FDA Panel Backs Much-Debated ALS Drug in Rare, 2nd Review

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.

Patients support drug

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, because of 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.

The FDA has the power to force companies to pull drugs from the market, though it’s generally faster if drugmakers voluntarily take that step. In cases where companies resist removal the regulatory process can drag on for years.

New data prompts second look

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.

Amylyx conducted one small, mid-stage trial of its drug that showed some benefit in slowing the disease, but it was plagued by missing data and other problems, according to FDA reviewers.

“The final result — for a single study — is borderline and not very statistically persuasive,” FDA statistician Tristan Massie told panelists.

Company says data shows drug extends life

The Cambridge, Massachusetts, company says follow-up data gathered after the study concluded showed the drug extended life. Patients who continued taking the drug survived about 10 months longer than patients who never took the drug, according to a new company analysis.

Panelists favoring the drug cited that data, along with the drug’s mild side effects, to suggest there would be little downside for patients even if it doesn’t ultimately slow ALS.

Hanging over the review is FDA’s controversial approval of the Alzheimer’s drug Aduhelm last year, which was reviewed by the same agency scientists and outside advisers.

In that case, the FDA disregarded the overwhelmingly negative vote by its outside advisers, three of whom resigned over the decision. The agency’s approval — which followed irregular meetings with drugmaker Biogen — is under investigation by Congress and federal inspectors.

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