Stiripentol (STP), an antiseizure medication (ASM), is primarily used as an adjunctive therapy for seizures associated with Dravet syndrome (DS), a severe form of childhood epilepsy. Its mechanisms of action (MoAs) include potentiating gamma-aminobutyric acid (GABA) neurotransmission through uptake blockade, inhibition of degradation, and positive allosteric modulation of GABA receptors, particularly those containing α3 and δ subunits. STP also blocks voltage-gated sodium and T-type calcium channels, contributing to its anticonvulsant and neuroprotective properties. Additionally, STP regulates glucose energy metabolism by inhibiting glucose-6-phosphate dehydrogenase (G6PD) and lactate dehydrogenase (LDH), and it inhibits several cytochrome P450 enzymes, affecting the metabolism of other ASMs. These MoAs make STP a promising treatment for DS and potentially other neurological and non-neurological diseases. Recent studies have explored its efficacy in combination with other ASMs and its potential in treating conditions like primary hyperoxaluria and glioblastoma. The multitargeted nature of STP's MoAs suggests a strong therapeutic potential, with ongoing research aiming to further understand and capitalize on these mechanisms.Stiripentol (STP), an antiseizure medication (ASM), is primarily used as an adjunctive therapy for seizures associated with Dravet syndrome (DS), a severe form of childhood epilepsy. Its mechanisms of action (MoAs) include potentiating gamma-aminobutyric acid (GABA) neurotransmission through uptake blockade, inhibition of degradation, and positive allosteric modulation of GABA receptors, particularly those containing α3 and δ subunits. STP also blocks voltage-gated sodium and T-type calcium channels, contributing to its anticonvulsant and neuroprotective properties. Additionally, STP regulates glucose energy metabolism by inhibiting glucose-6-phosphate dehydrogenase (G6PD) and lactate dehydrogenase (LDH), and it inhibits several cytochrome P450 enzymes, affecting the metabolism of other ASMs. These MoAs make STP a promising treatment for DS and potentially other neurological and non-neurological diseases. Recent studies have explored its efficacy in combination with other ASMs and its potential in treating conditions like primary hyperoxaluria and glioblastoma. The multitargeted nature of STP's MoAs suggests a strong therapeutic potential, with ongoing research aiming to further understand and capitalize on these mechanisms.