FDA Advisory Committee supports approval of AMX 0035 for the treatment of amyotrophic lateral sclerosis
Amylyx Pharmaceuticals, Inc. announced that the 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 AMX 0035 (sodium phenylbutyrate and taurursodiol [also known as ursodoxicoltaurine]) for the treatment of amyotrophic lateral sclerosis (ALS)
The 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.
ALS 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.
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