
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
NASA releases stunning new images captured by the Artemis II moon mission, including 'Earthset' and a solar eclipse from space - 2
FBI arrests Brian Cole Jr. in Jan. 6 pipe bomb investigation, ending 5-year hunt - 3
Five killed in Israeli air strikes on tents near Khan Younis, medics say - 4
Let them eat (Taylor Swift) cake: The baker turning A-listers into life-size desserts - 5
Analysis-NASA's moon mission tests aerospace old guard as SpaceX, Blue Origin hover
Winter storm warnings issued across Northeast as up to 9 inches of snow forecast; deadly atmospheric river in California snarls travel
7 Methods for further developing Rest Quality
These four astronauts could soon travel farther from Earth than anyone has gone before
5 Eating routine Well disposed Snacks to Keep You Fulfilled
Iran denies launching ballistic missiles towards Kurdistan region of Iraq
German finance minister sees advantages of smartphones in schools
The Job of a Migration Legal advisor: How They Can Help You
Iran begins cloud seeding to induce rain amid historic drought
After harsh winter, Ukrainians find joy in releasing bats rescued from war












