Ganaxolone (Gan)

Ganaxolone (CCD-1042) is a synthetic neuroactive steroid related to allopregnanolone that has sedative, anxiolytic, and anticonvulsant effects. It is a potent and selective positive allosteric modulator of GABAA receptors. Ganaxolone protects against seizures in diverse animal models, including the pentylenetetrazol, 6 Hz and amygdala kindling models. Unlike for benzodiazepines, there is no tolerance to the anticonvulsant effects of ganaxolone.

Ganaxolone is being investigated for potential medical use in the treatment of epilepsy. It is well tolerated in human trials (with exposure of >900 patients), with the main side-effects being sedation, dizziness, and headache. Trials in adults with partial seizures and in infantile spasms have recently been completed.