Neuren Pharmaceuticals (ASX:NEU) has announced that its partner Acadia Pharmaceuticals has submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for DAYBUE (trofinetide) for the treatment of Rett syndrome in adults and paediatric patients two years of age and older.
Neuren said Acadia anticipates potential approval in early 2026.
If granted marketing authorisation, DAYBUE will be the first and only approved therapy for Rett syndrome in the European Union. Acadia anticipates initiating Managed Access Programs in Europe in the second quarter of 2025. DAYBUE is already approved in the United States and Canada for Rett syndrome.
Neuren CEO Jon Pilcher said, “We are pleased to see the achievement of this very important milestone towards potentially bringing trofinetide to the Rett community in Europe.”
Under the worldwide licence agreement with Acadia, Neuren is eligible to receive milestone payments and royalties related to the development and commercialisation of DAYBUE outside North America, as detailed in the table below.