
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
Manual for Mountain Objections on the planet - 2
The Most Rousing Ladies Business visionaries of Today - 3
Ukraine proved this drone-killer works. Now, the West is giving it a shot. - 4
One perk to marrying Richard Marx later in life? 'We don't have time' for stupid arguments, says Daisy Fuentes. - 5
France's Senate backs ban on social media platforms for under-15s
Scientists solve the mystery of the prehistoric 'Burtele Foot'
When fake data is a good thing – how synthetic data trains AI to solve real problems
French Senate debates social media ban for under-15s
With Obamacare premium hikes, more people opting for no coverage or cheaper plans
Scientists captured female sperm whales on video working together during a birth to protect the calf
Melodic Event: A Survey of \Energies and Exhibitions Assessed\ Live concert
21 Incredibly Interesting Contemplations To Observe Consistently
How to watch 'A Charlie Brown Christmas' for less with this Apple TV Black Friday deal
Fundamental Home Items Each Animal person Needs













