Praxis Precision Medicines' Elsunersen Gets FDA Breakthrough Therapy Designation

Praxis Precision Medicines, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for elsunersen (PRAX-222). This designation is for the treatment of seizures associated with SCN2A Developmental and Epileptic Encephalopathy (DEE) caused by Gain of Function variants in SCN2A. The BTD, which enables expedited development and regulatory review, was supported by positive topline results from the EMBRAVE Part A trial, a Phase 1/2 study.