
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
Inside Plan with Houseplants: An Aide - 2
The Electric Bicycle Americans Can Confide in 2024 - 3
Improving as a Pioneer: Examples from My Vocation - 4
I visited the largest collection of public telescopes in the US in Oregon's high desert, and the dark skies blew me away - 5
6 Monetary Arranging Administrations for Your Necessities
Many European nations want Israel to cancel 19 new settlement plans
Germany paves the way for tighter EU asylum rules
A trip to Colombia in my 20s turned into 8 years freelancing in South America. Here's what I'd do differently.
The most effective method to Perceive the Early Side effects of Cellular breakdown in the lungs
Bombardier Global 8000 Enters Service
When preventable infections turn deadly behind bars | The Excerpt
Germany expresses 'great concern' over Israel's new death penalty law
Figure out How to Adjust Your Handshake to Various Societies
German police 'cleared path for fascists with batons,' protesters say













