Therapy Areas: Central Nervous System
US FDA Plans to Reconvene Advisory Committee to Review Amylyx Pharmaceuticals' AMX0035 NDA for the Treatment of ALS
5 July 2022 - - The US Food and Drug Administration has informed US-based Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) that the Agency is planning to reconvene the Peripheral and Central Nervous System Drugs Advisory Committee (PCNSDAC) to discuss the New Drug Application for AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO; also known as ursodoxicoltaurine]) for the treatment of amyotrophic lateral sclerosis on Wednesday, September 7, 2022, the company said.

The FDA will formally announce the scheduling of the planned PCNSDAC meeting in the Federal Register.

Discussions will focus on the additional analyses of data from the company's clinical studies that were determined by the FDA to constitute a major amendment to the NDA.

The Prescription Drug User Fee Act (PDUFA) target action date for the AMX0035 NDA is September 29, 2022, which was extended from June 29, 2022, to allow more time for the FDA to review additional analyses of data from the company's clinical studies.

The PCNSDAC previously met on March 30, 2022, to discuss the NDA for AMX0035 for the treatment of ALS.

AMX0035 (sodium phenylbutyrate and taurursodiol) is an oral fixed-dose medication with marketing applications pending in the United States and European Union, and approved with conditions as Albrioza to treat amyotrophic lateral sclerosis in Canada.

The combination of sodium phenylbutyrate and taurursodiol may reduce neuronal cell death, hypothesized to occur by simultaneously mitigating endoplasmic reticulum stress and mitochondrial dysfunction.

AMX0035 is also being explored for the potential treatment of other neurodegenerative diseases.

Amylyx is headquartered in Cambridge, Massachusetts and has operations in Canada and EMEA.


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