Ganaxolone (
INN, also known as
CCD-1042 and
3a-Hydroxy-3ß-methyl-5a-pregnan-20-one) is a CNS-selective GABA
A modulator that acts on well-characterized targets in the brain known to have
anxiolytic and
anticonvulsant effects. Ganaxolone protects against seizures in diverse animal models, including the pentylenetetrazol, 6 Hz and amygdala kindling models. Ganaxolone is a
positive allosteric modulator of the action of the
GABAA receptor and, unlike
benzodiazepines, there does not appear to be tolerance to the anticonvulsant effects of ganaxolone.