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.
Elevidys is administered as a one-time infusion, to try to slow the progression of Duchenne muscular dystrophy, which causes muscles, including the heart, to deteriorate. Most patients are young men who typically do not survive beyond 30.
Sarepta’s muscular dystrophy treatments have been a frequent source of controversy for the F.D.A., especially as parents and advocates desperately seek options for the devastating disease. For years, regulators approved company medications over the objections of career scientists and despite clinical trials that failed to show a clear benefit to patients.
One of the chief critics of those approvals was Dr. Vinay Prasad, a professor at the University of California, San Francisco, who joined the F.D.A. in May 2025 to lead its gene therapy division and work as its chief medical officer.
By June 24, soon after a second teenager had died of liver failure after getting the medication, the agency announced a safety review.
The drug’s prescribing information had warned of the risk of serious liver injury, but it did not include warnings about liver failure or death, the agency said.
Less than two weeks later, on July 3, Sarepta said it notified the F.D.A. about the death of a 51-year-old man who was enrolled in an early clinical trial for medication to treat limb-girdle muscular dystrophy, which causes progressive weakness around the hips and shoulders. That death was also related to liver toxicity.
The company stunningly refused the F.D.A.’s demand to stop shipping the treatment, saying it would continue to provide the medication to young boys who could still walk.
Two days later, a campaign targeting Dr. Prasad escalated. Laura Loomer, a right-wing activist who is influential with President Trump, cited Dr. Prasad’s old social media posts, saying he was a liberal obstructing the president’s “deregulatory agenda” by denying approval for several medications. Rick Santorum, a former Republican senator from Pennsylvania who has ties to Sarepta, contacted the White House to raise concerns about Dr. Prasad.
That led to Dr. Prasad’s sudden resignation, although lobbying by top U.S. health officials reversed his departure, and he is back at the agency overseeing vaccines, cell and gene therapies and acting as the chief medical and scientific officer.
In a quarterly financial report released this month, Sarepta said it was in talks with the F.D.A. about the proposed warning and limits on the drug’s use. The F.D.A. said on Friday that it would also advise doctors to monitor the liver function of patients for at least three months after treatment.
The company issued another announcement this month, revealing that a study failed to show benefits of drugs that were not gene therapies but also aimed at treating Duchenne.
Despite the setback, Sarepta said it would still seek full F.D.A. approval for the drugs, citing problems with the study related to the Covid pandemic. When pandemic-era data was excluded, the company said, a “meaningful treatment effect is seen.”
Christina Jewett covers the Food and Drug Administration, which means keeping a close eye on drugs, medical devices, food safety and tobacco policy.
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