
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
Top 10 Books That Will Have an impact on Your Viewpoint - 2
80 km. on foot: Sharren Haskel’s three-day march in protest of haredi draft bill - 3
Famous Versatile Brands: Your Decision - 4
At least 18 Palestinians killed in latest clashes in Gaza - 5
Here's how 'Bridgerton' fans can watch the first episode of Season 4 before its Netflix release later this month
The Best Portable Applications for Psychological wellness and Prosperity
Find the Effect of Web-based Entertainment on Society: Exploring the Computerized Scene
6 Top of the line Lodgings All over The Planet, Which One Do You Concur With
What to know about Jack Dorsey's new Vine revival, DiVine
The Specialty of Cleaning up: Change Your Space and Brain
Building Tough Connections: Individual Bits of knowledge on Association
Pick Your Number one sort of blossom
Story of ‘first Black Briton’ rewritten by advances in ancient DNA technology
Invigorating Spots To Go Kayaking All over The Planet











