Elsunersen

Drug

Drug Profile

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
Brand Names
  • elsunersen
  • PRAX-222
Indications
  • \Early-seizure-onset SCN2A developmental and epileptic encephalopathy (SCN2A-DEE)\

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