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• Posted September 6, 2022

FDA Panel Skeptical of Controversial ALS Drug Ahead of Vote

A U.S. Food and Drug Administration panel will once again consider approval for an experimental drug for ALS, a rare second review for a disease that has no cure.

The same panel that will meet Wednesday voted last March not to approve the drug for the deadly neurodegenerative condition that's also known as Lou Gehrig's disease.

But getting the drug, known as Albrioza (AMX0035), approved is a rallying cause for patients, their families and members of Congress, the Associated Press reported.

Still, federal regulators said in a briefing document filed on Friday that the company's new evidence was not "sufficiently independent or persuasive"to establish effectiveness.

The document did say that the experts can consider "the unmet need in ALS"and the flexibility the agency has in applying for approval of drugs that treat deadly diseases.

This suggests "there is a chance that the FDA is still looking for a way to approve the product,"SVB analyst Marc Goodman wrote in a note to investors. Goodman thinks the drug has about a 50% chance of approval, the AP reported.

When regulators first reviewed the drug in March, they voted 6-4 against it after finding data wasn't convincing that it would benefit those with the disease. The panel 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 puts 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 Pharmaceuticals, which makes the drug, said it has gathered follow-up data on the study that concluded that the drug extended the life of ALS patients by about 10 months.

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, according to Amylyx.

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

But the trial had missing data and implementation errors, according to FDA reviewers. It still "suffers from the same interpretability challenges,"they wrote.

Patients who have ALS eventually stop being able to walk, talk, swallow and breathe, typically dying within three to five years of diagnosis, as the disease methodically destroys nerve cells.

External input will come 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."

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