The Food and Drug Administration will request that the biotech company Sarepta Therapeutics halt all shipments of its treatment for a deadly muscle-wasting disease after three patients died from liver failure after taking the product or a similar therapy.
The troubled treatment, known as Elevidys, has fueled an ongoing debate over whether the F.D.A. has grown too lenient in approving drugs that are insufficiently safe or effective. New officials in the Trump administration, however, have rejected several new drug applications and narrowed the use of Covid vaccines over concerns about “unknown” side effects.
Elevidys, a gene therapy, is administered as a one-time intravenous infusion. It is intended to slow the progression of Duchenne muscular dystrophy, which causes muscles to deteriorate. The disease often kills patients, typically young men, before their 30th birthday.
Elevidys, first approved in 2023, is one of a handful of treatments for the disease that arrived in the past decade, with the help of fierce advocacy from patients and their families. But the approval of Elevidys was controversial because of limited evidence that its benefits outweighed its risks.
In recent months, two teenage boys who had received Elevidys died of liver failure, deepening critics’ concerns about the drug and prompting speculation that the F.D.A. would take strict action. In late June, the F.D.A. announced an investigation into the deaths. Liver injuries were among the side effects seen in clinical trials of the treatment.
On Tuesday, the company said it would halt shipments for patients with more advanced disease who use wheelchairs, a group that includes the teenagers who died and most Duchenne patients over age 12. At the time, the company planned to keep distributing the product for patients who could still walk.
A spokeswoman for Sarepta did not respond to a request for comment on Friday afternoon regarding whether it would now stop distributing the treatment for all patients, as the F.D.A. plans to request.
The company said on Wednesday that it would add a boxed warning to Elevidys. Also that day, Sarepta announced that a third patient, a 51-year-old man in a clinical trial, had died from liver failure while taking an experimental therapy Sarepta had been developing that used a technology similar to Elevidys’s.
The F.D.A. first approved the treatment under its accelerated approval program for young Duchenne patients who use wheelchairs. That pathway is for drugs that have had a successful study showing changes in the body but have not been proved to extend survival or markedly improve quality of life.
Dr. Peter Marks, who at the time was director of the agency’s vaccine and gene therapy division, pushed the approval through, overruling staff scientists who opposed the move.
The treatment had not been rigorously shown to improve patients’ motor abilities, such as standing, walking and rising. But it had been shown in clinical trials to increase production of a key protein, a signal that the company said was predictive of meaningful benefits for patients. Some patient groups applauded Dr. Marks’s decisions, saying they were willing to take a risk with such a devastating disease.
Four months after the initial approval, a late-stage clinical trial of 125 boys with Duchenne failed to meet its primary goal of improving patients’ motor abilities. Nonetheless, the next year, the F.D.A. expanded the approval to include older patients and those who are able to walk.
Dr. Vinay Prasad, who replaced Dr. Marks as director of the F.D.A.’s gene therapy division, has long been a sharp critic of the agency’s decisions to approve the treatment. Before Dr. Prasad joined the F.D.A., he cited the decisions in a social media post noting that “you could replace Peter Marks with a bobblehead doll that just stamps approval.”
In a recent interview with The New York Times, Dr. Marks defended the decision to approve Elevidys, saying that he felt “terribly” about the teenagers’ deaths. “There’s always benefit and risk in everything we do, and we take some risk, and hopefully we learn from it,” he said.
On Thursday, Sarepta, which is based in Cambridge, Mass., laid off about 500 employees, more than one-third of its work force. The company said it would pause most of its work on gene therapies, describing this as a financially motivated decision, but did not disclose the third patient death, which became public the day after.
On Monday, Sarepta filed regulatory paperwork disclosing promotions and raises for several of its top executives. The company’s stock price was down 36 percent on Friday.
Rebecca Robbins is a Times reporter covering the pharmaceutical industry. She has been reporting on health and medicine since 2015.
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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