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Systematic (IUPAC) name
Legal status
Legal status
  • investigational
CAS Number 38398-32-2
ATC code none
PubChem CID 6918305
Chemical data
Formula C22H36O2
Molar mass 332.520 g/mol[[Script error: No such module "String".]]
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Ganaxolone (INN, also known as CCD-1042) is a steroid drug related to allopregnanolone which has sedative, anxiolytic and anticonvulsant effects. It is a potent and selective positive allosteric modulator of GABAA receptors.[1] Ganaxolone protects against seizures in diverse animal models, including the 6 Hz and amygdala kindling models.[2][3][4] Unlike for benzodiazepines, there is no tolerance to the anticonvulsant effects of ganaxolone [5]

Ganaxolone is being investigated for potential medical use in the treatment of epilepsy. It is well tolerated in human trials, with the main side effects being sedation, dizziness and headache.[6] Further trials are underway and clinical development looks likely based on the results to date.[7][8]

See also


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  1. Carter RB, Wood PL, Wieland S, Hawkinson JE, Belelli D, Lambert JJ, White HS, Wolf HH, Mirsadeghi S, Tahir SH, Bolger MB, Lan NC, Gee KW. Characterization of the anticonvulsant properties of ganaxolone (CCD 1042; 3alpha-hydroxy-3beta-methyl-5alpha-pregnan-20-one), a selective, high-affinity, steroid modulator of the gamma-aminobutyric acid(A) receptor. Journal of Pharmacology and Experimental Therapeutics. 1997 Mar;280(3):1284-95. PMID 9067315
  2. Kaminski RM, Livingood MR, Rogawski MA. Allopregnanolone analogs that positively modulate GABA receptors protect against partial seizures induced by 6-Hz electrical stimulation in mice. Epilepsia. 2004 Jul;45(7):864-7. PMID 15230714.
  3. Reddy DS, Rogawski MA. Ganaxolone suppression of behavioral and electrographic seizures in the mouse amygdala kindling model. Epilepsy Res. 2010 Feb 19. [Epub ahead of print] PMID: 20172694.
  4. Reddy DS, Rogawski MA. Neurosteroid replacement therapy for catamenial epilepsy. Neurotherapeutics. 2009 Apr;6(2):392-401. PMID 20172694
  5. Reddy DS, Rogawski MA. Chronic treatment with the neuroactive steroid ganaxolone in the rat induces anticonvulsant tolerance to diazepam but not to itself. J Pharmacol Exp Ther. 2000 Dec;295(3):1241-8. PMID 11082461
  6. Monaghan EP, Navalta LA, Shum L, Ashbrook DW, Lee DA. Initial human experience with ganaxolone, a neuroactive steroid with antiepileptic activity. Epilepsia. 1997 Sep;38(9):1026-31. PMID 9579942
  7. Nohria V, Giller E. Ganaxolone. Neurotherapeutics. 2007 Jan;4(1):102-5. PMID 17199022
  8. Pieribone VA, Tsai J, Soufflet C, Rey E, Shaw K, Giller E, Dulac O. Clinical evaluation of ganaxolone in pediatric and adolescent patients with refractory epilepsy. Epilepsia. 2007 Oct;48(10):1870-4. PMID 17634060