
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
At least 490 protesters killed in Iran, activists say - 2
Jamaica reports deadly leptospirosis outbreak after Hurricane Melissa - 3
Trump says Venezuela will start 'turning over' oil to the U.S. Is that the reason he toppled Maduro — or is it something else? - 4
2024 Style: The It-Things You Want in Your Closet - 5
Study reveals links between global food systems, obesity, and climate change
Island Travel Guide: Must-Visit Objections for 2024
Watch live as near-Earth asteroid Eros buzzes the Andromeda Galaxy on Nov. 30 (video)
Bad flu season getting worse; skyrocketing cases set state record
I decided to become a single mother by choice. I wasn't ready to stop dating.
Some gifted dogs can learn new toy names by eavesdropping on owners
Find the Standards of Viable Nurturing: Supporting Blissful and Strong Kids
21 Things You Ought to Never Tell Your Childless Companion
These HGTV stars made a pledge to keep their kids off smartphones. Here's how it's going.
The Best Computer games for Multiplayer Fun













