ALS drug Albrioza to be voluntarily withdrawn from the market in Canada

Albrioza (sodium phenylbutyrate and ursodoxicoltaurine powder for suspension) was originally approved by Health Canada on June 1, 2022 for the treatment of patients with amyotrophic lateral sclerosis (ALS). At that time, the drug was granted marketing authorization with conditions (NOC/c), based on results from a Phase 2 clinical study and pending the results of a trial to verify the drug’s clinical benefit.1,2In March 2024, manufacturer Amylyx Pharmaceuticals announced that the PHOENIX phase 3 clinical trial of Albrioza (also known as MX0035) did not meet its primary or secondary efficacy endpoints. The 48-week study involved participants from the United States and Europe. It revealed no significant benefit from Albrioza compared to placebo, as measured by changes in the ALS Functional Rating Scale-Revised score. The drug was well-tolerated, with no new safety issues identified. However, there was no evidence that the drug slowed disease progression.3,4 Amylyx plans to publish the full results of the trial in a medical journal later this year.3In April 2024, Amylyx Pharmaceuticals announced that the company had started a process with Health Canada to voluntarily discontinue the marketing of Albrioza in Canada (and in the United States, brand name Relyvrio).5,6 Read: QUIZ: Test your knowledge of ALSThe current product monograph for Albrioza (updated May 31, 2024) states that the clinical benefit of the drug for ALS remains unconfirmed. Therefore, new patients must not be started on the drug. Albrioza is currently only available through an Amylyx Pharmaceuticals patient support program (1-877-710-0711 or support@amylyxcareteam.ca) to patients already receiving treatment with the drug.1,2 Healthcare professionals should discuss with their patients whether to continue treatment with Albrioza or switch to an alternate drug.2The manufacturer plans to withdraw Albrioza from the Canadian market no later than December 31, 2024.1 Two other disease-modifying therapies are approved for ALS treatment in Canada—riluzole (Rilutek, generics) and edaravone (Radicava). More details regarding ALS treatment can be found in the Canadian best practice recommendations for the management of ALS.7 Read: ALS drug wil be pulled from U.S. market after study showed patients didn’t benefit References1. Amylyx Pharmaceuticals Inc. Albrioza (sodium phenylbutyrate and ursodoxicoltaurine powder for suspension) product monograph. Cambridge, MA; May 31, 2024. https://pdf.hres.ca/dpd_pm/00075822.PDF(accessed June 11, 2024).2. Government of Canada. Health professional risk communication. Important safety and efficacy information on Albrioza (sodium phenylbutyrate and ursodoxicoltaurine) – market withdrawal and continued restricted access. May 7, 2024. https://recalls-rappels.canada.ca/en/alert-recall/important-safety-and-efficacy-information-albrioza-sodium-phenylbutyrate-and (accessed June 11, 2024). 3. Amylyx Pharmaceuticals Inc. Amylyx Pharmaceuticals announces topline results from global phase 3 PHOENIX trial of AMX0035 in ALS. March 08, 2024. https://www.amylyx.com/news/amylyx-pharmaceuticals-announces-topline-results-from-global-phase-3-phoenix-trial-of-amx0035-in-als (accessed June 11, 2024). 4. ALS Canada. PHOENIX Trial of AMX0035 did not meet endpoints. March 8, 2024. https://als.ca/media-room/phoenix-trial-of-amx0035-did-not-meet-endpoints/ (accessed June 11, 2024).5. Amylyx Pharmaceuticals Inc. Amylyx Pharmaceuticals announces formal intention to remove Relyvrio/Albrioza from the market; provides updates on access to therapy, pipeline, corporate restructuring, and strategy. April 04, 2024. https://www.amylyx.com/news/amylyx-pharmaceuticals-announces-formal-intention-to-remove-relyvrior/albriozatm-from-the-market-provides-updates-on-access-to-therapy-pipeline-corporate-restructuring-and-strategy (accessed June 11, 2024). 6. ALS Canada. Amylyx announces formal intention to remove ALBRIOZA from the market. April 4, 2024. https://als.ca/media-room/amylyx-announces-formal-intention-to-remove-albrioza-from-the-market/ (accessed June 11, 2024).7. Shoesmith C, Abrahao A, Benstead T, et al. Canadian best practice recommendations for the management of amyotrophic lateral sclerosis. CMAJ 2020;192:E1453-68. https://www.cmaj.ca/content/cmaj/192/46/E1453.full.pdf (accessed June 11, 2024).

ALS drug Albrioza to be voluntarily withdrawn from the market in Canada

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