Abstract and Introduction
Stiripentol was approved by the Food and Drug Administration (FDA) on August 20, 2018 as adjunctive therapy with clobazam for the treatment of seizures associated with Dravet syndrome in patients 2 years of age and older.[1,2] It is not currently approved as monotherapy. Dravet syndrome, also referred to as severe myoclonic epilepsy of infancy, is a rare disorder characterized by seizures unresponsive to most antiepileptics, motor impairment, and developmental disabilities. Nearly a quarter of patients die during childhood. Approximately 70% of patients have a mutation in the sodium channel alpha-1 subunit gene (SCN1A) leading to malfunction or loss of function in GABAergic neurons. As a treatment for a rare disorder, stiripentol has been designated as an orphan drug by both the European Medicines Agency and the FDA. It has been approved for use in Europe for more than a decade, with more recent approvals in Canada and Japan.
Pediatr Pharm. 2018;24(10) © 2018 University of Virginia