Details of the Drug

General Information of Drug (ID: DMXJMKF)

Drug Name

Ganaxolone

Synonyms

Ganaxolone [USAN]; CCD 1042; CCD-1042; Ganaxolone (USAN/INN); (3alpha,5alpha)-3-Hydroxy-3-methylpregnan-20-one; 1-[(3R,5S,10S,13S,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; 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; 3alpha-Hydroxy-3-methyl-5alpha-pregnan-20-one; 3alpha-Hydroxy-3beta-methyl-5alpha-pregnan-20-one

Indication

Disease Entry	ICD 11	Status	REF
Complex partial seizure	8A68.0	Phase 3	[1]
Postpartum depression	6E20.0	Phase 2	[2]

Drug Type

Small molecular drug

Structure

3D MOL

2D MOL

#Ro5 Violations (Lipinski): 0

Molecular Weight (mw)

332.5

Topological Polar Surface Area (xlogp)

Rotatable Bond Count (rotbonds)

Hydrogen Bond Donor Count (hbonddonor)

Hydrogen Bond Acceptor Count (hbondacc)

ADMET Property

Half-life: The concentration or amount of drug in body reduced by one-half in 1.3 - 1.9 hours [3]

Chemical Identifiers

Formula: C22H36O2
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
Canonical 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
InChIKey: PGTVWKLGGCQMBR-FLBATMFCSA-N

Cross-matching ID

PubChem CID: 6918305
CAS Number: 38398-32-2
DrugBank ID: DB05087
TTD ID: D0X0CH

Molecular Interaction Atlas of This Drug

Drug Therapeutic Target (DTT)

DTT Name	DTT ID	UniProt ID	MOA	REF
GABA(A) receptor delta (GABRD)	TTGXH6N	GBRD_HUMAN	Modulator	[4]
GABA(A) receptor gamma-3 (GABRG3)	TTEX6LM	GBRG3_HUMAN	Agonist	[2], [5], [6]

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Molecular Interaction Atlas (MIA)

MIA Detail

Jump to Detail Molecular Interaction Atlas of This Drug

References

1	ClinicalTrials.gov (NCT01963208) Phase 3 Study of Adjunctive Ganaxolone in Adults With Drug-resistant Partial Onset Seizures and Open-label Extension. U.S. National Institutes of Health.
2	Clinical pipeline report, company report or official report of the Pharmaceutical Research and Manufacturers of America (PhRMA)
3	Trend Analysis of a Database of Intravenous Pharmacokinetic Parameters in Humans for 1352 Drug Compounds
4	Ganaxolone, a selective, high-affinity steroid modulator of the gamma-aminobutyric acid-A receptor, exacerbates seizures in animal models of absence. Ann Neurol. 1998 Oct;44(4):688-91.
5	Clinical pipeline report, company report or official report of the Pharmaceutical Research and Manufacturers of America (PhRMA)
6	Clinical pipeline report, company report or official report of the Pharmaceutical Research and Manufacturers of America (PhRMA)
7	Antibodies and venom peptides: new modalities for ion channels. Nat Rev Drug Discov. 2019 May;18(5):339-357.
8	Gaboxadol--a new awakening in sleep.Curr Opin Pharmacol.2006 Feb;6(1):30-6.
9	WO patent application no. 2014,1515,17, Methods of improving microvascular integrity.
10	URL: http://www.guidetopharmacology.org Nucleic Acids Res. 2015 Oct 12. pii: gkv1037. The IUPHAR/BPS Guide to PHARMACOLOGY in 2016: towards curated quantitative interactions between 1300 protein targets and 6000 ligands. (Target id: 416).
11	Trusted, scientifically sound profiles of drug programs, clinical trials, safety reports, and company deals, written by scientists. Springer. 2015. Adis Insight (drug id 800007012)
12	The anaesthetic action and modulation of GABAA receptor activity by the novel water-soluble aminosteroid Org 20599. Neuropharmacology. 1996;35(9-10):1209-22.
13	Complex interactions between the steroid derivative RU 5135 and the GABAA-receptor complex. Eur J Pharmacol. 1992 Oct 1;227(2):147-51.
14	URL: http://www.guidetopharmacology.org Nucleic Acids Res. 2015 Oct 12. pii: gkv1037. The IUPHAR/BPS Guide to PHARMACOLOGY in 2016: towards curated quantitative interactions between 1300 protein targets and 6000 ligands. (Target id: 415).
15	Drugs@FDA. U.S. Food and Drug Administration. U.S. Department of Health & Human Services.
16	Stiripentol, a putative antiepileptic drug, enhances the duration of opening of GABA-A receptor channels. Epilepsia. 2006 Apr;47(4):704-16.
17	Drugs@FDA. U.S. Food and Drug Administration. U.S. Department of Health Human Services. 2020