Shares of Acadia Pharmaceuticals Inc. (NASDAQ:ACAD) climbed after a key advisory committee for the European Medicines Agency (EMA) endorsed approval of DAYBU (trofinetide) for the treatment of neurobehavioral symptoms associated with Rett syndrome. The Committee for Medicinal Products for Human Use (CHMP) delivered a positive opinion following a re-examination procedure, recommending the medicine for adults and pediatric patients aged five years and older.
The CHMP’s recommendation draws heavily on data from the Phase 3 LAVENDER study. That trial demonstrated statistically significant improvements in core features of Rett syndrome as measured by the Rett Syndrome Behaviour Questionnaire (RSBQ) and the Clinical Global Impression-Improvement (CGI-I) scale, according to the information released in connection with the committee’s opinion.
If the European Commission accepts the CHMP opinion and issues a marketing authorization, DAYBU would become the first therapy approved in the European Union specifically for the neurobehavioral symptoms of Rett syndrome. The marketing authorization, if granted, would extend across all 27 EU member states and also apply to Iceland, Liechtenstein, and Norway.
Regulatory timing remains unresolved; the European Commission is expected to issue a final decision in the coming months. Until that decision is rendered, the recommendation from the CHMP does not constitute an authorization and the therapy would not be available under an EU marketing authorization.
Rett syndrome is described as a rare neurodevelopmental disorder that occurs in approximately one of every 10,000 to 15,000 female births worldwide. The condition is typically linked to a mutation on the MECP2 gene and is associated with impaired communication skills, loss of purposeful hand use, and affected motor function.
Acadia’s Chief Executive Officer Catherine Owen Adams characterized the CHMP’s positive opinion as an important milestone toward making the therapy available in the EU, where no therapies are currently approved expressly for the neurobehavioral symptoms of Rett syndrome.
Contextual notes
- The CHMP recommendation followed a re-examination procedure and covers patients aged five years and older.
- The Phase 3 LAVENDER study provided the primary evidence cited by the committee, using RSBQ and CGI-I to measure benefit.
- Final regulatory authority rests with the European Commission; a positive CHMP opinion is a key step but not the final approval.