Praxis Precision announces U.S. FDA granted RPDD for relutrigine

Praxis Precision (PRAX) Medicines announced the U.S. Food and Drug Administration has granted Rare Pediatric Disease Designation, RPDD, for relutrigine in Dravet syndrome. In September, and updated at the recent 2024 American Epilepsy Society Annual Meeting, Praxis shared results for relutrigine in SCN2A and SCN8A patients in cohort 1 of the EMBOLD study that showed: Placebo-adjusted monthly motor seizure reduction of 46% during the double-blind period; Over 30% of patients achieved seizure freedom status while on relutrigine; Meaningful gains observed in alertness, communication and seizure severity; 77% reduction in median seizure rate seen for patients in the long-term extension. Based on the results of this study, Praxis has initiated and is enrolling patients in a second, registrational cohort for SCN2A and SCN8A patients, expecting topline results in the first half of 2026.