Acadia Pharmaceuticals Inc. has announced that Health Canada has accepted its New Drug Submission for trofinetide, a potential treatment for Rett syndrome, a rare neurodevelopmental disorder. This acceptance comes with Health Canada granting Acadia's submission priority assessment status.

Pamela di Cenzo, Vice President and Managing Director of Rare Diseases, Canada, at Acadia, emphasized the complexity and debilitating nature of Rett syndrome. She highlighted the variability in symptoms and the unpredictable nature of the disorder. If granted marketing authorization, trofinetide would become the first approved drug for treating Rett syndrome in Canada.

Priority review status from Health Canada is granted to drug submissions addressing serious, life-threatening, or severely debilitating conditions, with substantial evidence of effectiveness or significant benefits over existing treatments.

Sabrina Millson, President of the Ontario Rett Syndrome Association (ORSA), expressed satisfaction with Health Canada's decision, emphasizing the potential of trofinetide to address the unmet medical needs of Canadians with Rett syndrome.

The submission to Health Canada is supported by findings from the pivotal Phase 3 LAVENDER™ study, which demonstrated the efficacy and safety of trofinetide compared to placebo in 187 girls and young women with Rett syndrome. Key endpoints, including improvements in behavior and communication, were statistically significant.

Rett syndrome is a rare neurodevelopmental disorder primarily affecting females, typically manifesting after normal development during the first two years of life. It is caused by mutations in the MECP2 gene on the X chromosome and results in profound impairment of central nervous system functions, including loss of communication skills and movement abnormalities.

Approximately one in 10,000 to 15,000 female births worldwide are affected by Rett syndrome, with an estimated 600 to 900 cases in Canada alone. The disorder is characterized by a period of developmental regression followed by a plateau phase, often lasting years or decades, with diagnosis typically occurring around age three based on clinical assessment.

 

Source: businesswire.com

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