Medication Will Be Available for Patients Two Years of Age and Older in April
Acadia Pharmaceuticals Inc. recently announced that their medication, DAYBUETM (trofinetide) has gained approval from the U.S. Food and Drug Administration (FDA). DAYBUE is now the first and only drug approved for treating Rett Syndrome. The company expects the medication to be on the market by the end of April 2023 for patients two years and older. Rett Syndrome is a neurodevelopmental disorder that is classified as an autism spectrum disorder. Support for the FDA approval of the drug came from the results of a phase 3 trial that evaluated the efficacy and safety of trofinetide versus placebo in 187 female patients with Rett syndrome five to 20 years of age. The trial showed that treatment with DAYBUE delivered statistically significant improvement compared to placebo on both co-primary efficacy endpoints, as measured by the change from baseline in Rett Syndrome Behaviour Questionnaire (RSBQ) total score (p=0.018) and the Clinical Global Impression-Improvement (CGI-I) Scale score (p=0.003) at week 12. The RSBQ is a caregiver assessment that evaluates Rett syndrome symptoms including vocalizations, facial expressions, eye gaze, hand movements (or stereotypies), repetitive behaviors, breathing, night-time behaviors, and mood. The CGI-I is a universal physician assessment of whether a patient has improved or worsened. During the phase 3 trial, the most common side effects were diarrhea (82%) and vomiting (29%). Steve Davis, Acadia’s Chief Executive Officer, is excited about his company’s new product. He states, “Today marks an important milestone for the Rett community and Acadia. As the first FDA-approved drug for the treatment of Rett syndrome, DAYBUE now offers the potential to make meaningful differences in the lives of patients and their families who have lacked options to treat the diverse and debilitating array of symptoms caused by Rett syndrome. We are grateful to all of the Rett syndrome patients, caregivers, clinical investigators and our employees who have contributed to making today a reality and look forward to getting DAYBUE to patients as quickly as possible.”
