FDA Grants Breakthrough Therapy Designation to Praxis’s Elsunersen for SCN2A-DEE

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Praxis Precision Medicines shares rose after the U.S. FDA granted Breakthrough Therapy Designation to Elsunersen for treating seizures in SCN2A-DEE, a rare pediatric neurological disorder. The decision was based on Part A of the EMBRAVE study, where patients treated with Elsunersen experienced a 77% reduction in monthly seizure frequency compared with the sham control group, with improvements maintained for up to one year in the open-label extension. In the Phase 1/2 trial, 71% of treated pediatric patients achieved more than a 50% reduction in seizure frequency, and 57% experienced at least one 28-day seizure-free period during the six-month treatment phase. Praxis is continuing enrollment in its EMBRAVE3 registrational trial, a single-arm, baseline-controlled study of approximately 30 patients, under a streamlined development pathway agreed upon with the FDA. Elsunersen has also received Orphan Drug and Rare Pediatric Disease Designations in the U.S., as well as Orphan Drug and PRIME status in Europe. The stock is currently trading at $299.33, up 6.03%.

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