
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
Gaza receiving over 70,000 cubic meters of water per day, COGAT claims - 2
3 astronauts settle into their new life in orbit | On the International Space Station this week Dec. 1-5, 2025 - 3
Shas threatens to oppose 2026 state budget over haredi food-voucher exclusion - 4
Figure out how to Amplify Your Open Record Reward - 5
Select Your Definitive Pizza Decision
Several injured as man threatens attack on German high-speed train
New heart disease calculator predicts 30-year risk for young adults
Jeff Bezos’ Blue Origin launches landmark Mars mission in New Glenn rocket’s first big test
Most loved Amusement Park for Small children: Which One Do You Suggest?
The Delight of Perusing: Book Proposals for Each Class
Giant ‘toothed’ birds flew over Antarctica 40 million to 50 million years ago
‘It’s Israeli policy’: Report reveals abuse of Palestinians in prisons
Al-Sharaa denies he called for 80% of Syrians to return from Germany
The 10 Most Compelling Forerunners in Innovation













