
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
German finance minister sees advantages of smartphones in schools - 2
Hostages as leverage: Iran's secret demand aimed at crippling Israel's agriculture - 3
NASA shares first photos of Earth taken by Artemis II - 4
Best Veggie lover Dinner: What's Your Plant-Based Pick? - 5
IDF bans Android phones for senior officers, iPhones now mandatory, Army Radio reports
How 2025 became the year of comet: The rise of interstellar 3I/ATLAS, an icy Lemmon and a cosmic SWAN
Jenny & Dave Marrs Mourn Loss of Former ‘Fixer to Fabulous: Italiano’ Guest
A coup too far: Why Benin's rebel soldiers failed where others in the region succeeded
Arrow Exploration brings new Colombian oil well on stream ahead of schedule and under budget
Kissing is an ‘evolutionary conundrum.’ Scientists just mapped its unexpected origins
These 2 moon rovers used cameras and lasers to hunt for simulated water ice — and one looks like WALL-E
Gauging the Upsides and downsides of Visas: A Complete Aide
When a sperm whale gives birth, the mother gets help from her friends
RFK Jr.'s handpicked vaccine panel just voted to stop recommending hepatitis B shots for all newborns. Why experts object.













