Praxis Precision Medicines Receives FDA Acceptance And Priority Review For Relutrigine NDA In SCN2A and SCN8A Developmental And Epileptic Encephalopathies

Praxis Precision Medicines, Inc. announced that the U.S. Food and Drug Administration has accepted for priority review its New Drug Application (NDA) for relutrigine, a treatment for 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.

Marcio Souza, President and Chief Executive Officer of Praxis Precision Medicines, said that this NDA acceptance represents a major milestone in the company’s transition to a commercial-stage biopharmaceutical company. He noted that SCN2A/8A DEEs currently have no approved targeted therapies, and if approved, relutrigine would be the first disease-modifying treatment for children suffering from these severe and life-threatening conditions. He added that Praxis looks forward to collaborating closely with the FDA during the review process while continuing preparations for a potential launch.

The NDA submission for relutrigine 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 also received Orphan Drug Designation, Rare Pediatric Disease Designation, and Breakthrough Therapy Designation. If approved, it would become the first FDA-approved therapy for SCN2A/8A DEEs and would be eligible for a Pediatric Review Voucher.

In addition, relutrigine is being investigated for broader developmental and epileptic encephalopathies in the ongoing EMERALD trial, which is expected to be completed by the end of 2026. These studies collectively aim to establish relutrigine as a precision neuroscience therapy for children with these devastating disorders.