FDA Approves Acadia’s Daybue for Rett Syndrome

The FDA has approved Acadia Pharmaceuticals’ Daybue (trofinetide) to treat adult and pediatric patients two years and older with Rett syndrome, a rare neurodevelopmental disorder.

This approval makes Daybue the only drug approved to treat the disease, which is caused by a gene mutation and is characterized by a period of normal development until six to 18 months of age, followed by developmental regression with loss of communication skills and use of hands.

The agency decision was based on results from a phase 3 study, which showed that Daybue led to significant improvements in treated patients.

With the FDA approval, Acadia has received a Rare Pediatric Disease Priority Review Voucher, which can be used to obtain priority review for a subsequent application.

The company plans on making the drug available in the U.S. by the end of April.