relutrigine

Relutrigine (PRAX-562) is a first-in-class, orally active small molecule in development for the treatment of developmental and epileptic encephalopathy (DEE), including SCN2A-DEE and SCN8A-DEE. It acts as a preferential inhibitor of persistent sodium current (INa), specifically targeting disease-state sodium channel hyperexcitability. Relutrigine demonstrates potent and selective inhibition of persistent sodium currents induced by mutations or activators such as ATX-II or SCN8A N1768D, with IC50 values in the low nanomolar range. In preclinical models, it has shown dose-dependent seizure inhibition up to complete control of seizure activity. Clinical studies have demonstrated significant reductions in motor seizures and improvements across multiple domains such as alertness, communication, and behavior. The drug has been generally well-tolerated in clinical trials with no discontinuations due to adverse events. Relutrigine has received Orphan Drug Designation from both the FDA and EMA for its target indications[1][2][4][5][6].