FDA panel backs much-debated ALS drug in rare, 2nd review
A panel of federal health advisers has voted to recommend approval for an experimental drug to treat Lou Gehrig’s disease
WASHINGTON (AP) — A panel of federal health advisers voted Wednesday to recommend approval for an experimental drug to treat Lou Gehrig’s disease, a remarkable turnaround for the much-debated medication that was previously rejected by the same group earlier this year.
The Food and Drug Administration advisers voted 7-2 that data from Amylyx Pharma warranted approval, despite hours of debate about the strength and reliability of the company’s lone study. The FDA is not required to follow the group's advice, but its positive recommendation suggests an approval is likely later this month.
The FDA has approved only two therapies for the disease, amyotrophic lateral sclerosis, or ALS, which destroys nerve cells needed for basic functions like walking, talking and swallowing.
ALS patients and their families have rallied behind Amylyx’s drug, launching an aggressive lobbying campaign and enlisting members of Congress to push the FDA to grant approval.
