
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
Idris Elba is the king of the stress-watch - 2
Mountain Trekking on a Tight spending plan: Tracking down the Right Bicycle - 3
The most effective method to Offset Album Rates with Liquidity Needs - 4
RFK Jr.'s diet guidelines emphasize red meat, full-fat dairy. How healthy are they? - 5
Share your pick for the miniature headphones that you generally suggest!
Israel launches new wave of attacks against Hezbollah in Beirut
First Greenland, now Iceland? Annexation joke by Trump ally gets frosty response in the Arctic nation.
Doctor's orders: Eat ice cream, and other tips for a long and healthy life
Qatar LNG Ships U-Turn After Attempt to Pass Through Hormuz
Longtime United Launch Alliance CEO Tory Bruno resigns from space company. 'Finished the mission I came to do.'
7 Odd Apparatuses to Make Your Party Stick Out!
The largest sun of 2026 rises today as Earth draws closest to our parent star
Vote in favor of your Favored kind of pasta
Vote In favor of Your Favored Kind Of Cheddar












