2022-06-14| Trials & Approvals

Canada Approves Amylyx’s Experimental ALS Drug

by Fujie Tham
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Massachusetts, US-based Amylyx Pharmaceuticals today announced that Canada has approved its Albrioza (sodium phenylbutyrate + ursodoxicoltaurine) for treatment of amyotrophic lateral sclerosis (ALS). Health Canada is the world’s first to approve the therapeutic while the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) are still evaluating Amylyx’s submission.

AMX0035, or Albrioza in Canada, is approved conditionally under Health Canada’s Notice of Compliance with Conditions policy, one of the conditions is the data from the ongoing Phase 3 PHOENIX trial. Amylyx says the topline data is expected in 2024. 

While in the US, the FDA is scheduled to make its decision later this year, after giving a negative comment on AMX0035’s Phase 2 CENTAUR trial results. An FDA committee review found the drug to be safe, but argued against its effectiveness due to insufficient data.

Related article: Amylyx’s New ALS Drug Review Impeded as FDA Members Disagreed with Clinical Trial Results 


ALS, a Progressive and Fatal Neurodegenerative Disorder


Amyotrophic lateral sclerosis, or ALS, is a progressive nervous system disease that affects nerve cells in the brain and spinal cord, leading to the loss of muscle control. ALS often starts in the hands, feet or limbs, and then spreads to other body parts. As the disease develops and nerve cells are destroyed, muscles get weaker. This eventually affects chewing, swallowing, speaking, and breathing. 

ALS is diagnosed in more than 6,000 people worldwide each year and often causes death in two to five years. While treatments like riluzole, which can extend survival by several months, and edaravone, which slows progression, there is no cure for the disease as of now.

Amylyx’s AMX0035 is an oral fixed-dose medication consisting of sodium phenylbutyrate and ursodoxicoltaurine, proposed to reduce neuronal death by simultaneously mitigating endoplasmic reticulum stress and mitochondrial dysfunction. Too little is known about the underlying biological mechanisms of ALS and how AMX0035 addresses the disease to warrant an FDA accelerated approval.

“For more than 30 years, I have been involved with caring for people living with ALS, and we have had major breakthroughs in research and made strides in drug development. The approval of ALBRIOZA is a testament to this progress,” said Angela Genge, MD, Director of the ALS Global Centre for Excellence at the Montreal Neurological Institute. “New treatment options that can slow disease progression are critical to give people living with ALS more time as we work toward a cure.”

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