Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) has announced that Health Canada has granted marketing authorization for DAYBUE™ (trofinetide) to treat Rett syndrome in patients aged two and older. This approval, under the Priority Review process, makes DAYBUE the first and only medication approved in Canada for this condition.
Rett syndrome is a rare neurodevelopmental disorder primarily affecting females, characterized by significant regression after normal early development. In Canada, it is estimated that there are 600 to 900 patients with Rett syndrome, who may experience symptoms such as loss of communication skills, difficulties with purposeful hand use, gait abnormalities, and repetitive hand movements. Many individuals with Rett syndrome require constant care throughout their lives.
Catherine Owen Adams, Acadia’s CEO, stated, “Health Canada’s authorization of DAYBUE is a major milestone for the Rett syndrome community in Canada, reflecting our commitment to improving access to this treatment for patients and families.”
Dr. Anita Datta, a pediatric neurologist at BC Children’s Hospital, emphasized the challenges of treating Rett syndrome, noting that previous options have mainly focused on managing symptoms rather than addressing the disorder itself.
The approval was supported by positive outcomes from the Phase 3 LAVENDER™ study, which assessed the efficacy and safety of trofinetide in 187 girls and young women aged 5 to 20. Key results included statistically significant improvements in caregiver-reported symptoms and clinician assessments.
Sabrina Millson, President of the Ontario Rett Syndrome Association, called the approval a historic milestone, expressing hope for rapid access to the treatment through both private and public funding.
In Canada, DAYBUE is approved for use in adults and pediatric patients two years and older, weighing at least 9 kg.
About Rett Syndrome
Rett syndrome is a rare, complex disorder affecting approximately 1 in 10,000 to 15,000 female births worldwide. It typically involves developmental regression between 18-30 months, followed by a plateau phase. Diagnosis is usually made by age three based on clinical evaluation.
About DAYBUE™ (trofinetide)
Trofinetide is a synthetic analog of the N-terminal tripeptide of insulin-like growth factor 1. Its exact therapeutic mechanism in treating Rett syndrome remains unknown.
