Jun 21, 2024, 05:39 AM

The U.S. Food and Drug Administration (FDA) has expanded the approval of Sarepta Therapeutics' $3.2mn gene therapy, Elevidys, for the treatment of Duchenne muscular dystrophy (DMD) as of June 20. The approval now includes both ambulatory and non-ambulatory patients aged four and above with a confirmed mutation in the DMD gene. The decision converts the indication for ambulatory patients to full approval, while the non-ambulatory indication is under accelerated approval. This expansion significantly broadens the patient population eligible for Elevidys, which previously had a narrower authorization. Sarepta's stock surged approximately 36% in premarket trading following the announcement, reflecting investor optimism about the expanded market for the therapy. The approval process was marked by controversy as top FDA official Peter Marks overruled recommendations from three review teams and two top lieutenants who favored an additional confirmatory study. Despite the controversy, the expanded approval is seen as a major milestone for Sarepta and the DMD community.