News and media

Health Canada has agreed to review a New Drug Submission (NDS) for Tofersen, for the treatment of amyotrophic lateral sclerosis (ALS) in adults with a mutation in the superoxide dismutase 1 (SOD1) gene. A regulatory decision on the New Drug Submission is expected in early 2025. If approved, this therapy will become the first treatment in Canada to target a genetic cause of ALS. Read the...

Today, Amylyx announced that their Phase 3 PHOENIX trial, a global, 48-week, randomized, placebo-controlled study of AMX0035 (sodium phenylbutyrate and ursodoxicoltaurine; also known as ALBRIOZA) in people living with ALS, did not meet its prespecified primary or secondary endpoints. It's understandable if this outcome has left us all feeling disheartened and discouraged. Now, more than ever,...