What is the role of stiripentol (Diacomit) in the treatment of epilepsy?

Updated: Jan 28, 2020
  • Author: Juan G Ochoa, MD; Chief Editor: Selim R Benbadis, MD  more...
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Answer

Answer

The FDA approved stiripentol (Diacomit) in August 2018 for treatment of seizures associated with Dravet syndrome in patients aged 2 years or older who are taking clobazam. There are no clinical data to support the use of stiripentol as monotherapy in Dravet syndrome.

Stiripentol belongs to the group of aromatic allylic alcohols and is unrelated to other anticonvulsants. The precise anticonvulsant effect in humans is unknown. Possible mechanisms of action include direct effects mediated through the GABA-A receptor and indirect effects involving inhibition of cytochrome P450 activity with resulting increase in blood levels of clobazam and its active metabolite.

Approval was based on 2 multicenter placebo-controlled double-blind randomized studies, conducted according to similar protocols. To be enrolled in either study, patients were required to be aged 3 years to less than 18 years, to have Dravet syndrome (ILAE classification of epilepsy, 1989), and to be inadequately controlled on clobazam and valproate, with at least 4 generalized clonic or tonic-clonic seizures per month despite optimized therapy. In Study 1 (N=41), 71% of stiripentol-treated patients were responders vs 5% in the placebo group (P < 0.0001), while in Study 2 (N=23), 67% of stiripentol-treated patients were responders vs 9.1% in the placebo arm (P < 0.0094). Treatment with stiripentol was also superior to placebo for the reduction in mean frequency of generalized clonic or tonic-clonic seizures. [98]


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