Stiripentol for the treatment of seizures associated with Dravet syndrome.

Stiripentol for the treatment of seizures associated with Dravet syndrome.
Expert Rev Neurother. 2019 Mar 22;:
Authors: Chiron C
Abstract
Introduction Stiripentol is an orphan drug approved for the treatment of seizures associated with Dravet syndrome (since 2007 in Europe). Therapeutic options recently grew in this rare and severe early-onset epilepsy with the approval of stiripentol and cannabidiol in 2018 in the US and the positive trials just completed with fenfluramine. Areas covered First, the short-term efficacy of stiripentol as adjunctive therapy to clobazam and valproate, which was discovered by serendipity thanks to a basket study and then confirmed in 1998 despite the small number of samples in phase III trials. Second, the further observational series worldwide, which showed sustained efficacy and satisfactory tolerability for up to 20-year exposure. Third, why it took more than 20 years for stiripentol be approved in a number of countries despite these extensive data: drug-drug interactions between stiripentol and comedication will be addressed, as well as the experimental and pharmacogenetic data which support the anticonvulsant effect of stiripentol per se. Expert opinion Considering current and future competitors (cannabidiol and fenfluramine), efficacy seems lower for cannabidiol and seizure freedom seems occasionally be obtained with fenfluramine. Additionally, stiripentol could be especially useful in two critical conditions of the di...

Source: Expert Review of Neurotherapeutics - March 23, 2019 Category: Neurology Tags: Expert Rev Neurother Source Type: research