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Stiripentol is an anticonvulsant medication primarily used as adjunctive therapy for the treatment of seizures associated with Dravet syndrome, a severe genetic epilepsy that begins in early childhood. It is indicated for use in combination with clobazam (and often valproate) when seizures are not adequately controlled by these agents alone[1][2][5]. Stiripentol acts through multiple mechanisms, including potentiation of GABAergic transmission as a positive allosteric modulator of the GABA-A receptor, inhibition of voltage-gated sodium and calcium channels, and inhibition of lactate dehydrogenase (LDH), which affects neuronal energy metabolism[6][7][8]. Additionally, it inhibits cytochrome P450 enzymes (notably CYP1A2, CYP2B6, CYP3A4), thereby increasing blood levels and efficacy of other antiepileptic drugs such as clobazam[4][6]. Stiripentol may also have neuroprotective effects via modulation of protein-l-isoaspartyl methyltransferase activity[8].
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