Relutrigine (PRAX-562) - (EMBOLD)

March 30, 2026: Praxis Precision Medicines, Inc. (NASDAQ: PRAX) said the FDA accepted for priority review its NDA for relutrigine to treat SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs). The FDA assigned a PDUFA target action date of September 27, 2026.

The NDA is supported by positive results from the Phase 2 EMBOLD study, which was stopped early for efficacy after a successful interim analysis and a Data Monitoring Committee recommendation. Relutrigine has FDA Orphan Drug, Rare Pediatric Disease, and Breakthrough Therapy designations; if approved, it would be the first FDA-approved therapy for SCN2A/SCN8A DEE and would be eligible for a Pediatric Review Voucher.

Additional catalyst: relutrigine is being studied in broader DEEs in the EMERALD trial, expected to be completed by the end of 2026.