
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
Do you lean your seat back on the plane? These travel pros — and real-life couple — won't do it. - 2
She was moments away from giving birth. The hospital discharged her - 3
Europe’s EV Boom Was Real in 2025. The Real Fight Starts In 2026 - 4
Four countries to boycott Eurovision 2026 over Israel’s inclusion - 5
Authorities arrest 7 bodyguards in connection with a Mexican mayor's assassination
Remote Work Survival manual: Helping Efficiency at Home
Remote Headphones: Improve Your Sound Insight
Jury says Johnson & Johnson owes $40 million to 2 cancer patients who used talcum powders
Step by step instructions to Think about Disc Rates Across Various Banks
In these U.S. groups, deaths now exceed births. What’s happening?
Doggie diversity in size and shape began at least 11,000 years ago
Israel reports killing another senior Iranian oil official
This Flashy Old-School Design Trend From Italy Still Has A Place In Modern Kitchens
Figure out How to Upgrade Your Gold Speculation Portfolio: Vital Bits of knowledge and Strategies













