-- European Commission decision expected in the coming months

-- If approved, DAYBU® would become the first treatment for neurobehavioral symptoms of Rett syndrome in the European Union

Acadia Pharmaceuticals Inc. (NASDAQ:ACAD) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion following a re-examination procedure, recommending the granting of a marketing authorization for DAYBU® (trofinetide) for the treatment of neurobehavioral symptoms of Rett syndrome in adults and pediatric patients aged five years and older. If granted marketing authorization by the European Commission, DAYBU® would be the first therapy approved for this indication in the European Union (EU).

The CHMP’s recommendation 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. Importantly, these findings indicate that treatment with DAYBU® can address some of the most impactful aspects of Rett syndrome, which severely impact quality of life for patients and caregivers.

Following the CHMP recommendation, the European Commission will review the opinion and is expected to issue a final decision in the coming months. If DAYBU® is approved, the marketing authorization would apply to all 27 EU member states, as well as Iceland, Liechtenstein, and Norway.