Breakthrough Designation Granted for Zorevunersen to Treat Individuals with Dravet Syndrome

Zorevunersen (STK-001; Stoke Therapeutics, Bedford, MA), an antisense oligonucleotide (ASO) that acts on the NaV1.1 protein, has been granted Breakthrough Therapy designation by the Food and Drug Administration (FDA) for the treatment of individuals with Dravet syndrome with a mutation of the SCN1A gene not associated with gain-of-function. The decision was based on phase 1/2a clinical study and open-label extension data showing that treatment with zorevunersen was associated with reductions in seizure frequency and improvements in measures of cognition and behavior for people with Dravet syndrome. Results of the clinical trial were presented at the American Epilepsy Society (AES) 2024 Annual Meeting.

According to Mary Anne Meskis, Executive Director, Dravet Syndrome Foundation, “This designation brings new hope to the many patients with Dravet syndrome who continue to experience treatment-resistant seizures and a myriad of health and quality of life complications despite the availability of symptomatic treatments.”

Breakthrough Therapy designation is intended to expedite the development and review of drugs that treat serious conditions with positive preliminary clinical evidence. Products that receive Breakthrough Therapy designation are granted all the features of Fast Track designation as well as guidance in an efficient drug development program.