• Posted November 17, 2025

FDA Limits Duchenne Gene Therapy After Two Teens Die of Liver Failure

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.

The FDA decision limits the use of Elevidys, made by Sarepta Therapeutics, to boys who are 4 years and older who can still walk, the agency said.

It will no longer be allowed for boys who have already lost mobility, something that typically happens around age 12 for patients with Duchenne.

Officials said a safety review confirmed that the two teens developed severe liver damage after receiving the infusion.

Both later died.

The FDA is also adding its strictest boxed warning to highlight the risks of serious liver injury, acute liver failure and death.

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.

The company also said Elevidys has been used in 1,100 patients worldwide.

These labeling changes "will ensure that families and health-care professionals have clear information," to help guide decisions, Louise Rodino-Klapac, Sarepta’s president of research and development told The New York Times.

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.

The drug label had previously warned about liver injury, but it did not mention liver failure or death, according to the agency.

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.

Sarepta’s muscular dystrophy drugs have long been controversial at the FDA. For years, agency leaders approved treatments over concerns from scientists about limited clinical evidence.

One of the critics of the approvals, Dr. Vinay Prasad, a hematologist-oncologist at the University of California, San Francisco, joined the FDA in May 2025 to lead its gene therapy division.

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.

The FDA has told doctors they should monitor patients’ liver function for at least three months after treatment.

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.

The company says COVID-related disruptions affected the results and still hopes to seek full approval.

More information

The Muscular Dystrophy Association has more on Duchenne Muscular Dystrophy (DMD).

SOURCE: The New York Times, Nov. 14, 2025