FDA Grants Breakthrough Status to Praxis’s Relutrigine Post-EMBOLD Trial
Praxis Precision Medicines has been granted Breakthrough Therapy Designation (BTD) by the U.S. Food and Drug Administration (FDA) for relutrigine (PRAX-562).
This designation applies to its use in children with SCN2A and SCN8A-related developmental and epileptic encephalopathies (DEEs).
The decision was supported by compelling data from cohort 1 of the Phase 2 EMBOLD study, which investigated relutrigine in a highly treatment-resistant population. On average, these patients had already failed three previous therapies.
Results from the 16-week double-blind period showed a placebo-adjusted monthly motor seizure reduction of 46%, with over 30% of patients achieving seizure freedom.
Extended findings from the 11-month open-label extension indicated an average seizure reduction of around 90% and a sustained improvement in seizure-free periods, with patients experiencing an average of 67 days between seizures, compared with only 3 days at baseline. Improvements in alertness, communication and seizure severity were also observed.
Importantly, relutrigine continued to show a favorable safety profile throughout the trial, with no new safety concerns, no drug-related serious adverse events, and no need for dose reductions.
The EMBOLD registrational cohort 2 trial is ongoing and remains on track for topline data readout in the first half of 2026. A New Drug Application (NDA) submission is expected to follow.
Relutrigine is being developed as a potential targeted treatment option for patients with DEEs, a group of rare, severe epilepsy syndromes that often emerge early in life and are marked by drug-resistant seizures and developmental impairments.
The Breakthrough Therapy Designation is expected to accelerate its path toward regulatory approval.
