Diacomit® (Stiripentol) - new option in the therapy of Dravet syndrome

Treatment of patients with Dravet syndrome, also called "Severe Myoclonic Epilepsy in Infancy"(SMEI), is difficult, as the disease is resistant against a multitude of anti-epileptics. Since January 2008 the preparation Diacomit® (active substance: Stiripentol) from the company Desitin Arzneimittel GmbH, Hamburg, is now also available for therapy in Germany. At the beginning of December 2001 Stiripentol was first assigned approval by the EMEA as "Orphan drug" in combination with the anti-epileptics Clobazam and Valproat for use in severe myoclonic epilepsy in infants (SMEI) and was licensed as "Orphan drug" in 2007. Stiripentol functions as a direct GABA receptor agonist, by blocking synaptosomal uptake of the neurotransmitter, and as an indirect anti-epileptic by inhibiting several hepatic cytochrome-P450 isoenzymes, which causes an increase in the concentration of associated anti-epileptics. The two most important controlled approval studies for therapy of Dravet syndrome are the double-blind, placebo-controlled and randomized multicenter studies STICLO-FR and STICLO-IT. In both studies it could be shown, that the frequency of fits under therapy with Stiripentol significantly decreases. Because of the pharmacokinetic properties of Stiripentol, during treatment the dose of the accompanying medicines such as Valproat and Clobazam, should be reduced by approximately half. The opportunity for an exclusive interview with the scientist Catherine Chiron, MD, PhD, Inserm U663, Section of Neurology and Metabolism Necker Hospital, Paris, about the new anti-epileptic Diacomit® and her experiences with Stiripentol in France, presented itself to the editors of NeuroNews.de during a symposium, which had been organized by the company Desitin at the beginning of April 2008 in Jena within the cadre of the 34^th annual conference of the Society for Neuropediatrics.

full article