
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
Brilliant and Gleaming: Excellence and Skincare Practices - 2
Toyota’s Next Big Sports Car Might Apparently Be a Turbocharged All-Paw Beast - 3
Doctor's orders? ‘Belly laugh at least two to five days a week' - 4
Artemis II astronauts are more than halfway to the moon as they seek to break Apollo 13's record - 5
Exploring the Gig Economy: Illustrations from Consultants
Vote In favor of Your Favored Kind Of Organic product
Becoming the best at Discussion: Individual Procedures
How will the universe end?
Who is behind Al-Majd, the Israeli-linked evacuation group sending Gazans to South Africa?
I'm a woman who's into weightlifting. Was I man enough for the creatine-packed 'Man Cereal'?
Mars orbiter sees 'butterfly' crater spread its wings on the Red Planet
As her kidneys fail and time runs short, this activist fights to decriminalize euthanasia in Mexico
Carina Nebula shines with white-blue stars | Space photo of the day for Jan. 5, 2026
Air India chief resigns 10 months after devastating Ahmedabad crash and amid mounting financial troubles













