European regulators give second chance approval to Acadia’s Daybu for Rett syndrome

European Medicines Agency's CHMP has reversed its earlier negative vote and now recommends marketing authorization for Acadia Pharmaceuticals' drug Daybu (trofinetide) for Rett syndrome.

The positive opinion follows Acadia’s request for a re-examination after the February vote against the therapy. The CHMP’s recommendation will be sent to the European Commission, which must decide within 67 days.

Daybu was already approved by the U.S. FDA in 2023 for pediatric and adult patients with Rett syndrome, a rare genetic brain disorder. The EU currently has no approved treatments for the neurobehavioral symptoms of the disease.

Acadia said the CHMP’s positive opinion is an important milestone for bringing an innovative therapy to European patients.