
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
When will the Epstein files be released — and will they reveal anything new? - 2
Nordic people know how to beat the winter blues. Here's how to find light in the darkest months - 3
This Canadian crater looks like marbled meat | Space photo of the day for Jan. 6, 2026 - 4
Rediscovering Experience Through Excursions: Individual Travel Stories - 5
Which Switch Game Do You Suggest? Share Your Decision
Step by step instructions to Remain Spurred While Chasing after a Web-based Degree
CVS forecasts 2026 profit above estimates on strong performance
From Squid Game to Your Party! Six Entertaining Test Games That Will Have You in Join
Step by step instructions to Guarantee the Life span of Your Dental Inserts: Support and Care Guide
New York to require social media platforms to display mental health warnings
Procter & Gamble changes Crest toothpaste packaging to address Texas AG fluoride concerns
Putin, Netanyahu discuss Middle East in phone call, Kremlin says
What happened in 'Wicked' part 1 and will there be a 3rd movie? Recap and what Ariana Grande, Jon M. Chu have said about a sequel.
Turning into a Distributed Writer: My Composing Process












