Four years of durable OLE data and Phase 3 enrollment complete — zorevunersen's NDA case hinges on whether Phase 3 confirms the signal

Stoke Therapeutics is a clinical-stage company developing zorevunersen, a gene-targeting drug for Dravet syndrome, a severe childhood seizure disorder, which is not yet approved.

Zorevunersen, an antisense oligonucleotide that upregulates the intact SCN1A allele to reduce seizures in Dravet syndrome, has 162 patients enrolled with zero discontinuations and four years of open-label efficacy data supporting BTD. Phase 3 data readout determines whether that signal replicates in a controlled trial, which is the sole condition for NDA completion. The approval path itself carries risk: the NDA will rest on a single Phase 3, and the FDA's prior request for additional information after the BTD multidisciplinary meeting signals the agency has not yet fully aligned on the data package.