Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) announced that the company has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for trofinetide for the treatment of Rett syndrome in adults and pediatric patients two years of age and older. If granted marketing authorization, trofinetide will be the first and only approved therapy for Rett syndrome in the European Union (EU). Trofinetide is already approved in the United States and Canada for Rett syndrome in adults and pediatric patients two years of age and older.
“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,” said Catherine Owen Adams, Acadia’s Chief Executive Officer. “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 MAA is supported by positive results from the pivotal Phase 3 LAVENDER study evaluating the efficacy and safety of trofinetide versus placebo in 187 girls and young women five to 20 years of age with Rett syndrome. The co-primary endpoints were change from baseline in the Rett Syndrome Behaviour Questionnaire (RSBQ) total score, a caregiver assessment, and Clinical Global Impression–Improvement (CGI-I) scale score, clinician perspective, at week 12; both were statistically significant. The RSBQ is a 45-item rating scale completed by the caregiver that assesses a range of symptoms of Rett syndrome (breathing, hand movements or stereotypies, repetitive behaviors, night-time behaviors, vocalizations, facial expressions, eye gaze, and mood). The key secondary endpoint measuring the change from baseline to week 12 in the Communication and Symbolic Behavior Scales Development Profile Infant-Toddler Checklist–Social composite score (CSBS-DP-IT–Social) was also statistically significant when compared to placebo.1
“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,” said Becky Jenner, President of Rett Syndrome Europe. “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.”
