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Ganaxolone is a synthetic neuroactive steroid and a positive allosteric modulator of the gamma-aminobutyric acid type A (GABA-A) receptor. It is used for the treatment of seizures associated with cyclin-dependent kinase-like 5 deficiency disorder (CDKL5), an inherited condition that causes early-onset seizures and developmental delays in children. Ganaxolone acts by enhancing GABAergic inhibition in the central nervous system, binding to both synaptic and extrasynaptic GABA-A receptors at sites distinct from benzodiazepines or barbiturates, which results in reduced neuronal excitability. This dual action on synaptic and extrasynaptic receptors differentiates it from other GABA-potentiating antiseizure drugs. Ganaxolone is available as an oral suspension under the brand name Ztalmy[1][2][3][4][5][6].
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