Drug intelligence / Profile preview

stiripentol

Development stage
Approved
Lead developer
Biocodex
Modality
Small Molecules
Administration
Oral
01

Overview

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].

Brand names
Diacomit
Other names
STP
02

Targets

LDH (L-lactate dehydrogenase A chain)NaV (Voltage-gated sodium channels)CaV3 (Caveolin-3)GABAA (Gamma-aminobutyric acid receptor)Cyp (Cyclophilin family)

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