
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
Would you ever turn to AI for companionship? 6% of Americans say they could — or already have. - 2
Artemis II updates: NASA's moon mission breaks Apollo record for farthest distance humans have traveled from Earth - 3
Understanding Successful Compromise Standards to Cultivate Agreeable Connections - 4
Find the Historical backdrop of Common liberties: Advancing Equity and Equity Around the world - 5
Make your choice for the PS5 game that you love playing with companions!
Artemis II astronauts channel Apollo 8 with a striking Earthset photo
Hundreds are quarantined in South Carolina as measles spreads in 2 US outbreaks
Central African Republic's president sworn in for a third term after disputed election
Human evolution’s biggest mystery has started to unravel. How 2025 tipped the scales
This Asian country is the next hot travel destination, and this is one of its best hotels
Israel Police arrest twenty-one as anti-war protests grow despite broad support for Iran war
Between 600 to 800 aid trucks entering Gaza daily since start of ceasefire, COGAT confirms
McDonald's is bringing two 'KPop Demon Hunters' meals to McDonald's. Here's what they include and when they launch.
What to watch for in weight loss drugs in 2026













