The U.S. Food and Drug Administration is tightening restrictions on a gene therapy used to treat Duchenne muscular dystrophy after two teenagers died of liver failure. File Photo by Jim Lo Scalzo/EPA
The U.S. Food and Drug Administration is tightening restrictions on a gene therapy used to treat Duchenne muscular dystrophy after two teenagers died from liver failure linked to the medication.
The FDA decision limits the use of Elevidys, made by Sarepta Therapeutics, to boys who are 4 years and older who can still walk, the agency said.
It will no longer be allowed for boys who have already lost mobility, something that typically happens around age 12 for patients with Duchenne.
Officials said a safety review confirmed that the two teens developed severe liver damage after receiving the infusion.
