Second time's a charm for Amylyx Pharmaceuticals who announced that an FDA Advisory Committee, in a rare second meeting, has now recommended approval of the drugmaker's amyotrophic lateral sclerosis (ALS) candidate.
Yesterday, the FDA's Peripheral and Central Nervous System Drugs Advisory Committee voted 7-2 that the available evidence of effectiveness is sufficient to support approval of AMX0035. 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.
The panel had previously met in March to discuss Amylyx's NDA for the drug, which is an oral fixed-dose combination of sodium phenylbutyrate and taurursodiol. The panel found that data from Amylyx’s initial trial was insufficient to prove that the drug slowed ALS progression, and voted 4-6 against the approval. After the FDA determined that additional analyses from clinical studies constitute major amendments to the application, a second AdCom meeting was scheduled for Sept.
The Massachusetts-based pharma company was founded to try to take a new approach to ALS, focusing on the stress pathways in the endoplasmic reticulum and mitochondria that lead to the degeneration and death of neurons. Now, with a PDUFA date of Sept. 29, it may get that opportunity.