FDA Grants Priority Review to Praxis Precision Medicines' Relutrigine for Rare Epileptic Encephalopathies
The FDA has accepted Praxis Precision Medicines' NDA for relutrigine with priority review and set a PDUFA target date of September 27, 2026. The therapy targets SCN2A and SCN8A developmental and epileptic encephalopathies and could become the first approved treatment for these rare conditions. The company has bolstered its cash position to support commercial launch preparations.
The U.S. Food and Drug Administration has accepted for priority review Praxis Precision Medicines' New Drug Application for relutrigine, a potential treatment for SCN2A and SCN8A developmental and epileptic encephalopathies. The FDA has set a target action date under the Prescription Drug User Fee Act of September 27, 2026. If approved, relutrigine would be the first disease-modifying therapy for children suffering from these devastating and fatal conditions.
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 a first-in-class small molecule in development for the treatment of developmental and epileptic encephalopathies as a preferential inhibitor of persistent sodium current, shown to be a key driver of seizure symptoms in severe DEEs. 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.
The company is moving rapidly to construct a commercial platform capable of supporting two near-term neurology launches in the U.S. Praxis closed 2025 with $926 million in cash and securities, nearly doubling year-over-year after a $457 million increase, then added $621 million via a January 2026 offering. Pro forma cash of about $1.5 billion gives the company what it describes as runway into 2028, providing capacity to fund simultaneous launches and late-stage development.
Relutrigine is also being investigated in broad DEEs through the EMERALD trial, which is expected to be completed by the end of 2026. The company has established a diversified, multimodal CNS portfolio including multiple programs across movement disorders and epilepsy, with four late-stage product candidates.