FDA panel votes against controversial ALS drug candidate

FDA advisers on Sept. 27 voted against recommending an experimental treatment for Lou Gehrig's disease. 

In a vote, 17 members on the Cellular, Tissue and Gene Therapies Advisory Committee said Brainstorm's NurOwn did not show efficacy in treating mild to moderate amyotrophic lateral sclerosis. One voted yes, and another abstained. 

The company's study of 200 patients failed to find that NurOwn improved patient mobility, extended life or slowed the disease that affects about 31,000 people in the U.S., according to the CDC. Despite the lacking efficacy data, the FDA organized the committee meeting after receiving a petition with about 30,000 signatures from ALS patients and advocates. 

In the past year, the FDA has approved two ALS drugs, Amylyx Pharmaceuticals' Relyvrio and Biogen's Qalsody, after about 20 years without a new ALS drug, according to CBS News

The FDA does not have to follow the committee's vote, but the agency usually does. The final decision is expected by Dec. 8. 

Copyright © 2024 Becker's Healthcare. All Rights Reserved. Privacy Policy. Cookie Policy. Linking and Reprinting Policy.

 

Featured Whitepapers

Featured Webinars

>