Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a fully integrated, leading central nervous system (CNS) precision neuroscience biopharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has accepted for priority review its New Drug Application (NDA) for relutrigine, for the treatment of SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs). The FDA has set a target action date under the Prescription Drug User Fee Act (PDUFA) of September 27, 2026.
- FDA assigned PDUFA target action date of September 27, 2026
“Our first FDA acceptance of an NDA submission marks a significant milestone in our evolution to a commercial-stage company and an important step toward delivering innovative, precision neuroscience therapies to patients in need. SCN2A/8A DEEs have no currently approved targeted therapies and relutrigine, if approved, would be the first disease-modifying therapy for children suffering from these devastating and fatal conditions. We look forward to working closely with the FDA during the review process while continuing to advance our launch preparations,” said Marcio Souza, president and chief executive officer.
Relutrigine for treatment of SCN2A/8A DEEs
The NDA is supported by positive results from the EMBOLD study, which was stopped early for efficacy following a successful interim analysis and recommendation from the Data Monitoring Committee. Relutrigine has an Orphan Drug Designation, as well as a Rare Pediatric Disease Designation and a Breakthrough Therapy Designation. If granted approval, relutrigine will be the first FDA-approved therapy for SCN2A/8A DEE as well as be eligible for a Pediatric Review Voucher.
Relutrigine is also being investigated in broad DEEs through the EMERALD trial, which is expected to be completed by the end of 2026.
About Relutrigine
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 cohort 1 of the Phase 2 EMBOLD study demonstrated a well-tolerated, 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 and SCN8A-DEE.
