Systematic (IUPAC) name
Clinical data
Legal status
  • investigational
CAS number  YesY
ATC code None
ChemSpider  N
Chemical data
Formula C22H36O2 
Mol. mass 332.520 g/mol

Ganaxolone (INN, also known as 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 the GABAA receptor.[1] Ganaxolone protects against seizures in diverse animal models, including the pentylenetetrazol, 6 Hz and amygdala kindling models.[2][3][4] Unlike for benzodiazepines, there does not appear to be 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 exposure of >900 patients), with the main side-effects being sedation, dizziness, fatigue, and headache.[6] Trials in adults with partial seizures and in infantile spasms have recently been completed.[7][8][9]

See also


  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; 3α-hydroxy-3β-methyl-5α-pregnan-20-one), a selective, high-affinity, steroid modulator of the γ-aminobutyric acidA 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 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 May;89(2-3):254-60. 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
  9. ^ Farfel G, Tsai J, Shaw K, Nohria V, Rogawski MA (2013). ""Ganaxolone" in Bialer M, Johannessen SI, Levy RH, Perucca E, Tomson T, White HS: Progress report on new antiepileptic drugs: a summary of the Eleventh Eilat Conference (EILAT XI)". Epilepsy Res. 103: 2–30.