Acadia Pharmaceuticals has submitted a marketing authorisation application (MAA) to the European Medicines Agency (EMA) for trofinetide, aimed at treating Rett syndrome in adults and in children aged two years and older.
This rare genetic neurological and developmental disorder impacts brain development and affects mainly females.
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Authorisation would make trofinetide the first and only sanctioned therapy for this neurodevelopmental disorder in the European Union (EU).
Acadia Pharmaceuticals CEO Catherine Owen Adams stated: “This application underscores our continued dedication to the Rett community, and our commitment to making a meaningful impact for people living with Rett syndrome and their caregivers in the EU, who currently have no approved treatment options specifically for this condition.
“We look forward to working with the EMA to address this unmet need in the hopes of potentially bringing this therapy to families who are impacted by Rett syndrome.”
The application is based on outcomes from the Phase III LAVENDER trial, assessing the safety and efficacy of the therapy compared to placebo.
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By GlobalDataIt involved 187 girls and young women aged five to 20 with Rett syndrome.
Its co-primary endpoints were change from baseline in the total score of the 45-item rating scale the Rett Syndrome Behaviour Questionnaire (RSBQ), and clinical global impression improvement scale scores at week 12.
The RSBQ, completed by caregivers, evaluates symptoms across domains such as breathing, hand movements and mood.
The study’s key secondary endpoint — the change in the Communication and Symbolic Behavior Scales Development Profile Infant-Toddler Checklist–Social composite score (CSBS-DP-IT–Social) — was also statistically significant.
Rett syndrome affects almost one in every 10,000 to 15,000 female births globally. It progresses through four stages and can significantly impact the quality of life.
In March 2023, the US Food and Drug Administration approved Acadia’s Daybue (trofinetide) for treating Rett syndrome in adults and paediatric patients aged two years and above. The therapy is also approved in Canada for the same indication.
