FDA Grants Priority Review to Relutrigine for Rare Epileptic Encephalopathies
The FDA has granted Priority Review to relutrigine for SCN2A and SCN8A developmental and epileptic encephalopathies, with a PDUFA target date of September 2026. Phase 2/3 trial data showed a 53% placebo-adjusted reduction in motor seizures and 66.2% increase in seizure-free days. If approved, it would be the first targeted therapy for these rare, fatal childhood conditions.
The Food and Drug Administration has granted Priority Review to the New Drug Application for relutrigine, a novel sodium channel modulator, for the treatment of SCN2A and SCN8A developmental and epileptic encephalopathies. The Prescription Drug User Fee Act target date for the application is September 27, 2026.
The NDA submission is supported by data from the phase 2/3 EMBOLD study, which enrolled patients with early onset SCN2A DEE or SCN8A DEE. Study participants were randomly assigned to receive relutrigine once daily for 16 weeks or relutrigine once daily for 12 weeks and matching placebo for 4 weeks. The primary endpoint was the change from baseline in monthly motor seizure frequency.
Findings from cohort 2 showed treatment with relutrigine resulted in a 53% placebo-adjusted reduction in motor seizures. Relutrigine also met a key secondary endpoint, demonstrating a statistically significant 66.2% increase in motor seizure-free days. Statistically significant improvements in clinician and caregiver global impression scores were also observed.
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. The EMBOLD study data were presented at the 2025 American Epilepsy Society Annual Meeting.