Title: Ganaxolone
CAS Registry Number: 38398-32-2
CAS Name: (3a,5a)-3-Hydroxy-3-methylpregnan-20-one
Manufacturers' Codes: CCD-1042
Molecular Formula: C22H36O2
Molecular Weight: 332.52
Percent Composition: C 79.46%, H 10.91%, O 9.62%
Literature References: Synthetic analog of the neuroactive steroids known as epalons that allosterically modulate the g-aminobutyric acid type A (GABAA) receptor. Prepn: M. C. Cook et al., DE 2162555; eidem, US 3953429 (1972, 1976 both to Glaxo); D. J. Hogenkamp et al., J. Med. Chem. 40, 61 (1997). Binding study and anticonvulsant activity: R. B. Carter et al., J. Pharmacol. Exp. Ther. 280, 1284 (1997). Clinical pharmacology: E. P. Monaghan et al., Epilepsia 38, 1026 (1997). Determn in serum by HPLC-MS-MS: K. Ramu et al., J. Chromatogr. B 751, 49 (2001).
Properties: White crystals from methanol, mp 190-192°. [a]D +103°. Insol in aqueous media.
Melting point: mp 190-192°
Optical Rotation: [a]D +103°
Therap-Cat: Anticonvulsant; antimigraine.
Keywords: Anticonvulsant; Antimigraine.
Ganglefene Ganirelix Gardenins Gardinol Type Detergents Gardol?

Systematic (IUPAC) name
Clinical data
Legal status investigational
CAS number 38398-32-2 YesY
ATC code None
PubChem CID 6918305
ChemSpider 5293511 N
KEGG D04300 YesY
Chemical data
Formula C22H36O2 
Mol. mass 332.520 g/mol
 N (what is this?)  (verify)

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 GABAA receptors.[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 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 exposure of >900 patients), with the main side-effects being sedation, dizziness, and headache.[6] Trials in adults with partial seizures and in infantile spasms have recently been completed.[7][8][9]