Elsunersen (PRAX-222) is an investigational antisense oligonucleotide therapy in development for early-seizure-onset SCN2A developmental and epileptic encephalopathy. It is designed to reduce SCN2A expression in gain-of-function disease and has shown positive Phase 1/2 results with progression into pivotal EMBRAVE3 development. The therapy remains unapproved.
- Drug Class
- Antisense oligonucleotide (ASO)
- Approval Status
-
Investigational; positive Phase 1/2 data with pivotal EMBRAVE3 underway
- Mechanism of Action
- Selectively decreases SCN2A gene expression to target gain-of-function SCN2A disease biology
Indications
- \Early-seizure-onset SCN2A developmental and epileptic encephalopathy (SCN2A-DEE)\