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.
Despite a negative review published by FDA’s internal scientists ahead of the meeting, a majority of the outside panelists said Amylyx had presented enough evidence to suggest the drug is helping patients live longer. The same group of neurology experts narrowly voted against the drug in March, due to concerns about missing data and other issues in the company’s study.
“To deprive ALS patients of a drug that might work, it’s probably not something I would feel terribly comfortable with,” said Dr. Liana Apostolova of Indiana University’s School of Medicine, who voted for approval. “At the previous meeting it wasn’t that clear and it’s still questionable.”
Amylyx also appeared to benefit from an unusual exchange in which a company executive — at the FDA’s request — committed to pull the drug from the market if its benefits aren’t confirmed by a large, ongoing study.
“I’m somewhat assured that if an approval is issued it can be withdrawn in the future,” Apostolova noted.
Wednesday’s vote concluded a rare second meeting to review several new statistical analyses submitted by Amylyx in support of the treatment’s benefit in slowing disease and extending life.
The ALS drug review is being closely watched as an indicator of FDA’s flexibility in reviewing experimental medications for the terminally ill and its ability to withstand outside pressure.
Dr. Billy Dunn, FDA’s neurology review chief, opened the meeting by detailing the “concerns and limitations” with Amylyx’s data, while emphasizing the need for new treatment options.
“We are highly sensitive to the urgent need for the development of new treatments for ALS,” Dunn said.
Dunn also noted that a larger Amylyx study being conducted in the U.S. and Europe could provide “more definitive results” by 2024.
In a highly unusual move, Dunn suggested the agency might be more willing to approve the drug if Amylyx would commit to withdrawing its medication if the ongoing 600-patient trial fails to show a…
Read More: FDA panel backs much-debated ALS drug in rare, 2nd review