
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
Flight cancellations: Full list of 40 airports hit by FAA cuts amid government shutdown - 2
New portrait of the oldest-known supernova | Space photo of the day for March 27, 2026 - 3
Step by step instructions to Integrate Lab Precious stones into Special Adornments Pieces - 4
Defense Minister Katz moves to extend IDF service to 36 months - 5
Which Instax Camera Would it be a good idea for you to Purchase?
World’s tallest bridge and biggest museum named ‘greatest places of 2026’
Holiday spots Well known With Americans In 2024
Iran war drives global fertilizer prices up, raising food cost fears
We tasted one of the 10,000 Hershey's Dubai chocolate bars being resold on eBay. Is it worth the hype?
Couch Styles of 2024: What's Moving
Europe's powerful Ariane 6 rocket launches Sentinel-1D Earth-observation satellite to orbit (video)
Vote In favor of Your Favored Occasion Arranging Administration
Doctored NXT Summit footage falsely portrays Modi as declaring war on Iran and Pakistan
6 Useful Home Espresso Machines












