FDA extends PDUFA date for AMX 0035 a proposed treatment for amyotrophic lateral sclerosis .- Amylyx Pharma
Amylyx Pharmaceuticals announced that the FDA has extended the review timeline of the New Drug Application (NDA) for AMX 0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO; also known as ursodoxicoltaurine]) for the treatment of amyotrophic lateral sclerosis (ALS).
The updated Prescription Drug User Fee Act (PDUFA) goal date is September 29, 2022.
AMX 0035 is a proprietary oral fixed-dose combination of two small molecules: sodium phenylbutyrate (PB), which is a small molecular chaperone designed to reduce the unfolded protein response (UPR), preventing cell death resulting from the UPR, and taurursodiol (TURSO; also known as ursodoxicoltaurine), which is a Bax inhibitor designed to reduce cell death through apoptosis. PB and TURSO were combined in a fixed-dose formulation in an effort to reduce neuronal death and dysfunction. AMX 0035 is designed to target the endoplasmic reticulum and mitochondrial-dependent neuronal degeneration pathways in ALS and other neurodegenerative diseases.
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