FDA has set a PDUFA target date of September 27, 2026
BOSTON, March 30, 2026 (GLOBE NEWSWIRE) — Praxis Precision MedicineInc. (NASDAQ: PRAX), a fully integrated, central nervous system (CNS) precision neuroscience biopharmaceutical company, today announced that the US Food and Drug Administration (FDA) has accepted for priority review its New Drug Application (NDA), for review, and for the treatment of NSCN2. encephalopathies (DEEs). The FDA has set a target action date of September 27, 2026 under the Prescription Drug User Fee Act (PDUFA).
“The initial acceptance of our FDA submission marks a significant milestone in our evolution to a commercial-stage company and is an important step toward bringing innovative, precision neuroscience therapies to patients in need. We look forward to working closely with the FDA during the review process while continuing our launch preparations,” said Marcio Souza, President and CEO.
Relutrigine for the treatment of SCN2A/8A DEEs
The NDA is supported by positive results from the EMBOLD study, which was stopped earlier for efficacy after a successful interim analysis and recommendation by the Data Monitoring Committee. Relutrigine has orphan drug designation, as well as rare pediatric disease designation and breakthrough therapy designation. If approved, relutrigine will be the first FDA-approved treatment for SCN2A/8A DEE and also eligible for a pediatric review voucher.
Relutrigine is also being investigated in widespread 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 developmental encephalopathies (DEEs) as an effective inhibitor of persistent sodium influx, which has been shown to be a key driver of seizure symptoms in severe DEEs. Relutrigine’s precise sodium channel (NaV) modulation mechanism is consistent with its selectivity for NaV channel hyperexcitability in the disease state. In vivo studies of relutrigine have shown dose-dependent inhibition of seizures up to complete control of seizure activity in SCN2A, SCN8A and other DEE mouse models. Relutrigine was generally well tolerated in three phase 1 studies and demonstrated changes in biomarkers indicative of NaV channel modulation. Data from Cohort 1 of the Phase 2 EMBOLD study demonstrated well-tolerated, robust, short-term and long-term improvement in motor seizures in a largely pretreated population, despite preservation of 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 Drayt syndrome; Also Breakthrough Therapy Designation (BTD) from the European Medicines Agency for the treatment of SCN2A-DEE and SCN8A-DEE and ODD. For more information on studying Emerald, please visit Emerald | Resistance studies.
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