Orphan Drug Designation Granted to ALS Treatment

The Food and Drug Administration (FDA) has granted Orphan Drug designation to AMX0035 (Amylyx Pharmaceuticals), an oral therapeutic in clinical development for the treatment of amyotrophic lateral sclerosis (ALS).

AMX0035 is a combination of sodium phenylbutyrate (PB) and tauroursodeoxycholic acid (TUDCA). The molecules have shown safety, tolerability, and preliminary signs of efficacy when tested separately in ALS clinical trials.  

A Phase 2 randomized, double-blind, placebo-controlled trial of AMX0035 titled CENTAUR (nct03127514) has begun with 132 ALS participants enrolled. The primary objectives of the trial are to assess safety and tolerability as well as the impact of the drug on disease progress as measured by the revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R).

The trial will last for 24 weeks with an open-label extension so that participants have the option to continue treatment. 

For more information visit Amylyx.com/trials.