
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
Enormous Credit And All that You Really want To Be aware - 2
Intriguing Social Unesco World Legacy Locales All over The Planet - 3
The most effective method to Settle on Informed Conclusions about Senior Insuranc. - 4
This Week In Space podcast: Episode 186 — Snow on the Moon? - 5
IDF destroys Hamas shaft in northern Gaza with loaded 'ready to fire' rocket aimed at Sderot
The Main 15 Applications for Efficiency and Association
Vote In favor of Your Favored Comupter Game
British Columbia's Secret Lakeside Town With Hot Springs Is 'An Oasis Of Arts, Culture And Relaxation'
Sea Ice Hits New Low in Hottest Year on Record for the Arctic
Gulf of Mexico oil spill spread hundreds of miles, killed wildlife and polluted Mexican reserves
6 Tire Brands Reasonable for Seniors
Investigating Inside Plan and Home Style: Change Your Residing Space
Famous Restroom Beautifying Styles For 2024
A red meat allergy from tick bites is spreading – and the lone star tick isn’t the only alpha-gal carrier to worry about













