In a remarkable public dispute between drugmaker and regulator, the biotech company Sarepta Therapeutics is defying the Food and Drug Administration’s request that it halt distribution of its treatment for a deadly muscle-wasting disease.
In a news release on Friday evening, the agency said that it requested that the company voluntarily stop all shipments of the therapy, known as Elevidys, citing the deaths of three patients from liver failure who had taken the product or a similar therapy.
In its own news release later on Friday evening, Sarepta, which is based in Cambridge, Mass., said that it would continue to ship the treatment for patients who do not use wheelchairs. The company said its analysis showed no new safety problems in those patients and that it was committed to patient safety.
Dr. Marty Makary, the F.D.A. commissioner, said in the agency’s statement that its request to Sarepta demonstrated that the F.D.A. “takes swift action when patient safety is at risk.”
“We believe in access to drugs for unmet medical needs but are not afraid to take immediate action when a serious safety signal emerges,” he said.
In the past, the F.D.A. has sometimes asked companies to pause distribution of a drug until a new problem is better understood and mitigated. However, it can also press its case, and begin a process to revoke the drug’s license, which would begin with a formal notification and opportunity to respond and participate in a public hearing.
Typically, companies have quickly complied with F.D.A. requests to take a treatment off the market. In rare cases, drugmakers have gone through the F.D.A.’s license revocation process, which can be lengthy, as with a drug deemed ineffective to prevent preterm labor in 2023 and one for breast cancer in 2011 that it also judged ineffective and that had serious side effects.
Questions about the fate of Elevidys escalated over the course of this week, fueling an ongoing debate over whether the F.D.A. has grown too lenient in approving drugs that prove insufficiently safe or effective. New officials in the Trump administration 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 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, Sarepta 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. Then as now, the company said it planned to keep distributing the product for patients who could still walk.
The company said on Wednesday that it would add a “boxed warning” to Elevidys, the strongest admonition under the F.D.A. about a side effect. The next day, Sarepta confirmed news reports that a third patient, a 51-year-old man in a clinical trial, had died from liver failure while taking an experimental therapy that Sarepta had been developing, which uses a technology similar to that of Elevidys.
On Friday evening, the company said it initially reported the man’s death to the F.D.A. on June 20. The F.D.A. said in its statement on Friday that it was prohibiting the company from proceeding with studies of that technology.
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 F.D.A.’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 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 saying that “you could replace Peter Marks with a bobblehead doll that just stamps approval.”
In an interview with The New York Times earlier this month, 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.
In another interview on Friday evening, Dr. Marks said that he supported the decision to step back and re-evaluate the safety of the medication.
“This is a different finding than what we originally made the benefit-risk determination on,” he said. “So I don’t disagree with a statement that maybe it would be good to take a breather and look at what’s going on here, particularly given the potential implications for the larger field of gene therapy.”
On Monday, Sarepta filed regulatory paperwork disclosing promotions and raises for several of its top executives.
On Wednesday, Sarepta 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. However, the company did not disclose the third patient’s death, which became public the day after.
The company’s stock price fell 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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