FDA experts ask to authorize an experimental drug for amyotrophic lateral sclerosis

By Matthew Perrone

The panel of independent advisors of the Food and Drug Administration (FDA, for its acronym in English) approved this Wednesday (by 7 votes in favor and 2 against) the recommendation of a new experimental drug to treat amyotrophic lateral sclerosis, also known as Lou Gehrig's disease.

The experts considered that the information provided by the pharmaceutical company Amylyx Pharma allowed its approval, after debating for hours the solidity and reliability of the only study presented.

The FDA is not required to follow his recommendation, but it may approve it later this month.

The agency has approved only two therapies for this disease that destroys nerve cells for basic functions such as walking, speaking and swallowing.

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ALS patients and their families mobilized in favor of Amylyx's drug with a lobbying campaign that reached members of Congress, who asked the FDA to grant approval.

A negative review from internal FDA scientists was published before the meeting, but most outside experts felt that Amylyx presented enough evidence to suggest

In March, the same group of FDA experts voted against the drug, concerned about lack of data and other problems.

“Depriving ALS patients of a drug that could work is not something I would feel comfortable with,” said Dr. Liana Apostolova of Indiana University, who voted in favor, “at the previous meeting it was not so clear And it's still questionable."

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Amylyx benefited from a rare trade in which one of the drug's makers has pledged – at the FDA's request – to withdraw it from the market if its benefits are not confirmed by a large ongoing study.

"I am certain that if an approval is issued, it can be withdrawn in the future," Apostolova said.

Wednesday's vote ended a second meeting reviewing new statistical analyzes submitted by Amylyx to support the benefits of the treatment in curbing the disease and extending the lives of patients.

Dr. Billy Dunn, the FDA's neurology review chief, opened the meeting by detailing "concerns and limitations" with the Amylyx data, while emphasizing the need for new treatment options.

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"

", said Dunn who pointed out that a larger study of Amylyx, which is being carried out with 600 patients in the United States and Europe, could provide "more definitive results" in 2024.

Dunn suggested that the agency might be more willing to approve it if Amylyx agreed to withdraw it if the study shows no benefit, and called on the company's co-founders to take that step publicly.

Justin Klee, co-head of Amylyx, said yes, they will voluntarily withdraw their drug in that case.

I am certain that if an approval is issued, it can be withdrawn in the future.”

Liana Apostolova doctor

The FDA can force companies to remove drugs from the market, although it is usually faster if the manufacturers do so.

When they resist, the regulation process can take years.

“I think the FDA – with all due respect – underestimates the complexity and likelihood of removing the product from the market,” said Dr. Caleb Alexander of Johns Hopkins University, one of two panelists who voted against it.

In a small trial, riddled with incomplete data and other problems, Amylyx showed some benefit in slowing disease progression, according to FDA reviewers.

“The end result – for a single study –

,” said Tristan Massie, an FDA statistician.

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The Cambridge, Massachusetts-based company insists that follow-up data collected after the study's conclusion showed that the drug prolonged patients' lives and that those who continued to take it

Pro-drug experts cited this data and its mild side effects to suggest there would be few downsides.

I ask you to approve it because I know it works.

It's extending my life and I want that for others."

Greg Canter Patient with her

"The drug is not harmful, it appears to have a benefit, but there is no safety signal," said Dean Follmann, a statistician at the National Institutes of Health.

On Wednesday, more than 20 researchers, patients and family members told aides they supported approval.

The agency also received more than 1,200 written comments, the majority from patient advocates.

“I ask you to approve it because I know it works.

It is extending my life and I want that for others,” said Greg Canter, diagnosed with ALS in 2018 and part of the Amylyx study.

He said the drug has improved his lung capacity and slowed his functional decline.

The drug comes as a powder that combines two ancient drugs: one prescribed for liver disorders and a dietary supplement used in traditional Chinese medicine.