Acadia Secures EU Approval for DAYBU as First Rett Syndrome Treatment, Shares Rise

(RTTNews) – Acadia Pharmaceuticals Inc. (ACAD) announced Monday that the European Commission has granted marketing authorization for DAYBU (trofinetide) for the treatment of neurobehavioral symptoms of Rett syndrome in adults and pediatric patients aged 5 years and older.

The approval is 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), the co-primary endpoints.

Rett syndrome is a rare neurodevelopmental disorder that affects females and is generally caused by mutations in the MECP2 gene. The condition is characterized by developmental regression, loss of acquired communication skills and purposeful hand use, and motor abnormalities.

DAYBU (trofinetide) is a synthetic analog of the N-terminal tripeptide of insulin-like growth factor 1 (IGF-1), a hormone involved in the development and function of the nervous system. The drug is also approved for Rett syndrome in the United States and Canada.

According to Acadia, DAYBU is the first treatment authorized for Rett syndrome in the European Union (EU). The authorization extends to all 27 EU member states, as well as Iceland, Liechtenstein and Norway. The company said it will begin pricing and reimbursement negotiations with the national authorities following the approval.

ACAD closed Friday’s trade at $29.50, up 1.58%. In the pre-market trade, stock is up 4.73% at $30.89.

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