FDA advisory committee to review Amylyx ALS drug again

July 6, 2022

The FDA will reconvene its Peripheral and Central Nervous System Drugs Advisory Committee (PCNSDAC) to discuss Amylyx’s ALS drug candidate later this year, the company announced this week. 

The advisory board had previously met in March to discuss the New Drug Application (NDA) for AMX0035. After the FDA determined that additional analyses from clinical studies constitute major amendments to the application, the drug will be evaluated again for the treatment of amyotrophic lateral sclerosis in September of this year.   

AMX0035 is an oral-fixed dose medication currently approved with conditions under the brand name Albrioza to treat ALS in Canada. The drug works by combining sodium phenylbutyrate and taurursodiol which are believed to reduce neuronal cell death by mitigating stress and dysfunction of crucial organelles, or the cell’s organs.

 “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,” said Tammy Sarnelli, Global Head of Regulatory Affairs of Amylyx. “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.”