
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
Must-Have Wellness Gear: What to Purchase for Successful Exercises - 2
Sean 'Diddy' Combs faces new sexual assault allegations, currently under investigation by Los Angeles Sheriff's Department - 3
How federal officials talk about health is shifting in troubling ways – and that change makes me worried for my autistic child - 4
The 15 Most Rousing TED Chats on Self-awareness - 5
If someone's always late, is it time blindness, or are they just being rude?
National health ranking puts Georgia near bottom of list. Here's why
Robyn returns to music with 'Dopamine,' her 1st single in 7 years: 'Came to save music once again'
Mont Blanc road tunnel reopens to traffic after 15 weeks of repairs
In vogue Sleepwear Patterns for 2024
Pick Your Favored sort of cooking
Smooth countdown continues for Artemis II moon mission
Sustaining Public activity and Connections: Key Methodologies
A definitive Manual for Picking Electric Vehicle: Decision in favor of Your Number one
Jasmine Crockett in, Colin Allred out: A major shakeup for Democrats in their quest to finally win a Senate seat in Texas










