Acadia Pharmaceuticals receives positive CHMP opinion for DAYBU

• Acadia Pharmaceuticals (NASDAQ:ACAD) received a positive CHMP opinion recommending EU marketing authorization for DAYBU (trofinetide) for neurobehavioral symptoms of Rett syndrome.
• DAYBU would become the first approved therapy in the EU for this indication if the European Commission grants final approval.
• The recommendation is based on the Phase 3 LAVENDER study showing improvements in core symptoms of Rett syndrome.

Acadia Pharmaceuticals (NASDAQ:ACAD) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion recommending 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 approved by the European Commission, DAYBU would be the first therapy specifically authorized for this indication in the European Union.

“The CHMP’s positive opinion for DAYBU is an important milestone in our mission to bring this innovative therapy to the EU,” said Catherine Owen Adams, Acadia’s Chief Executive Officer.

The positive opinion follows a re-examination procedure and 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 and Clinical Global Impression-Improvement scale.

The European Commission is expected to issue a final decision in the coming months.

If granted, the marketing authorization would apply to all 27 EU member states as well as Iceland, Liechtenstein, and Norway.

Acadia Pharmaceuticals is focused on developing therapies for central nervous system disorders, with DAYBU representing a potential new treatment option for the Rett syndrome community in Europe.