Ganaxolone is a 3β-methylated synthetic analog of the neurosteroid allopregnanolone that allosterically enhances GABAA receptor current. At nanomolar concentrations, it can potentiate GABA-evoked currents at GABAA receptor complexes containing α1, α2, α3, β2, and γ2L subunits expressed in Xenopus oocytes. Ganaxolone has been shown to have a broad range of antiseizure activity in animal epilepsy models and human clinical trials.
Treatment of epilepsy and migraine.
Ganaxolone, is synthetic neuroactive steroid, which possess sedative, anxiolytic, and anticonvulsant properties. It is also a potent and selective positive allosteric modulator of GABAA receptors. It is also shown to be used for the treatment of epilepsy.
ChEBI: Ganaxolone is a corticosteroid hormone.
Potent positive allosteric modulator of GABA A receptors. Enhances GABA-evoked chloride currents in Xenopus oocytes expressing GABA A receptors (EC 50 values are 94, 122 and 213 nM for α 2 β 1 γ 2 L , α 3 β 1 γ 2 L and α 1 β 1 γ 2 L receptors respectively). Exerts anticonvulsive effects in a broad range of animal seizure models.
Ganaxolone (3alpha-hydroxy-3beta-methyl-5alpha-pregnane-20-one) is a positive allosteric modulator of the GABAA receptor subtype; synthetic analog of the endogenous neurosteroid allopregnanolone; effective against chemically induced seizures in rats and mice. Ganaxolone is an orally active analog of allopregnanolone that is not converted to the hormonally active 3-keto form. The enhanced anticonvulsant potency of ganaxolone after neurosteroid withdrawal supports the use of ganaxolone as a specific treatment for perimenstrual catamenial epilepsy.
