Stoke Therapeutics Announces Completion of Successful Meeting with the FDA to Align on Planned U.S. NDA for Zorevunersen, an Investigational Medicine for the Treatment of Dravet Syndrome
Business Wire•05/10/2026•07:00 ET
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Key Highlights
- ➤FDA alignment supports planned U.S. NDA for zorevunersen in second half of 2027
- ➤Phase 3 EMPEROR readout anticipated in third quarter of 2027
- ➤Vineland-3 cognition and behavior endpoint will assess four subdomains using MMRM
- ➤Potential U.S. approval and zorevunersen launch targeted for early 2028
- ➤Biogen holds exclusive commercialization rights outside the United States, Canada, and Mexico
Expert Statements
Ian F. Smith, Chief Executive Officer and Director of Stoke Therapeutics
“We are encouraged by our productive dialogue with the FDA and the Agency’s deepening understanding of the severity of Dravet syndrome and need for better treatments, and 5 years of safety and efficacy data for zorevunersen,”
Ian F. Smith, Chief Executive Officer and Director of Stoke Therapeutics
“We are pleased with the Agency’s willingness to accept a multicomponent assessment of Vineland-3 data measuring changes in cognition and behavior from patients treated in the Phase 3 EMPEROR study.”
Ian F. Smith, Chief Executive Officer and Director of Stoke Therapeutics
“We believe this is the most appropriate way to capture the effects of zorevunersen on people with Dravet syndrome who experience a wide range of debilitating neurodevelopmental impacts.”
Ian F. Smith, Chief Executive Officer and Director of Stoke Therapeutics
“We look forward to continuing our interactions with the FDA as we advance the EMPEROR study and prepare for the readout of data in the third quarter of 2027, followed quickly by the completion of our NDA submission in the second half of 2027.”
Stoke Therapeutics Announces Completion of Successful Meeting with the FDA to Align on Planned U.S. NDA for Zorevunersen, an Investigational Medicine for the Treatment of Dravet Syndrome
– Analysis of the Phase 3 EMPEROR key secondary endpoint of changes in cognition and behavior will be performed using a multicomponent assessment of four Vineland-3 subdomains –
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