The therapy will now carry a boxed warning for serious liver injury, liver failure and death<\/p>\n<\/h3>\n
By I. Edwards HealthDay Reporter<\/b>
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MONDAY, Nov. 17, 2025 (HealthDay News) \u2014 The U.S. Food and Drug Administration (FDA) is tightening restrictions on a gene therapy used to treat Duchenne muscular dystrophy after two teenagers died from liver failure linked to the medication.<\/p>\n
The FDA decision limits the use of Elevidys<\/a>, made by Sarepta Therapeutics<\/a>, to boys who are 4 years and older who can still walk, the agency said.<\/p>\n It will no longer be allowed for boys who have already lost mobility, something that typically happens around age 12 for patients with Duchenne.<\/p>\n Officials said a safety review confirmed that the two teens developed severe liver damage after receiving the infusion.<\/p>\n Both later died.<\/p>\n The FDA is also adding its strictest boxed warning to highlight the risks of serious liver injury, acute liver failure and death.<\/p>\n Sarepta, based in Cambridge, Massachusetts with offices worldwide, told regulators earlier this year that it had already stopped shipping the therapy to non-ambulatory patients.<\/p>\n The company also said Elevidys has been used in 1,100 patients worldwide.<\/p>\n These labeling changes “will ensure that families and health- care professionals have clear information,” to help guide decisions, Louise Rodino-Klapac<\/a>, Sarepta\u2019s president of research and development told The New York Times<\/em>.<\/p>\n Elevidys is a one-time infusion designed to slow Duchenne muscular dystrophy, a disease that causes muscles like the heart to weaken over time. Most patients are boys and many do not survive beyond age 30.<\/p>\n The drug label had previously warned about liver injury, but it did not mention liver failure or death, according to the agency.<\/p>\n Further concerns arose when Sarepta reported another liver-related death, this time in a 51-year-old man in an early trial for a different muscular dystrophy treatment.<\/p>\n Sarepta\u2019s muscular dystrophy drugs have long been controversial at the FDA. For years, agency leaders approved treatments over concerns from scientists about limited clinical evidence.<\/p>\n One of the critics of the approvals, Dr. Vinay Prasad<\/a>, a hematologist-oncologist at the University of California, San Francisco, joined the FDA in May 2025 to lead its gene therapy division.<\/p>\n Following pressure from political activists and outside groups who wanted approval of the drugs, Prasad briefly resigned, but he was later reinstated after agency officials intervened.<\/p>\n The FDA has told doctors they should monitor patients\u2019 liver function for at least three months after treatment.<\/p>\n Sarepta is also in discussions with the FDA after another recent study showed no clear benefit from non-gene therapy drugs aimed at treating Duchenne.<\/p>\n The company says COVID-related disruptions affected the results and still hopes to seek full approval.<\/p>\n More information<\/strong><\/p>\n The Muscular Dystrophy Association has more on Duchenne Muscular Dystrophy<\/a> (DMD).<\/p>\n