
WASHINGTON (AP) — The Food and Drug Administration said Friday it will add a new warning and other limitations to a gene therapy for Duchenne’s muscular dystrophy that's been linked to two patient deaths.
The infused therapy from Sarepta Therapeutics will carry a boxed warning — the most serious type — alerting doctors and patients to the risk of potentially fatal liver failure with the treatment, the FDA said in a release.
The one-time therapy, Elevidys, has been under FDA scrutiny since the company reported the first of two deaths of teenage boys in March. Following a second death reported in June, the FDA briefly called for halting all shipments of the drug. But the agency quickly reversed course after facing pushback from patient families and libertarian activists close to President Donald Trump.
Elevidys is the first U.S.-approved gene therapy for Duchenne’s muscular dystrophy, a fatal muscle-wasting disease that affects boys and young men.
In addition to the boxed warning, the FDA is also limiting the drug's approved use to patients who are 4 years old and up and can still walk. Previously the FDA had allowed the drug's use in immobile patients, who generally have more advanced disease.
New labeling will also recommend weekly liver function monitoring for the first three months of treatment, as well as other precautionary steps.
Elevidys is Sarepta's best-selling product and recent headwinds against the drug have weighed heavily on the company and its stock. In July, the Cambridge, Massachusetts-based company announced it would lay off 500 employees.
Sarepta Therapeutics Inc. shares rose 7.7% in trading after the FDA announcement, reflecting improved visibility for investors about the company's outlook.
___
The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute’s Department of Science Education and the Robert Wood Johnson Foundation. The AP is solely responsible for all content.
LATEST POSTS
- 1
FDA approves Wegovy pill for weight loss - 2
AfD in Brandenburg takes back suit against the intelligence service - 3
PHOTO ESSAY: Summer camp for kids with autoimmune diseases - 4
Flu activity rises sharply across US with 7.5 million cases, CDC data shows - 5
More than 800 flights canceled as FAA cuts traffic at 40 major airports. Here's what to know.
New trailer for 'Bridgerton' Season 4 teases Benedict's love story: Watch it here
Starship success, a private moon landing and more: The top 10 spaceflight stories of 2025
Figure out how to Arrange a Fair Settlement with the Assistance of a Fender bender Legal counselor
The Appearance of Experience: Embracing the Reduced Portage Horse
‘It’s Israeli policy’: Report reveals abuse of Palestinians in prisons
When Would it be a good idea for you to Look for Help from a Criminal Legal counselor?
The Best Internet based Retailers for Style and Frill
Czech Republic's new premier: No money for Ukraine
Dark matter may be made of pieces of giant, exotic objects — and astronomers think they know how to look for them












