Praxis Says FDA Grants Breakthrough Therapy Designation To Relutrigine For Rare Pediatric Epilepsies

(RTTNews) - Biopharmaceutical company Praxis Precision Medicines, Inc. (PRAX) announced Thursday that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for relutrigine, a sodium channel functional state modulator for pediatric use for the treatment of patients with SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs).

The BTD was granted based on the positive data from cohort 1 in the Phase 2 EMBOLD study, as well as 11-month data from the open-label extension (OLE) period of the trial.

The company said the EMBOLD cohort 2 pivotal trial is currently ongoing and continues to enroll, with topline results in the first half of 2026 with NDA filing to follow.

Praxis has recently initiated the EMERALD study investigating relutrigine broadly in DEEs.