
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
Liste des pr\u00eats qui ne n\u00e9cessitent pas de remboursement - 2
'The Boys' Season 5 premiere: How to watch for less, what to know about the final series and more - 3
Step by step instructions to Shield Your Wellbeing Around 5G Pinnacles\ - 4
Solid Propensities: Little Changes for a Superior Life - 5
Find the Effect of Web-based Entertainment on Psychological wellness: Exploring the Advanced Scene Securely
Takeda's AI-crafted psoriasis pill succeeds in late-stage studies
NASA's Artemis 2 astronauts are cruising to the moon. So why are they doing CPR tests today?
Top Smoothie Flavor: What's Your Mix?
A Past filled with Old Civilizations: The World's Most established Societies
German foreign minister backs abandoning EU's unanimity principle
Report in relation to renaming Herzog Park set to be withdrawn
75% of Arab Israelis support Arab party joining government coalition post-war, survey reveals
The most effective method to Quick Track Your Outcome in Advanced Showcasing with a Web-based Degree
Sally Rooney books may be withdrawn from UK sale over Palestine Action ban, court told












