Summary of Cancellation for Albrioza
Review decision
The Regulatory Decision Summary explains Health Canada’s decision for the product seeking market authorization. The Regulatory Decision Summary includes the purpose of the submission and the reason for the decision.
Product type:
Medicinal ingredient(s):
Sodium phenylbutyrate/ursodoxicoltaurine
Therapeutic area:
Other Nervous System Drugs
Type of submission:
Supplement to a New Drug Submission
Control number:
280378
Decision issued:
No decision was issued by Health Canada. The sponsor (Amylyx Pharmaceuticals) cancelled its submission before a final decision was issued.
Date of cancellation:
2024-05-02
What was the purpose of this submission?
The purpose of this submission was to fulfill a commitment outlined in the Qualifying Notice for Albrioza (sodium phenylbutyrate and ursodoxicoltaurine), by providing the study report for study A35-005:
An evaluation of multiple dose pharmacokinetics of phenylbutyrate (PB) and taurursodiol (TURSO) (in proprietary combination of Albrioza), as well as surrogate pharmacodynamic (PD) activity via measurement of Histone 3 and Histone 4 acetylation levels.
What did the company submit to supports its submission?
The sponsor submitted the study report for study A35-005, which comprised pharmacokinetic and pharmacodynamic data.
What was the status of the submission when it was cancelled? What was Health Canada’s assessment of the submission at the time of cancellation?
On March 8, 2024, the sponsor announced publicly the topline results from their Phase 3 clinical confirmatory trial of sodium phenylbutyrate and ursodoxicoltaurine, (PHOENIX) and indicated that the study did not meet its primary or secondary endpoints.
Review of this submission was paused and discussions (internal to Health Canada and with Amylyx) on how to proceed in light of these findings were initiated.
As a result of discussions between the sponsor and Health Canada, the sponsor agreed to initiate a sunset program during which patients currently taking Albrioza were permitted to continue if they wished, no new patients were to be started on Albrioza, and the drug would be voluntarily withdrawn from the market. The details were communicated to stakeholders through a Product Monograph revision, a Dear Healthcare Professional Letter, and the InfoWatch email newsletter.
Since the product was to be withdrawn from the market within a short timeframe, the sponsor cancelled this submission on May 2, 2024. Due to the cancellation, review of this submission was not resumed and there was no assessment by Health Canada at the time of the cancellation.
The Benefit-Harm-Uncertainty was not assessed as this submission was cancelled and review was stopped. The post-approval commitment was not fulfilled as the submission was cancelled and review was not completed.
The existing conditions associated with the NOC/c remain unchanged.
What consequences does the cancellation have for patients accessing the drug under the Special Access Programme (SAP), or via clinical trials?
Healthcare professionals were advised that Albrioza should not be initiated in new patients. Albrioza is now available only under Amylyx Pharmaceuticals Inc.'s Patient Support Program for patients currently receiving treatment with Albrioza.
There are no clinical trials for Albrioza underway in Canada.
Manufacturer:
Amylyx Pharmaceuticals Inc.
Drug Identification Numbers issued:
N/A
Date filed:
2023-10-26
Related Drug Products
| Product name | DIN | Company name | Active ingredient(s) & strength |
|---|---|---|---|
| ALBRIOZA | 02527707 | AMYLYX PHARMACEUTICALS INC. | SODIUM PHENYLBUTYRATE 3 G / SACHET URSODOXICOLTAURINE 1 G / SACHET |