(Reuters) -Sarepta Therapeutics said on Monday it will pause all shipments of Elevidys in the United States after two teenage boys with a rare condition called Duchenne muscular dystrophy, who had received the company’s gene therapy, died of liver failure this year.
The U.S. Food and Drug Administration on Friday asked Sarepta to voluntarily halt shipments of its gene therapy.
The company responded that it would continue providing the treatment to ambulatory patients — while maintaining a halt it implemented June 15 for non-ambulatory patients.
Sarepta’s shares are down 8.6% to $12.17 after the bell. The stock plummeted on Friday after it disclosed the dead of a third patient who had received an experimental gene therapy, deepening investor concerns over the company’s treatments.
While the 51-year-old man with limb girdle muscular dystrophy who died most recently was not taking Elevidys, the experimental therapy he took was based on similar gene technology, the FDA said.
The FDA put Sarepta’s clinical trials for limb girdle muscular dystrophy on hold due to safety concerns.
All shipments of Elevidys for Duchenne muscular dystrophy will be temporarily paused by close of business on Tuesday, Sarepta said Monday.
The company said that the pause will allow it the “time to respond to any requests for information and allow Sarepta and FDA to complete the Elevidys safety labeling supplement process.”
Sarepta said it anticipates a review and dialogue with the FDA.
The FDA did not immediately respond to a Reuters request for comment.
After the first two deaths, Sarepta in June suspended the use of Elevidys for patients whose disease had progressed to the point where they need a wheelchair.
Elevidys has full FDA approval for children with DMD who are still able to walk, and conditional approval for non-ambulatory patients.
Last week, Sarepta said it was adding a warning on Elevidys’ label for the risk of acute liver injury and liver failure in DMD patients who can walk. It also announced 500 job cuts and halted development of several gene therapies for limb girdle muscular dystrophy.
Separately, the Children’s Hospital Los Angeles, said on Monday it has paused usage of Elevidys in all patients with muscular dystrophy.
(Reporting by Rishabh Jaiswal and Mrinmay Dey in Bengaluru; Editing by Alan Barona)
Comments