Acadia Pharmaceuticals Announces DAYBUE™ (trofinetide) is Now Available for the Treatment of Rett Syndrome

Acadia Pharmaceuticals today announced that DAYBUE™ (trofinetide) is now available for the treatment of Rett syndrome in adult and pediatric patients two years of age and older in the United States. DAYBUE has demonstrated the potential to improve the signs and symptoms of Rett syndrome. DAYBUE was approved by the U.S. Food and Drug Administration (FDA) on March 10, 2023, and is the first and only drug approved by the FDA for the treatment of Rett syndrome.

“The Rett syndrome community has been waiting a long time for a drug to treat this debilitating disorder, and we have worked hard to make DAYBUE available as quickly as possible following FDA approval,” said Steve Davis, Chief Executive Officer of Acadia. “We are focused on providing robust patient support resources through Acadia Connect® to help caregivers and healthcare providers access this important new therapy.”

“Following my experience as an investigator in the Lavender Phase 3 study I have already initiated the process of prescribing DAYBUE for my patients. I am thrilled to now be able to offer DAYBUE to more people living with Rett syndrome, outside of a clinical trial,” said Alan Percy, M.D., Professor of Pediatrics, Neurology, Neurobiology, Genetics, and Psychology at University of Alabama, Birmingham. “Having a therapy that has been shown to address multiple symptoms of Rett syndrome provides a promising treatment option that may lead to meaningful impact for patients and their families.”

Rett syndrome is a rare, complex, neurodevelopmental disorder that may occur over four stages and affects approximately 6,000 to 9,000 patients in the U.S.