Sarepta stock rises after FDA adds safety warning but maintains approval

Investing.com -- Sarepta Therapeutics (NASDAQ:SRPT) stock rose 7% after the U.S. Food and Drug Administration approved new labeling for its gene therapy Elevidys that includes a Boxed Warning and limits its use to ambulatory patients four years and older with Duchenne muscular dystrophy (DMD).

The FDA’s decision follows reports of fatal acute liver failure in non-ambulatory patients treated with the therapy. Despite the safety concerns, investors appeared relieved that the agency maintained approval for Elevidys in ambulatory patients rather than implementing a more severe regulatory action.

The revised labeling includes the addition of the FDA’s most prominent safety warning and removes the indication for non-ambulatory DMD patients. This follows two reports of fatal acute liver failure in non-ambulatory pediatric males and an additional serious non-fatal case of acute liver injury after receiving Elevidys.

The updated safety information requires weekly liver function tests for at least three months after treatment and recommends patients remain near appropriate medical facilities for at least two months post-infusion. The FDA is also requiring Sarepta to conduct a postmarketing observational study of approximately 200 DMD patients to further assess the risk of serious liver injury.

Elevidys is an AAVrh74 adeno-associated virus vector-based gene therapy that was previously approved for a broader range of DMD patients. The manufacturer had voluntarily paused distribution for non-ambulatory patients in June 2025 following the initial safety reports.

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