Press Release: Daybue Approved by Health Canada but Not Reimbursed in Canada
FOR IMMEDIATE RELEASE
Ontario Rett Syndrome Association Responds to Canada’s Drug Agency Recommendation on Daybue™
London, Ontario – August 22, 2025 – The Ontario Rett Syndrome Association of Canada (ORSA Canada) is deeply disappointed with today’s recommendation from Canada’s Drug Agency (CDA-AMC) not to reimburse Daybue™ (trofinetide) for individuals with Rett syndrome under the public drug plan.
This decision comes despite Health Canada’s earlier approval of Daybue™ and the growing body of evidence from the LAVENDER and extension studies that showed meaningful improvements in communication, behaviour, and overall quality of life.
“Families tell us every day that these gains mean everything. For individuals living with Rett syndrome, even small improvements can change daily life in profound ways,” said Sabrina Millson, President of ORSA Canada. “The CDA’s recommendation is heartbreaking for our community and leaves many families without equitable access to the only approved treatment for Rett syndrome.”
Rett syndrome is a rare, complex neurological disorder that affects nearly every aspect of daily living, requiring 24/7 care. Without public reimbursement, families face the impossible challenge of affording a treatment that could provide life-changing benefits.
ORSA Canada will continue to advocate for access to Daybue™ with a resubmission as we work closely with policymakers, clinicians, associations and families to ensure that the voices of those living with Rett syndrome are heard.
View the full CDA report here: Trofinetide (Daybue)
Daybue™ in Canada FAQ–What Families Need to Know
Q: What’s the difference between Health Canada and the Canadian Drug Agency (CDA)?
A: Health Canada makes sure new medicines are safe and work as intended. When Health Canada approves a drug, it means doctors in Canada are legally allowed to prescribe it.
The Canadian Drug Agency looks at cost and value. They decide whether public drug plans (like provincial coverage) should pay for the medicine.
So, Health Canada can say YES because the medicine is proven safe and effective, while the CDA can say NO if they believe the cost is too high compared to their measure of benefits.
Q: What is Daybue?
A: Daybue™ (trofinetide) is the first-ever treatment approved in Canada specifically for Rett syndrome.
Q: Who approved it?
A: Health Canada reviewed the science and said Daybue is safe and works as intended.
Q: Why is it hard to access?
A: Because after Health Canada approval, the CDA decides if public drug plans should cover the cost. On Aug 22, 2025, the CDA gave its final recommendation, deciding not to reimburse Daybue.
Q: What does this mean right now?
A: Families without private insurance will need to pay out of pocket, which makes Daybue out of reach for most. Families with private insurance may be able to request coverage.
Q: Can CDA’s decision change?
A: Yes. There is an appeal (reconsideration) process. Advocacy groups, doctors, and the drug company can submit new evidence and family perspectives.
Q: Who makes the final decision?
A: Each province/territory decides if it will fund Daybue, but they usually follow CDA’s recommendation.
Q: How long could this take?
A: These processes can take months, sometimes longer. That’s why advocacy is so important to speed things up.
Q: Has Daybue been approved anywhere else?
A: Yes. It is already approved in the United States.
Q: What can families do?
A: Share your story, support advocacy efforts, and help spread awareness.
References:
https://www.canada.ca/en/health-canada.html
https://www.cda-amc.ca/sites/default/files/DRR/2025/SR0829-Daybue_FINAL_Recommendation.pdf
