08:11 AM EST, 12/04/2024 (MT Newswires) -- Stoke Therapeutics ( STOK ) said Wednesday it has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration for zorevunersen as treatment for Dravet syndrome with a confirmed mutation in the SCN1A gene.

Phase 1/2a and open-label extension studies of zorevunersen showed "substantial and sustained reductions in seizure frequency" and improvements in cognitive and behavioral measures, the company said.

Stoke Therapeutics ( STOK ) said it is in talks with the FDA and other regulatory bodies about advancing to a global, randomized, controlled phase 3 study and that it will provide an update on the phase 3 registrational plans by year-end.

Stoke Therapeutics ( STOK ) shares were up over 10% in recent premarket activity.