
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
Watching ‘Home Alone’ with the kids this holiday season? Brace yourself for '6-7.' - 2
Opening Achievement: 8 Methodologies for Compelling Using time productively - 3
Gulf countries continue to face Iran attacks as criticial energy infrastructure at risk - 4
This Huge Ocean Beast Shifts Sharks’ Evolutionary Timeline - 5
Conquering Language Boundaries: Individual Accounts of Multilingualism
Vote in favor of the Top Vegetable for Senior
Famous Network programs in Europe and America
5 Breakout Stars in Ongoing television Series
5 Cell phones of the Year
Tesla plans to expand production at German car plant
Getting Your Youngsters' Future: Grasping Legacy Regulations
Fossils unearthed in Morocco are first from little-understood period of human evolution
Anti-war protests held across Israel under wartime gathering limits
Computerized Moderation: Tracking down Equilibrium in the Advanced Age













