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News
A new analysis shows that zorevunersen, an investigational drug for Dravet syndrome, does more than reduce seizures — it meaningfully improves children's communication, social, and daily living skills. Compared to kids on standard therapy, those treated with zorevunersen showed significant gains across multiple developmental measures over roughly two years. A pivotal phase 3 trial is fully enrolled, with results expected mid-2027.
Zorevunersen, an investigational antisense oligonucleotide developed by Stoke Therapeutics and Biogen, is showing promise beyond seizure control in children with Dravet syndrome (DS). A matching-adjusted indirect comparison presented at the 16th European Epilepsy Congress found that DS patients treated with zorevunersen showed substantial, durable improvements in adaptive functioning — including communication, social relationships, and daily living skills — over approximately two years. In contrast, natural history patients on standard antiseizure therapy showed minimal change across the same measures.
The drug's long-term safety profile, spanning over 260 patient-years and 930+ doses, appears manageable. The most common side effect was cerebrospinal fluid (CSF) protein elevation, though no serious clinical consequences were linked to it. Three deaths were reported but all were assessed as unrelated to the drug.
By the Numbers:
Why it matters: Dravet syndrome affects nearly every aspect of a child's development, not just seizure frequency. Evidence that zorevunersen can narrow the developmental gap between these children and their neurotypical peers could be a meaningful step forward for families and clinicians managing this complex, lifelong condition.
