
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
In Antarctica, photos show a remote area teeming with life amid growing risks from climate change - 2
New materials, old physics – the science behind how your winter jacket keeps you warm - 3
Artemis 2 astronauts fly around the moon in record-breaking lunar loop by NASA - 4
Why is everyone talking about Paul Dano? George Clooney becomes the actor's latest defender in this 'time of cruelty.' - 5
Tata Motors, BMW among automakers set to raise prices in India
Instructions to Back Your Sunlight powered chargers: Tracking down Possible Choices
Storm Goretti sweeps United Kingdom, France with winds over 120 mph
ISS astronauts spy airglow and dwarf galaxy | Space photo of the day for Jan. 13, 2026
EU states agree first step for Ukraine reparations fund
Global measles cases drop 71% in 24 years as vaccination coverage improves, WHO says
The Tiny Channel Island With 65 Residents That Chefs And Foodies Go Out Of Their Way To Visit
Ukrainian foreign minister appeals for funds for drones
Protester climbs on to balcony of Iranian embassy in London
How to watch 'A Charlie Brown Christmas' for free this weekend












