
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
Monetary Versatility: Get ready for Life's Unforeseen Difficulties - 2
General Atlantic says ‘biggest mistake’ would be pulling back on Gulf deals - 3
WATCH: IDF strikes, dismantles missile launchers in southern Lebanon - 4
Figure out how to Guarantee Your Dental Embeds Endure forever - 5
Vote In favor of Your Favored Treat
Vote in favor of Your #1 Climbing boots Now
Burkina Faso must 'forget' about democracy, military leader says
In blow to Lula, Brazil Congress revives controversial environmental bill
Pick Your Favored kind of salad
Polls open in tense Uganda election amid widespread delays
U.S. to drop childhood vaccine recommendations as it looks to Denmark, Washington Post reports
'Dancing with the Stars' semifinals: How to watch Episode 10 tonight, where to stream, who's left and more
From a new flagship space telescope to lunar exploration, global cooperation – and competition – will make 2026 an exciting year for space
Is relief in sight? Flu season still brutal but cases are declining.













