Acadia Pharmaceuticals Inc. announced that Health Canada has accepted its New Drug Submission (NDS) for trofinetide for the treatment of Rett syndrome and has granted Priority Review. This decision paves the way for potential approval of the first treatment option for Rett syndrome in Canada.
Rett syndrome is a rare, complex neurodevelopmental disorder primarily affecting females, characterized by developmental regression, loss of communication skills, and stereotypic hand movements. It affects an estimated 600 to 900 individuals in Canada. The condition arises from mutations in the MECP2 gene on the X chromosome, leading to profound impairments in central nervous system function.
Clinical Evidence Supporting Trofinetide
The Health Canada filing is backed by data from the Phase 3 LAVENDER study, a pivotal trial evaluating trofinetide against placebo in 187 girls and young women with Rett syndrome. The study met its co-primary endpoints, demonstrating statistically significant improvements from baseline in the Rett Syndrome Behaviour Questionnaire (RSBQ) total score (caregiver assessment) and the Clinical Global Impression–Improvement (CGI-I) scale score (clinician perspective) at week 12. A key secondary endpoint measuring improvements in communication was also statistically significant.
Priority Review Significance
Health Canada grants Priority Review to drug submissions intended for serious, life-threatening, or severely debilitating illnesses where there is substantial evidence of clinical effectiveness, addressing unmet medical needs or providing an improved benefit/risk profile over existing therapies.
Expert Perspectives
Pamela di Cenzo, Vice President, General Manager, Rare Disease, Canada at Acadia, stated, "Rett syndrome is a profoundly debilitating and complex neurodevelopmental disorder that presents differently across patients and can lead to an array of unpredictable symptoms. If granted marketing authorization, trofinetide will be the first option available to treat Rett syndrome in Canada."
Sabrina Millson, President of the Ontario Rett Syndrome Association (O.R.S.A.), added, "O.R.S.A. is pleased that Health Canada has granted Priority Review for this promising treatment which, if approved, would be a significant step forward in addressing the unmet medical needs of Canadians living with Rett syndrome. Our community of patients, caregivers and supporters are excited at the prospect of having a treatment option for Rett syndrome."
Current Landscape and Future Implications
Trofinetide is already approved in the United States for treating Rett syndrome in adult and pediatric patients aged two years and older. Approval in Canada would represent a significant advancement in addressing the unmet medical needs of individuals with Rett syndrome in the country.
