Stiripentol.
Stiripentol (STP) is a new antiepileptic compound made by Biocodex. It recently proved to increase the GABAergic transmission in vitro in an experimental model of immature rat. Clinical studies were based on the fact that STP also acts as an inhibitor of CYP3A4, CYP1A2, and CYP2C19 in vivo in epileptic patients. Whereas the studies in adult patients were disappointing, the trials conducted in pediatric populations demonstrated a specific efficacy of STP in severe myoclonic epilepsy in infancy, Dravet syndrome, when combined with valproate and clobazam. Based on these results, STP was granted orphan drug status in the European Union for the treatment of Dravet syndrome. The French experience in compassionate use suggests that STP might also be of benefit when combined with carbamazepine in pediatric patients with pharmacoresistant partial epilepsy. The interactions of STP with a large number of drugs need to be carefully taken into account, with doses of the combined antiepileptic drugs adjusted to improve the tolerability of the therapeutic association.
Chiron
Catherine
C
INSERM, U663, Service de Neurologie et Métabolisme, Hôpital Necker, 149 rue de Sèvres, 75015 Paris, France. catherine.chiron@nck.aphp.fr
D000818:Animals; D000927:Anticonvulsants; D001921:Brain / Q000187:drug effects; D002648:Child; D002986:Clinical Trials as Topic; D004148:Dioxolanes; D004827:Epilepsy / Q000188:drug therapy*; D006801:Humans
D000927:Anticonvulsants; D004148:Dioxolanes; C021092:stiripentol