
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
7 Straightforward Moves toward Move Information from Your Old Cell phone to Your New One: A Thorough Aide - 2
The best overlooked performances of 2025 - 3
Divorce filings, feuds and legal trouble: The 'Mormon Wives' drama keeps piling up - 4
RFK Jr.'s vaccine panel delays hepatitis B shot vote after chaotic meeting - 5
Exploring Asia’s Realm of Flying Snakes
Consumer experts: German petrol hikes rule won't bring down prices
5 Wellbeing Applications Assist You With remaining Fit
Trial of pro-Palestine activist begins
This Unique National Park In Canada Is Famous For Its Otherworldly Limestone Monoliths
Kids with smartphones by age 12 are at higher risk of health issues, study finds
The Solution to Ecological Protection: Saving Nature for People in the future
Figure out How to Alter Your Volvo XC40 for Further developed Solace
How to avoid or deal with an outrageous medical bill
Family Holiday spots













