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 has received FDA Breakthrough Therapy Designation (BTD) for elsunersen (PRAX-222), an antisense oligonucleotide targeting seizures in patients with SCN2A Developmental and Epileptic Encephalopathy (SCN2A-DEE) due to gain-of-function variants. The designation was based on positive results from the EMBRAVE Part A trial, whi…