
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
8 Fundamental Stages: Novice's Manual for Secure Your Android with a VPN - 2
Every year, she thanks the trooper for the arrest that led to her sobriety - 3
The most effective method to Pick the Right Old Consideration Administration: Key Contemplations - 4
Vote In favor of Your Favored Menial helper Administration - 5
Cyclone causes blackout, flight chaos in Brazil's Sao Paulo
JFK's granddaughter reveals terminal cancer diagnosis, criticizes cousin RFK Jr.
Struggling to keep your New Year's resolutions? Here's how to keep yourself on track
New science points to 4 distinct types of autism
Smartwatches: Remain Associated and Dynamic
Winona Ryder didn't take the 'Stranger Things' plot lightly. How 'otherworldly' grief and a kidnapping in her hometown informed her character.
Gilead's new HIV prevention shot added to CVS's drug coverage lists, CEO says
Drenched in Pixels: A Survey of \Vivid Interactivity Experience\ Game
The Best Games Crossroads in History
Extravagance SUVs for Seniors: Solace, Innovation, and Security













