Ganaxolone

Drugbox
IUPAC_name = 1-​ [(3R,5S,8R,9S,10S,13S,14S,17S)-​3-​hydroxy-​3,10,13-​trimethyl-​1,2,4,5,6,7,8,9,11,12,14,15,16,17-​tetradecahydrocyclopenta [a] phenanthren-​17-​yl] ethanone


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CAS_number = 38398-32-2
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PubChem = 6918305
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chemical_formula =
C=22 | H=36 | O=2
molecular_weight = 332.520 g/mol
smiles = CC(=O) [C@H] 1CC [C@@H] 2 [C@@] 1(CC [C@H] 3 [C@H] 2CC [C@@H] 4 [C@@] 3(CC [C@@] (C4)(C)O)C)C
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legal_status = Investigational New Drug
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Ganaxolone (INN, also known as CCD-1042) is a steroid drug related to pregnanolone which has sedative, anxiolytic and anticonvulsant effects. It is a potent and selective positive allosteric modulator of GABA_A receptors. [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]

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. [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] Further trials are underway and clinical development looks likely based on the results to date. [Nohria V, Giller E. Ganaxolone. "Neurotherapeutics". 2007 Jan;4(1):102-5. PMID 17199022] [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]

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