
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
Astronomers spot white dwarf star creating a colorful shockwave - 2
How to sound like an astronaut as you follow the first human moon mission in more than half a century - 3
Careful Living: Embracing the Current Second - 4
Newly Identified ‘Lucy’s Hunter’ Was a 15-Foot Crocodile Lurking in Ancient Ethiopia - 5
Find Serenity: 10 Stunning Setting up camp Areas
Simple Consideration Plants for Home and Office: An Aide
Eleven Creations And Developments That Steered History
Instructions to Keep an Inspirational perspective After Cellular breakdown in the lungs Treatment
I watched more than 500 new movies this year. These are the 25 best ones.
Island Travel Guide: Must-Visit Objections for 2024
Lilly becomes first healthcare firm to join trillion-dollar club, Wall Street reacts
Safeguarding Your Senior Protection Against Extortion and Tricks.
NASA's SPHEREx telescope completes its 1st cosmic map of the entire sky and it's stunning!
Which Kind of Pet Makes the Incomparable Buddy?













