Praxis Precision Medicines Receives FDA Breakthrough Therapy Designation for Elsunersen
Praxis Precision Medicines has received US Food and Drug Administration (FDA) Breakthrough Therapy Designation for elsunersen (PRAX-222) to treat seizures associated with SCN2A Developmental and Epileptic Encephalopathy (SCN2A-DEE) caused by gain-of-function variants in the SCN2A gene.
The designation is based on positive results from the Phase 1/2 EMBRAVE Part A study, which evaluated elsunersen in children aged two to 12 years with early-seizure-onset SCN2A-DEE.
The treatment achieved a 77% sham-adjusted reduction in monthly seizures from baseline. More than 70% of treated patients experienced a seizure reduction of over 50%, while over half achieved at least 28 consecutive days without seizures.
Improvements were also reported in areas including sleep, motor function, muscle tone, attention and neuropsychomotor development.
Elsunersen was generally well tolerated, with no drug-related serious adverse events or treatment discontinuations reported.
Following discussions with the FDA, the ongoing EMBRAVE3 registrational study has been redesigned as a single-arm, baseline-controlled trial involving approximately 30 patients.
Elsunersen has also received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA, as well as Orphan Drug Designation and PRIME designation from the European Medicines Agency.
