Surprise FDA AdCom backing for approval of AMX0035 as ALS therapy

Shares of Amylyx Pharmaceuticals (Nasdaq: AMLX) rocketed up 74% in pre-market trading today on positive news from a regulatory advisory committee on its amyotrophic lateral sclerosis (ALS) drug candidate, having suffered earlier following the release of downbeat briefing papers from the US Food and Drug Administration’s (FDA) last Friday.

The FDA’s 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 Amylyx’ AMX0035 (sodium phenylbutyrate and taurursodiol [also known as ursodoxicoltaurine]) for the treatment of amyotrophic lateral sclerosis (ALS).

Reportedly, panel members agreed that the data could be more robust, but some changed their votes after seeing additional post-hoc analyses from Amylyx and hearing testimonies from clinicians and patients, a majority of whom advocated for AMX0035’s approval.