Stiripentol is an anticonvulsant drug used in the treatment of epilepsy.
Marketed as Diacomit.
It is approved for the treatment of Dravet syndrome.
Dravet syndrome is a serious genetic brain disorder.
Pregnancy category AU: B3
Routes of administration By mouth
The drug is unrelated to other anticonvulsants and belongs to the group of aromatic allylic alcohols.
It is indicated for use in conjunction with clobazam and valproate as adjunctive therapy of refractory generalized tonic-clonic seizures in people with severe myoclonic epilepsy in infancy whose seizures are not adequately controlled with clobazam and valproate.
It is indicated for the treatment of seizures associated with Dravet syndrome in people two years of age and older taking clobazam.
There are no clinical data to support the use of stiripentol as monotherapy in Dravet syndrome.
It may be used as an add-on therapy with sodium valproate and clobazam for treating children with Dravet syndrome whose seizures are not adequately controlled.
Stiripentol must not be used in people who have had psychosis, or attacks of delirium.
More than 10% of people have adverse effects include loss of appetite, weight loss, insomnia, drowsiness, ataxia, hypotonia, and dystonia.
Common adverse effects include neutropenia, aggressiveness, irritability, behavior disorders, opposing behavior, hyperexcitability, sleep disorders, hyperkinesias, nausea, vomiting, and elevated gamma-glutamyltransferase.
Stiripentol inhibits several cytochrome P450 isoenzymes and so interacts with many anticonvulsants and other medicines.
Its precise mechanism of action is unknown.
It is not known whether it remains useful as children become adolescents nor as they become adults.
Stiripentol increases GABAergic activity, enhancing central GABA neurotransmission through a barbiturate-like effect it increasing the duration of opening of GABA-A receptor channels in hippocampal slices.
It increases GABA levels in brain tissues by interfering with its reuptake and metabolism.
It inhibits lactate dehydrogenase, which is an important enzyme involved in the energy metabolism of neurons, making neurons less prone to fire action potentials, likely through activation of ATP-sensitive potassium channels.
Stiripentol improves the effectiveness of many other anticonvulsants, possibly by inhibiting enzymes that slow the drugs’ metabolism and increasing blood plasma levels.
Stiripentol is the only medication for which a double-blind placebo-controlled randomized controlled trial was performed and showed efficacy in trials.
Stiripentol acts as a GABAergic agent and as a positive allosteric modulator of GABAA receptor.
Stiripentol, can improve focal refractory epilepsy, as well as Dravet’s syndrome.
Adverse effects include loss of appetite, weight loss, insomnia, drowsiness, ataxia, hypotonia, dystonia, neutropenia, aggressiveness, irritability, behavior disorders, opposing behavior, hyperexcitability, sleep disorders, hyperkinesias, nausea, vomiting, and elevated gamma-glutamyltransferase.
It inhibits several cytochrome P450 isoenzymes and so interacts with many anticonvulsants and other medicines.
Stiripentol increases GABAergic activity.
Stiripentol enhances central GABA neurotransmission by increasing the duration of opening of GABA-A receptor channels in the hippocampus.
It increases GABA levels in brain tissues by interfering with its reuptake and metabolism.
It inhibits lactate dehydrogenase, which is an important enzyme involved in the energy metabolism of neurons.
Inhibition of lactate dehydrogenase enzyme can make neurons less prone to fire action potentials by activating of ATP-sensitive potassium channels.
It also improves the effectiveness of other anticonvulsants.