ganaxolone   Click here for help

GtoPdb Ligand ID: 11942

Synonyms: CCD-1042 | CCD1042 | Ztalmy®
Approved drug
ganaxolone is an approved drug (FDA (2022))
Compound class: Synthetic organic
Comment: Ganaxolone is a synthetic analogue of the neuroactive steroid allopregnanolone (5α-pregnan-3α-ol-20-one). Its anti-seizure effect is likely associated with its activity as a positive allosteric modulator of the GABAA receptor [1,3-4].
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Ligand Activity Visualisation Charts

These are box plot that provide a unique visualisation, summarising all the activity data for a ligand taken from ChEMBL and GtoPdb across multiple targets and species. Click on a plot to see the median, interquartile range, low and high data points. A value of zero indicates that no data are available. A separate chart is created for each target, and where possible the algorithm tries to merge ChEMBL and GtoPdb targets by matching them on name and UniProt accession, for each available species. However, please note that inconsistency in naming of targets may lead to data for the same target being reported across multiple charts.

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View interactive charts of activity data across species
2D Structure
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2D Structure

An image of the ligand's 2D structure. For small molecules with SMILES these are drawn using the NCI/CADD Chemical Identifier Resolver. Click on the image to access the chemical structure search tool with the ligand pre-loaded in the structure editor. For other types of ligands, e.g. longer nucleotides and peptides, a manually drawn representation of the molecule may be provided.

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Physico-chemical Properties
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Physico-chemical Properties

Calculated molecular properties are available for small molecules and natural products (not peptides). Properties were generated using the CDK toolkit. All properties were selected to enable the prediction of the Lipinski Rule-of-Five profile or ‘druglikeness’ for each ligand. For more info on each category see the help pages.

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Hydrogen bond acceptors 2
Hydrogen bond donors 1
Rotatable bonds 1
Topological polar surface area 37.3
Molecular weight 332.27
XLogP 6.51
No. Lipinski's rules broken 1
SMILES / InChI / InChIKey
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SMILES / InChI / InChIKey

SMILES (Simplified Molecular Input Line Entry Specification) A specification for unambiguously describing the structure of chemical molecules using short ASCII strings. Canonical SMILES specify a unique representation of the 2D structure without chiral or isotopic specifications. Isomeric SMILES include chiral specification and isotopes.

Standard InChI (IUPAC International Chemical Identifier) and InChIKey InChI is a non-proprietary, standard, textual identifier for chemical substances designed to facilitate linking of information and database searching. An InChIKey is a simplified version of a full InChI, designed for easier web searching.

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Canonical SMILES CC(=O)[C@H]1CC[C@@H]2[C@]1(C)CC[C@H]1[C@H]2CC[C@@H]2[C@]1(C)CC[C@@](C2)(C)O
Isomeric SMILES CC(=O)[C@H]1CC[C@@H]2[C@@]1(CC[C@H]1[C@H]2CC[C@@H]2[C@@]1(CC[C@@](C2)(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
InChI Key PGTVWKLGGCQMBR-FLBATMFCSA-N
No information available.
Summary of Clinical Use Click here for help
Ganaxolone (Ztalmy®) was authorised by the FDA in March 2022. It is indicated as a an oral medication for treating pediatric patients (≥ 2 yrs old) who suffer seizures due to the rare genetic form of epilepsy, cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD). In the EU, ganaxolone has been granted orphan designation for the treament of seizures caused by CDD and tuberous sclerosis.
Ganaxolone continues to be investigated in other conditions that are associated with difficult-to-treat and/or drug-resistant seizures and some depressive disorders.
Clinical Trials
Clinical Trial ID Title Type Source Comment References
NCT03572933 Study of Adjunctive Ganaxolone Treatment in Children and Young Adults With CDKL5 Deficiency Disorder Phase 3 Interventional Marinus Pharmaceuticals
NCT04391569 RAndomized Therapy In Status Epilepticus Phase 3 Interventional Marinus Pharmaceuticals
NCT01963208 Phase 3 Study of Adjunctive Ganaxolone in Adults With Drug-resistant Partial Onset Seizures and Open-label Extension Phase 3 Interventional Marinus Pharmaceuticals