US FDA to review Amylyx new drug application for AMX0035

AMX0035 is an investigational therapy for the treatment of amyotrophic lateral sclerosis (ALS).

Amylyx Pharmaceuticals announced that a virtual meeting of the U.S. Food and Drug Administration’s (FDA) Peripheral and Central Nervous System Drugs Advisory Committee to review the New Drug Application (NDA) for AMX0035 (sodium phenylbutyrate and taurursodiol also known as ursodoxicoltaurine]) has been scheduled for March 30, 2022.

AMX0035 is an investigational therapy for the treatment of amyotrophic lateral sclerosis (ALS). As previously reported, the U.S. FDA has granted Priority Review and assigned a Prescription Drug User Fee Act date for AMX0035 of June 29, 2022.

“There are few treatments approved for ALS, a devastating disease that impacts a person’s ability to move, speak, eat and breathe,” said Lahar Mehta, Head of Global Clinical Development of Amylyx. “We look forward to a robust scientific discussion with the members of the advisory committee panel regarding the clinical data submitted to support our New Drug Application for AMX0035.”

AMX0035 is a proprietary oral fixed-dose combination of two small molecules: sodium phenylbutyrate (PB) and taurursodiol, which is a Bax inhibitor designed to reduce cell death through apoptosis.