
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
Israel intensifies Lebanon attacks and hits areas not in Hezbollah's control - 2
Cheetos and Doritos to launch new versions without artificial dyes - 3
Figure out How to Use Your Nursing Abilities for Better Compensation - 4
Charli xcx recorded original songs for 'Wuthering Heights' — what to know about the new album for Margot Robbie's film - 5
UAE-backed Yemeni Southern Transitional Council denies disbandment rumors
Becoming amazing at Arranging Pay Raises
Ariana Grande says Eternal Sunshine 2026 tour will be her last for a 'long, long time': 'One last hurrah'
Israel faces tough choices over haredi draft exemptions, legal expert warns
I spent the last year transforming my life. Becoming a Rockette for a day made me confront a fear I couldn't shake.
Dr. Vinay Prasad's memo raises concerns about COVID-19 vaccines and pediatric mortality
2026 will be the year NASA astronauts fly around the moon again — if all goes to plan
Pick Your #1 Kind Of Treat
Manual for extravagance SUVs for seniors
Authentic Urban areas: Rich Legacy and Lively Societies











