Overview
- Peer‑reviewed Phase 1/2a and open‑label extension studies in the U.K. and U.S. enrolled 81 children aged 2–18, with 75 continuing in extensions.
- Children given the highest initial 70 mg dosing recorded 59–91% seizure reductions over the first 20 months of follow‑up, alongside gains in language, motor skills, behavior and quality of life.
- Zorevunersen was administered by lumbar puncture, and patients in extension studies received 45 mg every four months.
- Most adverse events were mild to moderate, with post‑lumbar puncture syndrome common initially and cerebrospinal fluid protein increases frequent in extensions, and one serious event deemed treatment‑related.
- A double‑blind, placebo‑controlled Phase 3 trial is in progress to confirm efficacy and safety, and if approved the drug would be the first gene‑regulation therapy for epilepsy.