Stoke Therapeutics reviews 5-year data at FDA meeting
- Stoke Therapeutics announced the successful completion of an FDA meeting regarding its zorevunersen drug application.
- The regulatory alignment on Phase 3 trial data endpoints resulted in the company's share price trading higher.
- The company expects to submit a New Drug Application in the second half of 2027 to support potential approval.
Stoke Therapeutics (NASDAQ:STOK) announced the successful completion of a meeting with the U.S. Food and Drug Administration regarding its investigational zorevunersen treatment.
The regulatory meeting aligned key aspects of the planned New Drug Application submission based on 5 years of accumulated clinical safety and efficacy data.
"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," said Stoke Therapeutics Chief Executive Officer and Director Ian F. Smith.
The biotechnology company will now utilize a multicomponent assessment of four prespecified Vineland-3 subdomains to evaluate patient cognition and behavior in the ongoing Phase 3 EMPEROR study.
Following the announcement, Stoke Therapeutics' share price was up at $25.77.
The Phase 3 EMPEROR study randomizes participants to receive either zorevunersen via intrathecal administration or a sham comparator for a 52-week treatment period.
The primary treatment effect will evaluate the estimated change in major motor seizure frequency from baseline through the interval immediately preceding week 28.
