
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
Find the Standards of Powerful Cooperation: Accomplishing Cooperative energy and Coordinated effort - 2
6 Tire Brands Reasonable for Seniors - 3
Top 20 Wellbeing and Wellness Applications for a Sound Way of life - 4
The most effective method to Pick the Right Old Consideration Administration: Key Contemplations - 5
How to watch 'A Charlie Brown Christmas' for free this weekend
My skin feels drier, my lips thinner and my makeup heavier. How do I adjust my routine for aging skin?
White House responds to Sabrina Carpenter after pop star slams 'evil' ICE video using her song
'Malcolm in the Middle' reboot releases 1st trailer, reuniting Frankie Muniz and Bryan Cranston: Watch here
Setbacks in Texas and elsewhere put Republicans' redistricting hopes in doubt as key deadlines loom
Scientists find twisting magnetic waves on the sun. Could this help solve a huge solar mystery?
Watch This Glacier Race into the Sea
10 Demonstrated Tips to Dominate Video Altering on Your Cell phone in 2023
Can a mammogram help identify heart disease?
Must-See Attractions in Australia












