
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
German inflation soars to 2.7% in March as Iran war drives up prices - 2
Merck sees over $5 billion opportunity in Cidara's experimental flu drug - 3
Partner of crime boss Steven Lyons arrested in Dubai - 4
China bans storing cremated remains in empty 'bone ash apartments' - 5
ONE returns to Red Sea with new service
Wegmans recalls mixed nuts over salmonella contamination fears
Canada Awards C$1.5 Billion Defense Contracts to L3Harris, Airbus
Study reveals links between global food systems, obesity, and climate change
A mom's viral post is raising the question: Do kids need snacks? Dietitians have answers.
Understanding Preschool Projects: Cultivating Abilities and Advancement
Decrease in Home Buy Credits and Home loan Renegotiating Rates: An Outline of Latest things
Remain Fit: Powerful Wellness and Work-out Schedules for a Better You
Watch interstellar comet 3I/ATLAS speed away from the sun in free telescope livestream on Nov. 16
Takeaways from AP’s report on potential impacts of Alaska’s proposed Ambler Access Road












