
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
Former Australian soldier arrested over alleged Afghan war crimes - 2
Brazil passes anti-gang law allowing seized crypto to fund security forces - 3
Heart disease risk greater for women with a common condition they may not be aware they have - 4
Commonsense Ways to work on Your Funds with a Restricted Pay - 5
Remains of banker missing since 1999 found on California beach by family looking for seashells
German economic institutes cut forecast in half over Iran war
Are multiverses real? An astrophysicist explains why it depends on how you define ‘real’
Book excerpt: "Enough" by Dr. Ania Jastreboff and Oprah Winfrey
Must-See Attractions in Australia
Step by step instructions to Guarantee the Life span of Your Dental Inserts: Support and Care Guide
RFK Jr.'s handpicked vaccine panel just voted to stop recommending hepatitis B shots for all newborns. Why experts object.
Winter storm headed for Midwest to Northeast. Here's how much snow to expect.
How did Hugh Jackman nail his latest role? Sequins, tighty-whities and embracing 'zero embarrassment.'
Spain’s Picos de Europa: What to see and do in ‘the world’s most beautiful place’













