Acadia Pharmaceuticals announces commercialisation of Daybue for Rett syndrome
pharmafile | April 19, 2023 | News story | Sales and Marketing |
Acadia Pharmaceuticals has announced that Daybue (trofinetide) is now available for adult and paediatric patients over the age of two with Rett syndrome in the US. This commercialisation follows the FDA’s approval of the drug on 10 March 2023, making it the first and only FDA approved drug for the treatment of Rett syndrome.
Rett syndrome is a rare neurodevelopmental disorder which usually presents between six and 18 months of age, when a child’s development appears to slow down or stagnate. Patients with Rett syndrome will experience motor deterioration throughout their lives. It is estimated that an approximate 6,000 to 9,000 patients in the US with Rett syndrome, although only 4,500 are currently diagnosed.
Steve Davis, Acadia’s CEO, commented: “The Rett syndrome community has been waiting a long time for a drug to treat this debilitating disorder. We have worked hard to make Daybue available as quickly as possible following FDA approval. We are focused on providing robust patient support resources through Acadia Connect to help caregivers and healthcare providers access this important new therapy.”
Alan Percy, MD, professor of Paediatrics, Neurology, Neurobiology, Genetics and Psychology at the University of Alabama, Birmingham, US, added: “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 [and have] a therapy that has been shown to address multiple symptoms of Rett syndrome [which] provides a promising treatment option that may lead to meaningful impact for patients and their families.”