Praxis Precision Medicines Announces Plans to File an NDA for Relutrigine in SCN2A and SCN8A Developmental and Epileptic Encephalopathies in Early 2026

Core Insights - Praxis Precision Medicines has successfully completed discussions with the FDA, confirming the path to file a New Drug Application (NDA) for relutrigine in early 2026 [1][2] Company Overview - Praxis Precision Medicines is a clinical-stage biopharmaceutical company focused on developing therapies for central nervous system (CNS) disorders, particularly those related to neuronal excitation-inhibition imbalance [4] - The company utilizes genetic insights to create therapies for both rare and prevalent neurological disorders through its proprietary platforms, Cerebrum™ and Solidus™ [4] Product Development - Relutrigine is a first-in-class small molecule aimed at treating developmental and epileptic encephalopathies (DEEs) by inhibiting persistent sodium current, which is a key factor in seizure symptoms [3] - In vivo studies have shown that relutrigine can achieve dose-dependent inhibition of seizures, with complete control observed in certain mouse models [3] - The EMBOLD study demonstrated a well-tolerated safety profile for relutrigine, with significant improvements in motor seizures among heavily pre-treated patients [3] Regulatory Designations - Relutrigine has received Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation from the FDA for SCN2A-DEE, SCN8A-DEE, and Dravet syndrome, as well as Breakthrough Therapy Designation (BTD) from the European Medicines Agency [3]