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NewsA promising antisense drug for Dravet syndrome is advancing toward FDA approval. Stoke Therapeutics completed a key FDA meeting to align on its NDA plans for zorevunersen, an investigational therapy for children with Dravet syndrome. The phase 3 EMPEROR study will evaluate both seizure reduction and neurodevelopmental outcomes like communication and social skills.
Stoke Therapeutics has completed a productive meeting with the FDA to align on its planned New Drug Application (NDA) for zorevunersen, an antisense oligonucleotide being developed for children with Dravet syndrome — a severe, treatment-resistant epilepsy. The discussion covered safety and efficacy data from earlier studies and confirmed the analysis plan for the phase 3 EMPEROR trial.
What sets this program apart is its dual focus: the trial isn't just measuring seizure control — it's also tracking neurodevelopmental functioning. Using four Vineland-3 subdomains (expressive communication, receptive communication, interpersonal relationships, and personal skills), the study aims to capture whether zorevunersen improves children's day-to-day cognitive and social abilities, not just their seizure counts.
Key Takeaways:
Why it matters: Dravet syndrome causes lifelong cognitive and behavioral challenges beyond seizures. If EMPEROR succeeds, zorevunersen could become the first therapy to meaningfully address both seizure burden and neurodevelopment in these children — a significant leap forward for patients and families.
