What is "ganaxolone"

n. An epalon anesthetic related to allopregnanolone, with sedative, anxiolytic, and anticonvulsant effects.

Ganaxolone ( INN (developmental code name CCD-1042) is a CNS-selective GABA 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 GABA receptor and, unlike benzodiazepines, there does not appear to be 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 >1,300 patients), with the commonly reported side effects being somnolence (sleepiness), dizziness, and fatigue.Trials in adults with focal onset seizures and in children with infantile spasms have recently been completed. There are ongoing studies in patients with focal onset seizures, PCDH19 pediatric epilepsy, and behaviors in Fragile X syndrome. There are plans to conduct a study in patients with status epilepticus.