DAYBU® (trofinetide) Recommended for Approval in the European Union by CHMP

About this update from Acadia Pharmaceuticals Inc.

-- 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 SAN DIEGO, June 26, 2026--(BUSINESS WIRE)--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 positive opinion for DAYBU® is an important milestone in our mission to bring this innovative therapy to the EU, where there are no therapies specifically approved for the neurobehavioral symptoms of this devastating condition," said Catherine Owen Adams, Acadia's Chief Executive Officer. "Our commitment is to make a meaningful difference in the lives of patients, caregivers, and the wider Rett community by addressing this significant unmet need, and we are very pleased with the outcome of the re-examination process." 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. "For decades, families in Europe affected by Rett syndrome have had no medicine specifically approved for the neurobehavioral symptoms of this condition, despite the profound impact they have on almost every aspect of daily life," said Pedro Rocha, President of Rett Syndrome Europe. "The CHMP's positive opinion represents hope for thousands of European Union individuals living with this devastating condition, their families and caregivers." Following the CHMP recommendation, the European Commission will review th...

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