Amylyx Pharmaceuticals (AMLX) Announces FDA Plan to Reconvene Advisory Committee to Review AMX0035 NDA for the Treatment of ALS on September 7

Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today announced that the U.S. Food and Drug Administration (“FDA” or the “Agency”) has informed the Company that the Agency is planning to reconvene the Peripheral and Central Nervous System Drugs Advisory Committee (PCNSDAC) to discuss the New Drug Application (NDA) for AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO; also known as ursodoxicoltaurine]) for the treatment of amyotrophic lateral sclerosis (ALS) on Wednesday, September 7, 2022. 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.

“We remain engaged with the FDA to advance AMX0035 through the review process as efficiently as possible,” said Tammy Sarnelli, Global Head of Regulatory Affairs of Amylyx. “We are pleased that the members of the advisory panel will review additional analyses from our clinical studies, including recently published analyses, supporting the previously reported functional and overall survival benefit for AMX0035. As we have heard from the ALS community, there is a crucial need for new and effective treatments in ALS, and our team will continue to work around the clock to advance treatments for ALS in the U.S.”

About AMX0035

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

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