
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
Czech Republic's new premier: No money for Ukraine - 2
Greece eyes migrant repatriation centres outside the EU - 3
From Squid Game to Your Party! Six Entertaining Test Games That Will Have You in Join - 4
Nuno Loureiro, MIT physicist, fatally shot at home; police investigate - 5
Rediscovering Euphoria: Individual Accounts of Conquering Despondency
Indian rocket launches record-breaking BlueBird 6 smartphone satellite to orbit (video)
ByHeart baby formula from all lots may be contaminated with botulism bacteria, tests show
Motivational Travel Objections for History Buffs
The Best Design Bloggers for Style Motivation
Takeaways from AP’s report on potential impacts of Alaska’s proposed Ambler Access Road
Getting breast implants was a mistake I live with every day. Why I’m sharing my story now, at 70, in pain and afraid.
Flu activity in US could continue to rise for weeks, top CDC epidemiologist says
Eleven arrested over mass shooting in South Africa tavern
Vinicultural Investigation: A Survey of \Enjoying Fine Vintages\ Wine sampling













