
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
One month of war on Iran cost Arab countries up to $194bn: UNDP - 2
SUVs Known for Their Looks As opposed to Their Capacity - 3
The Main 20 Gaming Control center Ever - 4
Top 20 Wellbeing and Wellness Applications for a Sound Way of life - 5
McDonald's is bringing two 'KPop Demon Hunters' meals to McDonald's. Here's what they include and when they launch.
Find the Effect of Web-based Entertainment on Society: Exploring the Computerized Scene
Manual for Big name Work out schedules
Bold Colors, Playful Shapes, Handmade Textures: This Mexico City Home Is 100% Joyful!
The Quadrantid meteor shower peaks tonight, but will the full 'Wolf Moon' outshine the show?
The most effective method to Decisively Use Open Record Rewards
Ancient Pompeii construction site reveals the process for creating Roman concrete
Iran’s Crown Prince Reza Pahlavi backs protests: Join your fellow citizens in the streets
Warming winters lead to more nitrate pollution in the drinking water near farms
These Are the Journalists Israel Has Killed Since the Start of the Iran War













