
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
Home Security Frameworks with Shrewd Elements - 2
How did I get my own unique set of fingerprints? - 3
A hospital discharged a woman in labor. This lawmaker wants change. - 4
Fact Check: Some Bridge Photos Circulating Do NOT Show The Hongqi Bridge That Collapsed In Southwest China Nov. 11, 2025 - 5
Iran war pushes Germany's deficit to 4.2% as growth outlook is cut by 50%
EU waters down plans to end new petrol and diesel car sales by 2035
Could the Star of Bethlehem have actually been a comet?
Vote In favor of Your Favored Language Interpretation Administration
Germany's Pistorius: NATO protects Europe from Iranian missiles
She's been a Bond girl and a mutant. Now she's grappling with Hollywood's obsession with 'eternal youth.'
Artemis 2 astronauts arrive at Kennedy Space Center ahead of NASA's historic launch around the moon
ChatGPT served as "suicide coach" in man's death, lawsuit alleges
How a Middle East War Becomes a Retail Price Hike
Astronomers detect black hole blasting winds at incredible speeds













