Acadia Pharmaceuticals Inc announced that the European Commission (EC) has granted Marketing Authorisation (MA) for DAYBU (trofinetide) for the treatment of neurobehavioral symptoms of Rett syndrome in adults and pediatric patients aged 5 years and older, making it the first and only treatment approved for Rett syndrome in the European Union (EU).

Catherine Owen Adams, Chief Executive Officer (CEO), Acadia, said, “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. For people living with Rett syndrome, a devastating rare neurodevelopmental disorder, there have been no approved treatment options in the EU. We are proud to make DAYBU available and look forward to supporting patients, caregivers, and healthcare providers gain access to treatment."

The DAYBU MA in the EU 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 (RSBQ) and Clinical Global Impression-Improvement (CGI-I) scale co-primary endpoints.

Nadia Bahi, MD, PhD, Pediatric Neurology, Necker-Enfants Malades University Hospital, Paris Cité University, Paris, France, said, “Rett syndrome places a profound burden on individuals, families, and caregivers, yet treatment options have remained extremely limited. Today's approval in the European Union is an important milestone that recognises the significant unmet medical need in this rare disease. It provides clinicians with a new treatment option to help address the neurobehavioral symptoms of Rett syndrome and represents progress for the Rett community.”

With this approval, DAYBU is authorised to be marketed in all 27 EU member states, as well as Iceland, Liechtenstein, and Norway. As a next step, Acadia will now begin pricing and reimbursement negotiations with relevant national authorities to potentially bring DAYBU to patients across the EU.