Ganaxolone

Ganaxolone

Clinical data
ATC code	none
Legal status
Legal status	investigational
Identifiers
IUPAC name (3α,5α)-3-hydroxy-3-methylpregnan-20-one; 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
Synonyms	CCD-1042 3β-Methyl-3α-ol-5α-pregnan-20-one; 3α-Hydroxy-3β-methyl-5α-pregnan-20-one
CAS Number	38398-32-2 Y
PubChem CID	6918305
ChemSpider	5293511 N
UNII	98WI44OHIQ
KEGG	D04300 Y
ChEMBL	CHEMBL161915 N
Chemical and physical data
Formula	C22H36O2
Molar mass	332.520 g/mol
3D model (Jmol)	Interactive image
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
InChI InChI=1S/C22H36O2/c1-14(23)17-7-8-18-16-6-5-15-13-20(2,24)11-12-21(15,3)19(16)9-10-22(17,18)4/h15-19,24H,5-13H2,1-4H3/t15-,16-,17+,18-,19-,20+,21-,22+/m0/s1 N Key:PGTVWKLGGCQMBR-FLBATMFCSA-N N
NY (what is this?)

Ganaxolone is an experimental CNS-selective GABA_A modulator that is under development by Marinus Pharmaceuticals as an anxiolytic and anticonvulsant agent. Ganaxolone has been shown to protect against seizures in animal models, and to act a positive allosteric modulator of the GABA_A receptor.

Ganaxolone is being investigated for potential medical use in the treatment of epilepsy. It is well tolerated in human trials, with the most 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.

The exact mechanism of action for ganaxolone is unknown; however, results from animal studies suggest that it acts by blocking seizure propagation and elevating seizure thresholds.

Ganaxolone is thought to modulate both synaptic and extrasynaptic GABA_A receptors to normalize over-excited neurons. Ganaxolone's activation of the extrasynaptic receptor is an additional mechanism that provides stabilizing effects that potentially differentiates it from other drugs that increase GABA signaling.

Ganaxolone binds to allosteric sites of the GABA_A receptor to modulate and open the chloride ion channel, resulting in a hyperpolarization of the neuron. This causes an inhibitory effect on neurotransmission, reducing the chance of a successful action potential (depolarization) from occurring.