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.