Zorevunersen Seems Safe, Yields Clinical Improvement in Dravet Syndrome

MONDAY, March 9, 2026 (HealthDay News) -- For pediatric patients with Dravet syndrome, zorevunersen, an antisense oligonucleotide that upregulates NaV1.1 sodium channels, seems safe and demonstrates initial clinical improvement, according to a study published in the March 5 issue of the New England Journal of Medicine.Linda Laux, M.D., from the Northwestern University Feinberg School of Medicine in Chicago, and colleagues enrolled 81 patients aged 2 to 18 years with Dravet syndrome in two phase 1 to 2a, open-label, multicenter studies. The studies included a single-ascending-dose cohort (10 to 70 mg administered on day 1) and a multiple-ascending-dose cohort (20 to 70 mg administered two or three times in a three-month period). Seventy-five patients were then enrolled in two open-label extension studies and continued to receive zorevunersen (≤45 mg) every four months.The researchers found that most adverse events were mild or moderate; the most common was post-lumbar puncture syndrome in the phase 1 to 2a studies and elevated protein level in the cerebrospinal fluid in the extension studies (25 and 45 percent, respectively). Suspected unexpected serious adverse reactions occurred in one patient, and one had an adverse event leading to study withdrawal. Three patients died: two from sudden unexpected death in epilepsy and one from malnutrition. The median change from baseline in convulsive-seizure frequency ranged from −58.82 to −90.91 percent across one-month intervals during the first 20 months of the extension studies among patients who received 70 mg in the phase 1 to 2a studies and up to 45 mg in the extension studies. With continued treatment for up to 36 months in the extension studies, the data supported improvements in overall clinical status, quality of life, and adaptive behavior."Our data support zorevunersen safety and tolerability, as well as improvement in overall clinical status, quality of life and adaptive behavior following continued dosing in the extension studies," Laux said in a statement.Several authors disclosed ties to biopharmaceutical companies, including Stroke Therapeutics, which is developing zorevunersen and funded the study.Abstract/Full Text (subscription or payment may be required)

Zorevunersen Seems Safe, Yields Clinical Improvement in Dravet Syndrome

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