ALBRIOZA (AMX0035)

On April 4, 2024, Amylyx Pharmaceuticals announced that they had started a process with Health Canada to discontinue the marketing authorization for ALBRIOZA. This means that ALBRIOZA will no longer be available for new patients in Canada. People currently on the therapy in Canada who, in consultation with their physician, wish to stay on treatment can be transitioned to a free drug program.

ALBRIOZA was approved by Health Canada under the Notice of Compliance with Conditions (NOC/c) pathway in June 2022. Amylyx’s decision to remove ALBRIOZA from the market is based on the recent PHOENIX trial results of AMX0035, marketed as ALBRIOZA in Canada and RELYVRIO in the United States, which failed to show an effect on primary and secondary outcome measures.

ALBRIOZA (AMX0035) is a combination of two compounds (sodium phenylbutyrate and ursodoxicoltaurine) in the form of a powder dissolved in liquid. It was approved for use in Canada based on Phase 2 clinical trial results that demonstrated slowing disease progression and lifespan extension in treated participants.

However, results from the Phase 3 PHOENIX clinical trial did not show changes in survival or a slowing of the disease’s progression rate.

Soon after the pCPA negotiations concluded, our community used the e-advocacy tool developed by ALS Canada and emailed the Minister of Health in their province, asking that ALBRIOZA be made accessible through public reimbursement without further delay. Over 800 Canadians sent letters as part of the campaign and utilized our “Advocacy in a Box” toolkit to approach the government in your province and call for Health Canada-approved treatments to be reimbursed and available to Canadians living with ALS in a timeframe that more accurately reflects the urgency of loss faced by this community.

In October 2021, CADTH, the agency responsible for provincial reimbursement recommendations, posted a call for patient input on AMX0035. ALS Canada invited the community to contribute to the submission by sharing their experiences through a survey. More than 600 Canadians participated in the survey, the results of which informed the patient input submission. In July 2022, ALS Canada provided feedback as a patient group in response to CADTH’s reimbursement recommendations for ALBRIOZA to help ensure coverage criteria reflect the realities of the diagnosis and treatment of ALS.

Our community was disappointed that Health Canada did not grant ALBRIOZA a priority review. We raised our voices to highlight access to therapies as an urgent issue for people living with ALS and advocated for priority review status for all innovative ALS therapies. We remained engaged with Health Canada and the Federal Minister of Health to advocate for this vital issue.

ALS Canada also provided resources for the ALS community to use their social media channels to make their voices heard. Our community raised its voices on social media and told the federal government that all ALS therapies must be granted priority review status.

Within The Time is Now position paper, we propose that ALS treatments be reviewed for approval and public reimbursement through a single condensed timeframe. This pathway includes Health Canada’s priority review for all ALS therapies.