
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
How Google, Microsoft, Walmart, and other corporate giants are preparing for an aging workforce - 2
Poll: By a 2-to-1 margin, Americans say Trump has done more to raise prices than lower them - 3
6 U.S. States for Climbing - 4
Displaced Palestinian families suffer as heavy rains flood Gaza tent camps - 5
James Webb Space Telescope spies mysterious high-energy radiation in star nursery
New movies to watch this weekend: See 'We Bury the Dead' in theaters, rent 'Wicked: For Good,' stream 'The Unbreakable Boy' on Starz
I’m a neuroscientist who taught rats to drive − their joy suggests how anticipating fun can enrich human life
Manual for Conservative SUVs For Seniors
Artemis II's moonbound toilet is working again to astronauts' relief after overnight fix
Scientists find new clues to why female fertility declines with age
ISS astronaut snaps stunning nighttime photo of Florida and Cuba | Space photo of the day for Dec. 29, 2025
6 Popular Ladies' Aromas On the planet
The Significance of Prenuptial Arrangements in Separation Procedures
More than 800 flights canceled as FAA cuts traffic at 40 major airports. Here's what to know.












