Stoke Therapeutics Wins FDA Breakthrough Therapy Designation for Dravet Syndrome Treatment

Stoke Therapeutics (Nasdaq: STOK) announced that its investigational therapy, zorevunersen, has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for the treatment of Dravet syndrome with a confirmed mutation in the SCN1A gene. This designation recognizes the potential of zorevunersen to offer substantial improvement over existing treatments for this severe and progressive genetic epilepsy.

Clinical data from Phase 1/2a and open-label extension (OLE) studies demonstrated significant and sustained reductions in seizure frequency, along with improvements in cognition and behavior, even when used alongside the best available anti-seizure medications. Zorevunersen was generally well tolerated, with more than 600 doses administered to date and some patients on treatment for over three years.

Stoke is actively engaging with global regulatory agencies to finalize plans for a Phase 3 randomized, controlled registrational trial. The company expects to provide an update on these plans by year-end, marking an important step toward developing the first disease-modifying therapy for Dravet syndrome.