European Commission Approves DAYBU® (trofinetide) as the First and Only Treatment for Neurobehavioral Symptoms of Rett Syndrome in the European Union
Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that the European Commission (EC) has granted marketing authorization for DAYBU (trofinetide) for the treatment of neurobehavioral symptoms of Rett syndrome in adults and pediatric patients aged five years and older, making it the first and only treatment approved for Rett syndrome in the European Union (EU).
“The approval of DAYBU marks a significant milestone for the Rett syndrome community in the EU and advances our mission to bring this innovative treatment to patients and families who have long faced a profound unmet medical need,” said Catherine Owen Adams, Acadia’s Chief Executive Officer. “For people living with Rett syndrome, a devastating rare neurodevelopmental disorder, there have been no approved treatment options in the EU. We are proud to make DAYBU available and look forward to supporting patients, caregivers, and healthcare providers gain access to treatment."
The DAYBU marketing authorization in the EU is primarily based on results from the Phase 3 LAVENDER™ study, which demonstrated statistically significant and clinically meaningful improvements in core features of Rett syndrome, as measured by the Rett Syndrome Behaviour Questionnaire (RSBQ) and Clinical Global Impression-Improvement (CGI-I) scale co-primary endpoints.
