
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
100 new alien worlds: Scientists find hidden haul in data from NASA exoplanet-hunting spacecraft - 2
Pat Finn, actor from 'The Middle,' dies at 60 after bladder cancer diagnosis - 3
Purdue Pharma's deal means money for some victims, end of Purdue company name. Here's what to know - 4
5 State of the art Advancements in Computer generated Simulation - 5
NASA's Artemis 2 astronauts are cruising to the moon. So why are they doing CPR tests today?
Hand Skin Is Additionally Significant - What You Ought to Realize About Hand Cream
How much should a kid's birthday party cost? One mom spent $190 for pizza and ice cream at a park. Another paid $2K for a playspace and goodie bags.
Washington resident contracts bird flu, first human case in U.S. since February
Solar storms have influenced our history – an environmental historian explains how they could also threaten our future
Unwinding the Starting points of America: An Excursion Through History
New electric car registrations rise sharply in Germany in March
Figure out How to Amplify Innovation and Infotainment Frameworks for Senior Drivers in SUVs
A Manual for SUVs with Less Noteworthy Gas Mileage
Embrace Effortlessness: Moderation and Cleaning up Tips













