
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
Best Augmented Simulation Ride: Which One Feels Generally Genuine? - 2
Sports Shoes of 2024: Upgrade Execution and Solace - 3
Europe picks companies to help build Argonaut moon lander - 4
A Concise History Of The Entertainment world - 5
What’s your chronotype? Knowing whether you’re a night owl or an early bird could help you do better on tests and avoid scams
‘It’s Israeli policy’: Report reveals abuse of Palestinians in prisons
Bolsonaro briefly leaves Brazilian prison for medical tests after a fall from his bed
Revvity says it will exceed 2025 profit forecast range
How does Spotify Wrapped calculate your listening age? What your number says about you.
A new mom skipped a routine appointment. An infected cut led to a devastating diagnosis
Merck urges science-led US vaccine schedule after CDC trims childhood vaccine list
Concern for couple jailed in Tehran as British embassy closes
I’m a dad to an autistic child. Here’s how you can make the holidays easier for all of us.
Vote in favor of your #1 Kind of Cap












