FDA grants Breakthrough Therapy Designation for Praxis Precision Medicines' elsunersen for SCN2A-DEE

Praxis Precision Medicines, Inc.

• FDA granted Breakthrough Therapy Designation for elsunersen (PRAX-222), an antisense oligonucleotide for seizures in SCN2A Developmental and Epileptic Encephalopathy.
• BTD expedites development and regulatory review; supported by positive topline data from EMBRAVE Part A trial.
• EMBRAVE Part A was a randomized, sham-controlled Phase 1/2 study in nine pediatric patients aged 2–12 years with early-seizure-onset SCN2A-DEE.
• The designation covers Gain of Function variants in SCN2A.