Praxis Precision Medicines Introduces Breakthrough Relutrigine Therapy
Praxis Precision Medicines has introduced relutrigine as a first-in-class investigational therapy to receive U.S. FDA Breakthrough Therapy designation for the treatment of seizures associated with SCN2A and SCN8A-related developmental and epileptic encephalopathies (DEEs).
Relutrigine is a sodium channel functional state modulator designed to target persistent sodium currents that drive seizure activity. It aims to restore the balance between neuronal excitation and inhibition, helping to reduce seizure frequency and severity in affected children.
There are currently no approved therapies that specifically target the underlying sodium channel dysfunction in SCN2A and SCN8A-related DEEs, which are rare, severe, and often resistant to standard treatments.
The FDA’s Breakthrough Therapy designation highlights the strong clinical potential of relutrigine. It was granted based on data from the EMBOLD Phase 2 study, where patients experienced an average seizure reduction of approximately 90% in the open-label extension and extended periods between seizures. Improvements in communication, alertness, and overall seizure severity were also observed, with no new safety concerns reported.
Relutrigine has also received Orphan Drug Designation and Rare Paediatric Disease Designation from the FDA for SCN2A-DEE, SCN8A-DEE, and Dravet syndrome. The therapy holds Orphan Drug Designation from the European Medicines Agency as well.
The EMBOLD registrational cohort 2 study is currently ongoing, with topline results expected in the first half of 2026. Additionally, Praxis has launched the EMERALD study to evaluate relutrigine in a broader DEE population regardless of genetic cause.
Relutrigine is the first and only investigational therapy to receive U.S. FDA Breakthrough Therapy designation for the treatment of SCN2A and SCN8A-related developmental and epileptic encephalopathies.
