Acadia Submits Trofinetide To European Medicines Agency For Rett Syndrome Treatment

Acadia Pharmaceuticals Inc. has provided a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for approval for trofinetide in order to treat Rett syndrome in patients ages two and above. If the medicine gets approval, trofinetide will be the only medicine available for patients with this rare neurological disease in the European Union (EU). The drug has recently been approved for children in the US and Canada for the same purpose as adjunct therapy.

“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,” said Catherine Owen Adams, Acadia’s Chief Executive Officer. 

The application is accessed on the basis of promising results from the Phase 3 LAVENDER™ study, which evaluated trofinetide's safety and effectiveness in 187 girls and young women aged 5 to 20 with Rett syndrome. Using caregivers’ data from the Rett Syndrome Behaviour Questionnaire (RSBQ) and clinicians’ data from the Clinical Global Impression-Improvement (CGI-I) scale at week 12, the study met its co-primary endpoints. The secondary outcome, based on the CSBS-DP-IT – Social subscale, was also statistically significantly different from placebo.

Becky Jenner, President of Rett Syndrome Europe, also mentioned, “Rett syndrome is a severe and complex neurodevelopmental disorder that manifests differently in each affected person, leading to a wide range of symptoms over the course of their lives. The Rett syndrome community in Europe is excited by the prospect of having an available treatment option. Rett syndrome has a significant impact on people living with this condition and also impacts caregivers and families who often have to navigate challenging symptoms that impact their daily lives.”