
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
This Miraculous, Cliff-Perched Town In The South Of France Is A Sacred European Gem - 2
Pulsars to the extreme: Spinning dead stars found blasting radio signals from the 'edge of their magnetic reach' - 3
How will the universe end? - 4
Journey through Pages: A Survey of \Plunging into Scholarly Universes\ - 5
Vote in favor of Your #1 BWM Vehicles
8 key takeaways from Savannah Guthrie's 'Today' interview on the disappearance of her mother
November Lease Deals for the 2025 Kia EV6 are Too Good to Pass Up
Illumina unveils dataset to speed up AI-powered drug discovery
Instructions to Boost Your True capacity with a Brain research Degree
How a niche Catholic approach to infertility treatment became a new talking point for MAHA conservatives
Interstellar comet 3I/ATLAS to pass closest to Earth on Friday
Israel has clear objectives south of Litani River, but will face difficult choices further north
Little Italy Mercato brings fresh food and community to downtown San Diego
How C-reactive protein outpaced ‘bad’ cholesterol as leading heart disease risk marker













