
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
Several injured as man threatens attack on German high-speed train - 2
12 times rockets and spacecraft crashed and burned in 2025 - 3
The Most Famous Virtual Entertainment Powerhouses of the Year - 4
5 Fundamental Ways to employ a Criminal Legal counselor - 5
Rick Steves' Favorite Time To Visit Spain Has Lower Prices And Fewer Crowds
Sheinelle Jones will cohost fourth hour of 'Today' with Jenna Bush Hager: Here's what to know about her
Geminid shooting stars: One of 2025's most exciting meteor showers begins tonight
Artemis II crew take new photo of far side of the moon
Figure out How to Utilize Your Web based Advertising Degree to Break into the Tech Business
From Amateur to Master: My Involvement in Photography
Avoid Large Crowds In Bali & Swim At This Peaceful Waterfall With A Gorgeous, Natural Pool
My skin feels drier, my lips thinner and my makeup heavier. How do I adjust my routine for aging skin?
Pulsars to the extreme: Spinning dead stars found blasting radio signals from the 'edge of their magnetic reach'
Last supermoon of the year, the cold moon, seen across the U.S.: See the photos












