GlobeNewswire·Dec 11, 2025, 8:00 AM ET

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

Praxis Precision Medicines confirmed via FDA discussion that it will file a New Drug Application for relutrigine in early 2026.
The NDA filing is based on compelling results from the EMBOLD study, which showed statistically significant & clinically meaningful improvements for children with developmental & epileptic encephalopathies.
Relutrigine is a first-in-class small molecule acting as a preferential inhibitor of persistent sodium current to address neuronal hyperexcitability in SCN2A-DEE, SCN8A-DEE, & Dravet syndrome.
In vivo studies demonstrated dose-dependent seizure inhibition up to complete control in multiple DEE mouse models, while Phase 1 & 2 trials showed a well-tolerated safety profile.
The drug has received Orphan Drug Designation, Rare Pediatric Disease Designation, & Breakthrough Therapy Designation from the FDA, plus Breakthrough Therapy Designation & Orphan Drug Designation from the EMA for specified indications.
Praxis is advancing this therapy using its proprietary Cerebrum™ small molecule platform & Solidus™ antisense oligonucleotide platform to treat CNS disorders characterized by excitation-inhibition imbalance.