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

A panel at the Food and Drug Administration has recommended approval for an experimental drug for amyotrophic lateral sclerosis (ALS).

HealthDay News — In a rare second review, a U.S. Food and Drug Administration panel on Wednesday recommended approval for an experimental drug for amyotrophic lateral sclerosis (ALS). The FDA is not obligated to follow its advisers’ 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 fatal neurodegenerative condition.

Getting the drug approved has become a rallying cause for patients, their families, and members of Congress, the Associated Press reported. They have contended that enough evidence exists to support the safety and effectiveness of Albrioza 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.

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.

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.

Associated Press Article