Rufinamide is an orally active, structurally novel compound (1-[(2,6-difluorophenil1) methyl1]-1 hydro 1,2,3-
triazole-4 carboxamide), which is structurally distinct from other
anticonvulsant drugs. It was granted orphan drug status for the adjunctive treatment of
Lennox-Gastaut syndrome (LGS) in the United States in 2004, and released for use in Europe in 2007. In January 2009,
rufinamide was approved by the United States Food and Drug Administration for treatment of LGS in children 4 years of age and older. It is also approved for adjunctive treatment for
partial seizures in adults and adolescents.
Rufinamide's efficacy mainly against atonic/
tonic seizures in patients with LGS seems nowadays indubitable and has been confirmed both in randomized controlled trial and in open label extension studies. More recently,
rufinamide was evaluated for the adjunctive treatment of childhood-onset epileptic
encephalopathies and
epileptic syndromes other than LGS, including epileptic
spasms, multifocal epileptic
encephalopathy with
spasm/
tonic seizures,
myoclonic-astatic epilepsy,
Dravet syndrome and malignant migrating
partial seizures in infancy. This review updates the existing literature data on the efficacy and safety/tolerability of
rufinamide in childhood-onset
epilepsy syndromes.