(Reuters) -The U.S. health regulator’s advisers on Wednesday voted against BrainStorm Cell Therapeutics’ therapy designed to treat patients with amyotrophic lateral sclerosis (ALS), a potentially fatal neurodegenerative disease.
While 17 panel members voted that the data presented does not demonstrate substantial evidence of the effectiveness of the therapy, NurOwn, for the treatment of mild-to-moderate ALS, 1 voted for its use and 1 panel member abstained.
The panel’s decision follows years of back-and-forth between the agency and the company on the clinical benefit of NurOwn after the therapy failed to meet the main goal in a late-stage study in 2020.
Brainstorm has said data from new analyses showed that the therapy was beneficial in treating a smaller subset of patients with ALS and reduced markers of nerve degeneration.
The U.S. Food and Drug Administration’s (FDA) staff reviewers also said on Monday there is not enough evidence to support NurOwn’s effectiveness, that there are large amounts of missing data in the company’s application, and pointed to substantial concerns over the product’s manufacturing.
The FDA generally follows the advise of its panel, but is not bound to do so. The agency is expected to make a decision on the drug by Dec. 8.
ALS, also known as Lou Gehrig’s disease, is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord.
ALS affects about 20,000 U.S. patients, and most patients die within 3 to 5 years after the onset of symptoms, according to government data.
(Reporting by Mariam Sunny in Bengaluru; Editing by Shinjini Ganguli, Shounak Dasgupta and Shweta Agarwal)
