Amylyx Pharmaceuticals (Nasdaq:AML) has received Health Canada approval for Albrioza (AMX0035).
The FDA is anticipated to decide on Albrioza, which consists of sodium phenylbutyrate and ursodoxicoltaurine, by September 29, 2022.
Amylyx is the first new therapy for ALS to win approval in Canada since 2018.
The approval also is the first global regulatory approval for the drug.
Health Canada decided to greenlight the drug after reviewing clinical trial data showing that it slowed disease progression and loss of functional decline in ALS patients.
“We are excited with Health Canada’s decision to approve Albrioza with conditions. Albrioza is a therapy that demonstrated in our CENTAUR trial a statistically significant and clinically meaningful impact on function, alone or in addition to existing ALS therapies,” said Justin Klee and Joshua Cohen, co-CEOs and co-founders of Amylyx, in prepared remarks. “We are grateful to the people who participated in our clinical trials, their families, the researchers, and the ALS community for helping to make this milestone happen.”
The Phase 2 CENTAUR study enrolled 137 ALS patients, including a 6-month randomized placebo-controlled phase and an open-label long-term follow-up phase.
Patients who received the drug had an average score on the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) that was 2.32 points higher at 24 weeks.
Several companies are developing drugs for ALS, including the privately-held biotech QurAlis with its first-in-class molecule QRL-201 and Irvine, Calif.-based Eledon Pharmaceuticals. The latter recently announced data from a Phase 2a study of tegoprubart, which reduced inflammatory biomarkers associated with the progression of amyotrophic lateral sclerosis (ALS).