Canada Approves Amylyx’s ALS Drug Albrioza

Health Canada has approved Amylyx Pharmaceuticals’ Albrioza (sodium phenylbutyrate and ursodoxicoltaurine) for treatment of patients with the rare progressive nervous system disease amyotrophic lateral sclerosis (ALS) disease, also known as Lou Gehrig's disease.

The conditional approval requires the company to submit data from an ongoing phase 3 clinical trial and from additional studies to support a full approval of the oral drug.

The conditional approval was based on results of a phase 2 study in 137 participants. Patients who received Albrioza demonstrated a 2.32-point higher average score on the ALS Functional Rating Scale compared with those given a placebo.

In March, the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee voted not to recommend the drug for FDA approval. The agency is still reviewing the company’s application and has  a user fee decision date of Sept. 29.