
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
Mussolini's summer villa on Adriatic coast sold for €1.2 million - 2
Conquering Social Generalizations: Individual Accounts of Strengthening - 3
Innovative Versatility: Examples of overcoming adversity from Entrepreneurs - 4
Putin critic gets six years in penal colony, vows hunger strike - 5
The Best Web-based Courses for Ability Advancement
Report in relation to renaming Herzog Park set to be withdrawn
It's official: NASA's Artemis 2 moon mission will break humanity's all-time distance record
Al-Sharaa denies he called for 80% of Syrians to return from Germany
5 Side interests That Work on Psychological wellness
What to know as New York City nurses strike for a 3rd day
Over 1,800 killed since junta seized power in Burkina Faso, rights group says
Meet Beef the bulldog, who takes slow walks with his 78-year-old friend
Pick Your Favored kind of salad
Bitcoin momentum builds in Abu Dhabi as global interest surges













