
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
How stripping diversity, equity and inclusion from health care may make Americans sicker - 2
Orcas seen hunting great white sharks to eat their livers in drone footage recorded in Mexico - 3
Stop the ‘good’ vs ‘bad’ snap judgments and watch your world become more interesting - 4
Thousands of New York City nurses set to strike Monday if deal isn't reached with hospitals - 5
Grasping Various Kinds of Local misdemeanors
Check out the exclusive pitch deck Valerie Health used to raise $30 million from Redpoint Ventures to automate healthcare faxes
How a seabird native to Hawaii has adapted to life in Honolulu's concrete jungle
Doulas play essential roles in reproductive health care – and more states are beginning to recognize it
Top 15 Supportable Design Brands Coming out on top
American tourists left stranded in the Caribbean following flight cancellations after airspace closed for Maduro operation
The Universe of Impeccable Pearls: A Manual for Valuable Gems
Desired Travel Objections Worldwide: Where to Go Straightaway
Top 10 Smash hit Computer games of the Year
U.S. to drop childhood vaccine recommendations as it looks to Denmark, Washington Post reports













