Business
Amylyx Pharmaceuticals Announces FDA Advisory Committee Supports Approval of AMX0035 for the Treatment of ALS
- FDA Advisory Committee voted 7:2 that the available evidence of effectiveness is sufficient to support approval of AMX0035 for the treatment of ALS - If
About this update from Amylyx Pharmaceuticals, Inc.
[{"type":"text","content":"\n- FDA Advisory Committee voted 7:2 that the available evidence of effectiveness is sufficient to support approval of AMX0035 for the treatment of ALS\n\n- If approved, AMX0035 will be the first treatment in ALS that has demonstrated a significant slowing of disease progression and functional decline, as well as extended survival, in a randomized, placebo-controlled clinical trial, as a standalone therapy or when added to existing approved treatments\n\n- In December 2021, the FDA granted Priority Review and is expected to make a decision on AMX0035 by September 29, 2022, under the Prescription Drug User Fee Act\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nAmylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) announced today that the U.S. Food and Drug Administration’s (FDA) Peripheral and Central Nervous System Drugs Advisory Committee (PCNSDAC) voted (7 yes votes and 2 no votes) that the available evidence of effectiveness is sufficient to support approval of AMX0035 (sodium phenylbutyrate and taurursodiol [also known as ursodoxicoltaurine]) for the treatment of amyotrophic lateral sclerosis (ALS).\n\nThe PCNSDAC’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, PCNS meeting.\n\n“The Committee’s thoughtful review of the data and support of the benefit that AMX0035 may bring to the ALS community, if approved, is promising,” said Jamie Timmons, M.D., Head of Scientific Communications of Amylyx. “The CENTAUR trial data has consistently demonstrated potential benefits of AMX0035 on function and overall survival. We are grateful to the advocacy community, our trial participants and their family members, the ALS clinicians, and countless others who continue to support our mission of ensuring that people living with ALS around the world can access promising new therapies as quickly and efficiently as possible.”\n\nALS is a relentlessly 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.\n\nThe PCNSDAC recommendations, while not binding, will be considered by the FDA in its review of the ...