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A group of scientific advisors for the Food and Drug Administration is scheduled to review an experimental drug to treat amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease.
The research that led to the development of the drug, which was made by Amylyx Pharmaceuticals, was funded in part by donations raised by the Ice Bucket Challenge. The treatment is designed to slow the progression of the fatal neurodegenerative disease. The drugmaker said that the Phase 2 clinical trial involved 137 patients and that those who took the drug lived six months longer than those who were given a placebo.
On Monday (March 28), the FDA released a negative review of the drug. The agency said that the single study that Amylyx submitted was "not persuasive," citing several issues, including missing data and errors in enrolling patients.
The FDA's Peripheral and Central Nervous System Drugs Advisory Committee is expected to take a non-binding vote during their meeting on Wednesday. Even if the panel votes against approving the new drug, the FDA could overrule their decision.
The agency has faced immense pressure from ALS patients and advocacy groups to authorize the treatment, even if it only provides a minimal benefit. There is currently no cure for ALS.
"This is a uniformly fatal disease, so any drug that shows this benefit— and even if that benefit is modest— is fantastic for patients who have one path forward with this disease: death," Johns Hopkins University's Dr. Jeffrey Rothstein, who helped enroll patients in the Amylyx study, told the Associated Press.