
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
What Yogurt Types Do You Know05.06.2024 - 2
The Solution to Defeating Tarrying: Systems for Expanded Efficiency07.07.2023 - 3
Portable Installment Answers for Independent ventures06.06.2024 - 4
Jasmine Crockett in, Colin Allred out: A major shakeup for Democrats in their quest to finally win a Senate seat in Texas08.12.2025 - 5
New findings suggest atmosphere could exist on exoplanet TOI-561b12.12.2025
Bolsonaro briefly leaves Brazilian prison for medical tests after a fall from his bed
When will the Epstein files be released — and will they reveal anything new?
Turning into a Distributed Writer: My Composing Process
IDF bans Android phones for senior officers, iPhones now mandatory, Army Radio reports
Fake new headlights rule steer Australian drivers astray
Feeling wonder every day improves our health. Here’s how to do it.
Ultra-Orthodox protests erupt across Israel on haredi IDF enlistment day
How to identify animal tracks, burrows and other signs of wildlife in your neighborhood
Pat Finn, actor from 'The Middle,' dies at 60 after bladder cancer diagnosis











