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A gene therapy for Duchenne muscular dystrophy caused complications to the liver, prompting a review of its use for younger patients.
Nov. 14, 2025Updated 4:03 p.m. ET
The Food and Drug Administration said on Friday that it would narrow approval of a gene therapy treatment for young men with a muscle-wasting disease.
The agency’s decision formalized an agreement reached with the drugmaker Sarepta in July to stop shipping it to certain patients after two teenagers using the medication died from liver failure, a known complication.
The agency said it was limiting the approved use of Elevidys after a safety review confirmed that the teenagers suffered liver damage that led to their hospitalizations and deaths.
The F.D.A. said it had decided to restrict the therapy to boys 4 years and older who were still able to walk. It would no longer be authorized for boys who have lost that mobility, which tends to happen around the age of 12 for those with the disease.
The agency is also adding a boxed warning — its strictest — to the therapy label, emphasizing the risks of serious liver injury, acute liver failure and death.
The gene therapy has been central to the business model of Sarepta Therapeutics, a publicly traded company valued at about $2 billion. The company said in a financial filing that it stopped shipping the drug to non-ambulatory patients in June.
