Business
Praxis Precision Medicines Announces Plans to File an NDA for Relutrigine in SCN2A and SCN8A Developmental and Epileptic Encephalopathies in Early 2026
EMBOLD data to serve as basis of efficacy and safety following discussion with the FDA BOSTON, Dec. 11, 2025 (GLOBE NEWSWIRE) -- Praxis Precision Medicines,
About this update from Praxis Precision Medicines, Inc.
[{"type":"text","content":"EMBOLD data to serve as basis of efficacy and safety following discussion with the FDA\nBOSTON, Dec. 11, 2025 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance, today announced the successful completion of the planned discussion with the FDA leading to the confirmation to file a New Drug Application (NDA) for relutrigine in early 2026. “We are very pleased with the collaborative discussions we've had with the FDA, which support our path toward an NDA submission in early 2026,” said Marcio Souza, president and chief executive officer. “Building on the unprecedented and compelling results from the EMBOLD study, where relutrigine demonstrated statistically significant and clinically meaningful improvements, we are advancing toward delivering a much-needed therapy for children living with these devastating diseases.” About Relutrigine (PRAX-562)Relutrigine is a first-in-class small molecule in development for the treatment of developmental and epileptic encephalopathies (DEEs) as a preferential inhibitor of persistent sodium current, shown to be a key driver of seizure symptoms in severe DEEs. Relutrigine’s mechanism of precision sodium channel (NaV) modulation is consistent with superior selectivity for disease-state NaV channel hyperexcitability. In vivo studies of relutrigine have demonstrated dose-dependent inhibition of seizures up to complete control of seizure activity in SCN2A, SCN8A and other DEE mouse models. Relutrigine has been generally well-tolerated in three Phase 1 studies and has demonstrated biomarker changes indicative of NaV channel modulation. Data from the Phase 2 EMBOLD study demonstrated a well-tolerated safety profile with robust, short- and long-term improvement in motor seizures in a heavily pre-treated population, alongside maintained seizure freedom in some patients with SCN2A- and SCN8A-DEE. Relutrigine has received Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation from the FDA for the treatment of SCN2A-DEE, SCN8A-DEE and Dravet syndrome; as well as Breakthrough Therapy Designation (BTD), and ODD from the European Medicines Agency for the treatment of SCN2A-DEE ...