• Posted September 8, 2022

In Rare Move, FDA Panel Gives Support to Controversial ALS Drug in 2nd Review

In a rare second review, a U.S. Food and Drug Administration panel on Wednesday recommended approval for an experimental drug for ALS (amyotrophic lateral sclerosis).

The FDA is not obligated to follow its advisors' recommendations, though it usually does.

Wednesday's vote was 7-2 for approval. The same panel voted 6-4 last March not to approve the drug, called Albrioza (AMX0035), for the deadly neurodegenerative condition that's also known as Lou Gehrig's disease.

Getting the drug approved has become a rallying cause for patients, their families and members of Congress, the Associated Press reported. They have contend that enough evidence exists to support Albrioza's safety and effectiveness in patients with a disease that has no cure.

In the second review -- convened after a massive lobbying effort by the drug's supporters -- members of the FDA panel debated for hours, the AP said. The panelists' main focus was the strength and reliability of the one study conducted by the drug's maker, Amylyx Pharmaceuticals Inc.

In the end, a majority of panelists backed the medication.

"To deprive ALS patients of a drug that might work, it's probably not something I would feel terribly comfortable with,"Dr. Liana Apostolova of Indiana University's School of Medicine, who voted for approval, told the AP. "At the previous meeting, it wasn't that clear and it's still questionable."

One new factor encouraging a "yes" vote from panelists may have been a deal struck between Amylyx and the FDA to remove Albrioza from the market if its effectiveness is not borne out in a new large, ongoing study. Amylyx co-CEO Justin Klee said the company would voluntarily withdraw its drug should that come to pass.

"I'm somewhat assured that if an approval is issued it can be withdrawn in the future,"Apostolova said.

At the opening of the Wednesday meeting, Dr. Billy Dunn, chief of neurology review at the FDA, said the data supplied by Amylyx still raised "concerns and limitations." But he also said that "we are highly sensitive to the urgent need for the development of new treatments for ALS."

There are currently only two FDA-approved medications for ALS, which kills nerve cells and slowly robs patients of the ability to walk, talk or even swallow. Most people with ALS die within 3 to 5 years of diagnosis, usually from respiratory failure.

When regulators first reviewed the drug in March, they voted against it after finding data wasn't convincing that it would benefit those with the disease. The panel then gave the agency until Sept. 29 to review any further data the company submitted.

Canadian regulators have already approved the drug for ALS patients, which put the FDA in a "precarious position,"bioethicist Holly Fernandez-Lynch told the AP.

"They typically like to be out ahead when making approval decisions,"said Fernandez-Lynch, who teaches at the University of Pennsylvania. "They like to make the argument that they are not a barrier to patients accessing things that might help them."

Amylyx said it had gathered follow-up data on the study that concluded that the drug extended the life of ALS patients by about 10 months. Albrioza also appears to have only mild side effects.

The medication combines a dietary supplement used in traditional Chinese medicine with an existing prescription drug for liver disorders. The combination shields cells from premature death, Amylyx contends.

Approval of the combined medication could require insurers to cover it.

Two panelists still voted against approval. One of them, Dr. Caleb Alexander of Johns Hopkins University, said he wasn't persuaded by Amylyx's promise to pull Albrioza from the market if it failed in the new trial.

"I think the FDA -- with all due respect -- significantly understates the complexity and likelihood of their pulling the product from the market,"Alexander told the AP.

External input came from patient and advocacy groups, including I AM ALS, founded by patient Brian Wallach. Wallach takes the dietary supplement piece of the treatment.

"Patients do their homework -- we know this isn't going to cure us,"Wallach, who was diagnosed with ALS in 2017 and spoke through an interpreter, told the AP. "But we also know it might keep us here until the next drug comes along and that one might be a cure."

Greg Canter was diagnosed with ALS in 2018 and participated in Amylyx's study. "I'm asking you to approve it because I know it works. It's extending my life and I want that for others,"he said. Canter credits the drug with improving his lung capacity and slowing his functional decline.

More information

The U.S. National Institute of Neurological Disorders and Stroke has more on ALS.

SOURCE: U.S. Food and Drug Administration, briefing document, Sept. 2, 2022; Associated Press