Access to Sephience in Canada
Sephience (sepiapterin) has received Health Canada approval for the treatment of PKU. As with all newly approved therapies in Canada, additional review and reimbursement processes must occur before public coverage decisions are made.
CanPKU+ is gathering community perspectives to inform submissions to the Canadian Drug Agency (CDA). Patient and caregiver input helps decision makers understand the lived experience of PKU and the potential impact of new treatment options.
How You Can Participate
1. Share your experience.
Complete the community survey to help inform our submission to the Canadian Drug Agency. Personal experiences provide valuable context during reimbursement reviews. - This survey has closed. In order to tell your story we suggest providing it in story format (written, video and/or podcast style)
2. Encourage participation.
If you or someone you know is affected by PKU, consider sharing this opportunity so more voices are represented.
3. Stay informed.
This page will be updated as information becomes available regarding the review and reimbursement process.
Why Coverage Matters
For individuals living with PKU, access to additional treatment options may support improved metabolic control, dietary flexibility, and quality of life.
In Canada, public drug coverage decisions are made at the provincial and territorial level following national review processes. Ensuring that patient perspectives are included in those processes helps promote informed and equitable decision making.
Resources and Updates
- Survey for Patients and Caregivers on the Impact of PKU - Now closed
- Download the Sephience Fact Sheet (PDF)
- Message from Tristan Audet (French - Facebook)
- Carnet Phénylcétonurie - Sephience information page (French and English)
- CDA Submission Summary - Coming Soon
Regulatory Milestones
- Health Canada issued a Notice of Compliance to PTC Therapeutics International Limited for Sephience (October 2025).
View Health Canada Summary Basis of Decision - U.S. FDA approval for sepiapterin-responsive PKU (July 2025).
Read announcement - Approval by Japan’s Ministry of Health, Labour and Welfare (December 2025).
Read coverage
Questions or would you like to share your experience?
Email advocacy@canpku.org
CanPKU+ participates in public policy processes in a non-partisan manner to support equitable access to care for individuals and families affected by PKU.