FDA to ask Sarepta to stop shipping Duchenne gene therapy
The Food and Drug Administration will ask Sarepta Therapeutics to halt all shipments of its marketed gene therapy for Duchenne muscular dystrophy, a source familiar with the matter confirmed to BioPharma Dive.
Sarepta last month paused shipments for certain older Duchenne patients following the death from acute liver failure of a second teenager treated with the therapy, called Elevidys. The FDA subsequently began a formal investigation of Elevidys’ liver risks.
Reuters first reported news of the FDA’s request. FDA Commissioner Martin Makary told Bloomberg in an interview Friday that his agency is weighing whether to withdraw Elevidys from the market entirely.
The news comes on the heels of Sarepta reporting this week the death of a 51-year-old man who received an experimental gene therapy it’s developing for another kind of muscular dystrophy known as limb-girdle. The man died of complications from acute liver failure in June.
While the two treatments are constructed differently, they both use the same kind of engineered virus to deliver a replacement gene to the body’s muscles.
Editor’s note: This is breaking news and this story will be updated.
