
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
UAE used military bases in Red Sea region to aid Israel's war against Hamas, leaks reveal - 2
Sa'ar warns German delegation: 'A Palestinian state would be a Hamas terror state' - 3
Japan deploys the military to counter a surge in bear attacks - 4
Starbucks' new 'Bearista' cup is causing a stir — and is being listed on eBay for $600 - 5
Top Frozen yogurt Flavor: Cast Your Vote!
Pick Your #1 Sort Of Espresso
A Manual for Pick High Evaluated Food Conveyance Administrations In Significant Urban communities For 2024
The most effective method to Guarantee Simple Availability in Seniors' SUVs
6 Famous kind of practice on the planet
Careful Connections: Building Association and Trust
Fascinating Fishing Objections From Around The World
US FDA approves Kura-Kyowa's blood cancer therapy
Support Your Investment funds with These Individual accounting Thoughts
Should you get an RSV vaccine this fall? What to know and where to get a shot












