
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
The most effective method to Pick The Right Speakers - 2
Eco-Accommodating Kitchen Machines: 4 Picks for a Manageable Home - 3
FDA official discusses potential link between COVID-19 vaccines and pediatric deaths - 4
'The Secret Lives of Mormon Wives' Season 3 premieres tomorrow. Here's what you need to know to catch up. - 5
Figure out How to Store Your Gold Ventures: A Thorough Aide safely
CDC vaccine panel votes to remove universal hepatitis B birth dose recommendation
Step by step instructions to Appropriately Keep up with Your Sunlight powered chargers for Most extreme Productivity
A Republican elected governor in California? It's not as far-fetched as it sounds.
Surging measles cases are 'fire alarm' warning that other diseases could be next
Pick Your Favored kind of sandwich
What is colostrum? And should you be taking it?
10 Moves toward Start Your Own Effective Business
Remain Cool and Solid: Top Summer Food sources for 2024
Well known Travel Booking Locales: What's Your Pick?












