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 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 shares were up over 10% in recent premarket activity.
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