In Reversal, Amylyx ALS Drug Receives FDA Advisory Committee Support

Amylyx Pharmaceuticals announced today that the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee voted 7 to 2 that the available evidence of effectiveness is sufficient to support approval of the drug the company has developed for the treatment of ALS (amyotrophic lateral sclerosis), also known as Lou Gehrig’s disease. The committee’s decision was based on a review of all available evidence, including new analyses submitted for discussion at the September 7 meeting and the information presented at the March 30, 2022 advisory committee meeting. The committee had voted 6 to 2 against approval at the earlier meeting.

The advisory committee’s recommendations, while not binding, will be considered by the FDA in its review of the pending New Drug Application (NDA) for AMX0035. As previously reported, the Prescription Drug User Fee Act target action date for the NDA is September 29, 2022, which was extended by the FDA to allow more time to review additional analyses of data from clinical studies.

ALS is a progressive and fatal neurodegenerative disorder caused by motor neuron death in the brain and spinal cord. Motor neuron loss in ALS leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis, and eventually, death. The company’s drug, a combination of sodium phenylbutyrate and taurursodiol is an oral fixed-dose medication with marketing applications pending in the US and EU and approved with conditions to treat ALS in Canada. The combination of sodium phenylbutyrate and taurursodiol may reduce neuronal cell death, hypothesized to occur by simultaneously mitigating endoplasmic reticulum (ER) stress and mitochondrial dysfunction. It is also being explored for the potential treatment of other neurodegenerative diseases. (Source: Amylyx Pharmaceuticals Website, 07 September, 2022)