FDA Panel Skeptical of Controversial ALS Drug Ahead of Vote

TUESDAY, Sept. 6, 2022 (HealthDay News) — A U.S. Food and Drug Administration panel will once again consider approval for an experimental drug for amyotrophic lateral sclerosis (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. 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 fatal 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 percent chance of approval, the AP reported.

When regulators first reviewed the drug in March, they voted 6-4 against it after finding no convincing data 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.

Associated Press Article