
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
New science points to 4 distinct types of autism - 2
A volcanic eruption may have catalyzed the plague's arrival in Europe, study suggests - 3
'All Her Fault' ending explained: The shocking conclusion to the psychological thriller inspired by true events - 4
Pick Your Favored sort of cooking - 5
Smartwatches: Remain Associated and Dynamic
Saturn shines with the waxing moon at sunset on Nov. 29
Radiate brilliantly: The 5 Precious stone Rings to Purchase in 2024
Director of Swiss hospital describes the rush to treat the injured from Alpine resort bar fire
New movies to watch this weekend: See 'They Will Kill You' in theaters, rent 'Send Help,' stream 'Pretty Lethal' on Prime Video
Dave Coulier reveals he has tongue cancer, his 2nd diagnosis in a year, after beating non-Hodgkin lymphoma
This Week In Space podcast: Episode 192 — Space, 2026!
The Solution to Ecological Protection: Saving Nature for People in the future
NASA Artemis II tracker: Where is the Orion now and when will it reach the moon?
General Atlantic says ‘biggest mistake’ would be pulling back on Gulf deals












