Praxis Precision Medicines Receives FDA Breakthrough Therapy Designation for Elsunersen for the Treatment of Seizures Associated with SCN2A Developmental and Epileptic Encephalopathy Caused by Gain of Function Variants
Praxis Precision Medicines secured FDA Breakthrough Therapy Designation for elsunersen (PRAX-222) for SCN2A-DEE, driven by topline data showing a 77% sham-adjusted reduction in monthly seizures in the Phase 1/2 EMBRAVE Part A study. The designation enables a streamlined, single-arm design for the pivotal EMBRAVE3 study, which is enrolling approximately 30 patients to evaluate changes in countable motor seizures over 24 weeks.
Key figures
- Drug Name
- elsunersen (PRAX-222)
- Phase Of Trial
- Phase 1/2
- P value
- 0.015
- Embrave part a patients
- 9
- Embrave3 patients target
- 30
- Seizure reduction percent
- 77%
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