Stiripentol Approved by FDA for Treatment of Dravet Syndrome
The Food and Drug Administration (FDA) has approved stiripentol (Diacomit; Biocodex, Gentilly, France) as an adjunct to clobazam for the treatment of seizures associated with Dravet syndrome (DS). Stiripentol has been previously approved in 27 European countries, Japan, and Canada as an adjunctive treatment for children age 2 and up taking clobazam or valproate for the seizures associated with DS. The FDA approval of stiripentol is also for use in children age 2 and up, and the drug is expected to be available in US pharmacies in January, 2019.
Approval of stiripentol is based on data from 2 multicenter placebo-controlled trials, STICLO France and STICLO Italy that both had primary endpoints of responder rate, with response defined as a 50% or more reduction in seizure frequency from baseline over a 30-day treatment period. In STICLO France, the responder rate for patients treated with stiripentol was 71% (95% confidence interval [CI]: 52%-91%], compared to 5% (95% CI: 0%-15%] for patients receiving placebo. In STICLO Italy, the responder rate for patients receiving stiripentol was 67% (95% CI: 40%- 93%), compared to 9.1% (95% CI: 0%-26%) for patients receiving placebo.
The mechanism of action for stiripentol is thought to be direct effects mediated through the GABAA receptor and indirect via cytochrome P450 activity with resulting increases in blood levels of clobazam and its active metabolite.
Jean-Marie Lefèvre, President and CEO of Biocodex said, "Diacomit is the result of Biocodex's own research and has been the subject of substantial investment. Our research work, along with the preclinical and clinical studies carried out in close collaboration with teams of university experts from around the world, have enabled us to obtain approval in Europe, Japan, Canada and, as of now, in the US. Our efforts will not stop there. We are continuing our research on stiripentol in order to do everything in our power to improve treatment for children and adults suffering from pharmacoresistant epilepsy."