Praxis Precision Medicines Announces Rare Pediatric Disease Designation Granted for Relutrigine in Dravet Syndrome

Core Insights - The FDA has granted Rare Pediatric Disease Designation (RPDD) for relutrigine in the treatment of Dravet syndrome, marking a significant milestone for Praxis Precision Medicines [1][2] - Relutrigine has shown promising clinical data, demonstrating a 46% reduction in monthly motor seizures and over 30% of patients achieving seizure freedom [4] - Praxis plans to initiate an all-DEE trial (EMERALD) in the first half of 2025, which will include Dravet syndrome patients [1] Company Overview - Praxis Precision Medicines is a clinical-stage biopharmaceutical company focused on developing therapies for central nervous system disorders, particularly those related to neuronal excitation-inhibition imbalance [8] - The company utilizes genetic insights to create therapies for both rare and prevalent neurological disorders through its proprietary platforms [8] Product Information - Relutrigine is a first-in-class small molecule designed to treat developmental and epileptic encephalopathy (DEE) by inhibiting persistent sodium currents, which are key drivers of seizure symptoms [5] - The drug has received Orphan Drug Designation (ODD) and RPDD from the FDA, as well as ODD from the European Medicines Agency for SCN2A and SCN8A DEEs [5] Clinical Data - In the EMBOLD study, relutrigine demonstrated a placebo-adjusted monthly motor seizure reduction of 46% and a 77% reduction in median seizure rate during long-term extension [4] - The company is currently enrolling patients in a second registrational cohort for SCN2A and SCN8A patients, with topline results expected in the first half of 2026 [4]