
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
4 Family SUVs: Joining Solace and Style - 2
The Most Paramount Crossroads in Olympic History - 3
Figure out How to Amplify the Resale Worth of Your Kona SUV - 4
Shooting of MIT professor Nuno Loureiro has police searching for a suspect - 5
1st-ever disease gene fix, Alzheimer's blood test: 7 medical breakthroughs in 2025
The most effective method to Integrate Compact disc Rates into Your Retirement Arranging
Accor signs agreement to transform El Gouna resort as Sofitel
A decade after Brazil’s deadly dam collapse, Indigenous peoples demand justice on the eve of COP30
‘Inoculation’ helps people spot political deepfakes, study finds
Scientists document a death from a meat allergy tied to certain ticks
In the background: Visiting Notable Film Areas All over the Planet
James Webb Space Telescope's mysterious 'little red dots' may be black holes in disguise
Wolf Bites Woman in Shocking Attack at Busy Shopping Center
Iran-backed militias reassert power in Iraq, proving the Islamic axis is still standing













