
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
Exploring ways to reduce the impact of space junk on Earth - 2
Vote in favor of the bloom plan that adds a bit of excellence to your life! - 3
What did the gov’t approve for Israel’s 2026 state budget? - 4
Figure out How to Reveal Stowed away Open Record Rewards - 5
When faith comes under fire: How Iran’s repression of religious minorities has increased
Most loved Amusement Park for Small children: Which One Do You Suggest?
‘The White Lotus’ sparked online interest in risky anxiety pills, study says
Nikki Glaser returns as host of the 2026 Golden Globes: Everything the comedian has said about the upcoming awards show
Cuba fights to contain spread of mosquito-borne chikungunya virus
The most effective method to Stay away from Normal Traps While Recruiting a Material Organization
Vaccine makers raise concerns over US panel's shift away from hepatitis B shots for newborns
Lahav 433 head Asst.-Ch. Meni Benjamin named as police officer investigated for breach of trust
Coalition led by Iraqi PM al-Sudani wins parliamentary elections
Will your baby get a hep B vaccine? What RFK panel's ruling means.












