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Acadia gets EU approval for DAYBU Rett syndrome treatment
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Acadia gets EU approval for DAYBU Rett syndrome treatment

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  • Acadia Pharmaceuticals received European Commission approval for DAYBU (trofinetide) to treat Rett syndrome symptoms in patients aged five years and older.
  • The approval covers all 27 European Union member states, plus Iceland, Liechtenstein, and Norway, but the company did not provide a stock price update in the announcement.
  • Acadia stated that it will begin pricing and reimbursement discussions with national authorities to support potential patient access.

Acadia Pharmaceuticals (NASDAQ:ACAD) announced that the European Commission approved DAYBU (trofinetide) for Rett syndrome in adults and pediatric patients aged five years and older, making it the first approved Rett syndrome treatment in the European Union.

The approval is based mainly on results from the Phase 3 LAVENDER study, which showed statistically significant improvements in core Rett syndrome symptoms measured by the Rett Syndrome Behaviour Questionnaire and Clinical Global Impression-Improvement scale.

“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 Acadia Pharmaceuticals Chief Executive Officer Catherine Owen Adams.

DAYBU is authorized across all 27 European Union member states, as well as Iceland, Liechtenstein, and Norway, while Acadia stated it will begin pricing and reimbursement negotiations with relevant national authorities.

Acadia stated that the EU authorization expands the potential availability of DAYBU for Rett syndrome patients.

Acadia Pharmaceuticals focuses on developing and commercializing medicines for neurological and rare diseases, with the company continuing efforts to expand access to approved treatments in additional markets.

Rett syndrome affects approximately one in 10,000 to 15,000 female births worldwide and is typically associated with mutations in the MECP2 gene that affect neurological development and function.

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