
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
Amateur's Manual for Venture Strategies for Tenderfoots - 2
The Universe of Impeccable Pearls: A Manual for Valuable Gems - 3
How stripping diversity, equity and inclusion from health care may make Americans sicker - 4
Far-right leader Le Pen to attend Brigitte Bardot's funeral - 5
Excelling at Discussion: Genuine Examples of overcoming adversity
The 10 Most Persuasive Forerunners in Innovation
Barry Manilow to have surgery for early-stage lung cancer and postpones January concerts
Lebanon says Israeli strike killed 13 people near Palestinian refugee camp
Trump declares Christmas Eve and Dec. 26 federal holidays: What does that mean?
Archaeologists uncover details about the Hjortspring boat's origins
Elite Execution Wall televisions for Film Darlings
Shas threatens to oppose 2026 state budget over haredi food-voucher exclusion
Instructions to Augment the Presentation of Your Kona SUV
Turkey's Erdogan denounces Israel-Greece-Cyprus trilateral summit, affirms support for Gaza












