
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
India’s delayed climate plan sets modest emission target - 2
What were the little white pills found in Tiger Woods's pocket at the scene of his crash? What to know about hydrocodone. - 3
The most effective method to Oversee Unsold SUVs in the Car Business - 4
Flourishing in a Cutthroat Work Market: Vocation Methodologies - 5
6 Useful Home Espresso Machines
Vial marked 'Polonium 210' sparks scare during German Easter egg hunt
NASA's Apollo 8 moonshot saved 1968. Could Artemis 2 do the same in 2026?
Shipping: The Corridors of Trade and the Coming of Another Period
Fundamental Home Exercise center Hardware: Amplify Your Exercises
Pick Your Favored kind of soup
The most effective method to Boost Benefits in Gold Speculation: Master Techniques and Tips
Relive NASA's historic Artemis 2 launch to the moon in these stunning photos
First Alert: Light snow through this evening
This Week In Space podcast: Episode 186 — Snow on the Moon?













