
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
Germany's first Omani LNG shipments arrive despite Middle East disruptions - 2
Find the Specialty of Public Speaking: Drawing in and Convincing Crowds with Certainty - 3
Four new luxury hotel openings in Italy you need to know about - 4
Your kid wants it now. What saying yes, no or not yet teaches kids about money and instant gratification. - 5
Rights group: At least 2,500 deaths during protest crackdown in Iran
Violence 'never part' of break-in plan, court told
China bans storing cremated remains in empty 'bone ash apartments'
Songbirds swap colorful plumage genes across species lines among their evolutionary neighbors
Find the Marvels of the World with These Travels
Favored Vehicle for Seniors: Make Your Determination
The Ascent of the Kona SUV: How Hyundai's Reduced Hybrid Is Vanquishing the Streets
The Best Cell phone Brands for Tech Aficionados
Our 10 favorite Space.com reader astronomy photos of 2025
Southern Californians, your health insurance costs could rise in 2026













