Sarepta’s DMD AAV gene therapy receives updated label with safety warnings

WASHINGTON D.C. – November 14, 2025 — The U.S. Food and Drug Administration (FDA) has officially restricted the label for Sarepta Therapeutics’ AAV gene therapy, Elevidys (delandistrogene moxeparvovec-rokl), for Duchenne muscular dystrophy (DMD), following two reported deaths from acute liver failure in non-ambulatory patients.

The FDA’s action limits the one-time AAV gene therapy’s indication to ambulatory DMD patients who are at least 4 years of age. Crucially, the non-ambulatory patient population—those no longer able to walk independently—can no longer receive the treatment.

New Black Box Warning Added to AAV Therapy

The official label restriction comes five months after Sarepta and its ex-U.S. partner Roche voluntarily suspended giving the AAV therapy to non-ambulatory patients. The decision followed the report of a second recipient who died after developing acute liver failure, a known risk associated with AAV vector-based gene therapies.

The updated label now includes a new Boxed Warning—the FDA’s most serious safety warning—describing the potentially deadly risks of serious liver injury and acute liver failure associated with the AAV treatment.

AAV Treatment Requires Enhanced Caution

In a further tweak to the AAV therapy’s label, the FDA has added a new “limitation of use.” Elevidys is now “not recommended in patients with preexisting liver impairment, recent vaccinations, or recent/active infections.” According to Sarepta, “recent” is defined as within four weeks. The prior label had only advised that treatment be “carefully considered” in patients with preexisting liver impairment or chronic hepatic viral infection.

Despite the restrictions and safety updates—which were anticipated following the company’s third-quarter earnings report—Sarepta’s stock price was up about 7% on Friday as of publication time, reflecting market relief that the AAV therapy remains available for the ambulatory population.

Elevidys is an AAV vector-based gene therapy that delivers a transgene encoding a micro-dystrophin protein, an engineered and shortened version of the full dystrophin protein that is absent in DMD patients. The FDA has also asked Sarepta to conduct a postmarketing observational study on approximately 200 individuals to further assess the risk of serious liver injury.