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Ganaxolone

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Also known as CCD 1042CCD-1042CCD1042DEA NO. 2401GanaxolonaZtalmySID50112899SID90341411SID29217560SID50111707SID144205817SID170466317

Summary

Ganaxolone (CHEMBL1568698) is an approved small molecule (ATC N03AX27) targeting GABRA1; indicated across 12 conditions including visual epilepsy and epilepsy.

At a glance

  • Status: Approved (max clinical phase 4)
  • Modality: Small molecule
  • ATC class: N03AX27
  • Targets: 1 (GABRA1)
  • Indications: 12 conditions
  • Clinical trials: 23
  • Chemistry: 332.5 Da · C22H36O2

Identifiers

Drug identity and classification

FieldValue
ChEMBL IDCHEMBL1568698
NameGanaxolone
TypeSmall molecule
Max phase4
FDA approvedyes
PubChem CID6918305
ATCN03AX27
Molecular formulaC22H36O2
Molecular weight332.5
InChIKeyPGTVWKLGGCQMBR-FLBATMFCSA-N

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

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

Also known as: CCD 1042, CCD-1042, CCD1042, DEA NO. 2401, Ganaxolona, Ganaxolone, Ztalmy, SID50112899, SID90341411, SID29217560, SID50111707, GANAXOLONE

Patent coverage: 495 distinct patent families (1,657 SureChEMBL compound mentions), from 1 matched compound structure(s). Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

Primary targets (GtoPdb curated mechanism): the Cancer dependency column is the DepMap CRISPR fitness signal (% of screened cell lines dependent on the target).

GeneTargetActionpAffinityCancer dependencyUniProt
GABRA1GABAA receptor α1 subunitPotentiation6.590%P14867

Broader ChEMBL bioactivity targets: 6 (assay-derived). Sample: GABA-A receptor; anion channel, Menin/Histone-lysine N-methyltransferase MLL, GABA-A receptor; anion channel, GABA-A receptor; alpha-1/beta-2/gamma-2, GABA-A receptor; agonist GABA site, Gamma-aminobutyric acid receptor subunit alpha-2/beta-1/gamma-2.

Bioactivity

ChEMBL activities: 9 potent at pChembl ≥ 5 of 9 total. Top 100 by potency (10 = 0.1 nM, 6 = 1 µM):

TargetpChemblTypeValueUnitActivity ID
GABRP7.38IC5042nMCHEMBL_ACT_15244147
O090287.1IC5080nMCHEMBL_ACT_1183517
GABRB17EC50100nMCHEMBL_ACT_25107074
GABRA16.99EC50103nMCHEMBL_ACT_15244891
GABRA16.7EC50200nMCHEMBL_ACT_25107072
GABRA16.58EC50260nMCHEMBL_ACT_1183519
GABRA16.58EC50266nMCHEMBL_ACT_15244127
GABRP6.1Ki790nMCHEMBL_ACT_26123569
MEN15.6Potency2512nMCHEMBL_ACT_4552051

Target pathways

Aggregated over 1 target gene(s): GABRA1.

Top Reactome pathways

2 total, by targets touching each:

PathwayTargetsGenes
Signaling by ERBB41GABRA1
GABA receptor activation1GABRA1

Dominant GO biological processes

GO termTargets
gamma-aminobutyric acid signaling pathway1
synaptic transmission, GABAergic1
chloride transmembrane transport1
inhibitory synapse assembly1
monoatomic ion transport1
chloride transport1
monoatomic ion transmembrane transport1
regulation of postsynaptic membrane potential1

Indications & clinical

Indications

2 approved indications. FDA phase 4, plus an anticancer drug’s labelled cancer uses (which ChEMBL often logs at phase 3).

IndicationPhaseMONDOEFO
visual epilepsy4MONDO:0001386HP:0001250
epilepsy4MONDO:0005027EFO:0000474

5 diseases in clinical trials (phase 1–3, investigational — not approved indications). Highest ChEMBL trial phase per disease; a non-cancer approved use is occasionally logged at phase 3 here.

Disease (in trials)PhaseMONDOEFO
tuberous sclerosis3MONDO:0001734MONDO:0001734
complex partial epilepsy2MONDO:0006710EFO:1000877
post-traumatic stress disorder2MONDO:0005146EFO:0001358
focal epilepsy2MONDO:0005384EFO:0004263
fragile X syndrome2MONDO:0010383MONDO:0010383

5 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 23.

Phase distribution

PhaseTrials
PHASE213
PHASE38
Not specified2

Top trials by phase / activity

NCTPhaseStatusTitle
NCT05249556PHASE3NOT_YET_RECRUITINGDouble-blind, Randomized, Placebo-controlled Trial of Ganaxolone in CDKL5 Deficiency Patients 6 Months to Less Than 2 Years Old
NCT01963208PHASE3COMPLETEDPhase 3 Study of Adjunctive Ganaxolone in Adults With Drug-resistant Partial Onset Seizures and Open-label Extension
NCT02519439PHASE3TERMINATEDA Two-year Open-label Extension Study of Ganaxolone in Patients With Drug-resistant Partial-onset Seizures
NCT03572933PHASE3COMPLETEDStudy of Adjunctive Ganaxolone Treatment in Children and Young Adults With CDKL5 Deficiency Disorder
NCT04391569PHASE3COMPLETEDRandomized Therapy In Status Epilepticus
NCT05323734PHASE3COMPLETEDAdjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy
NCT05604170PHASE3TERMINATEDOpen-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy
NCT05814523PHASE3WITHDRAWNTo Evaluate the Efficacy, Safety, and Tolerability of Intravenous Ganaxolone Added to Standard of Care in Refractory Status Epilepticus (RSE)
NCT00441896PHASE2COMPLETEDA Randomized, Controlled Trial of Ganaxolone in Patients With Infantile Spasms
NCT00442104PHASE2TERMINATEDOpen-label Extension to Protocol 1042-0500
NCT00465517PHASE2COMPLETEDA Randomized, Controlled Trial of Ganaxolone in Adult Uncontrolled Partial-Onset Seizures
NCT00512317PHASE2COMPLETEDOpen-label Extension to Protocol 1042-0600
NCT01002820PHASE2COMPLETEDA Treatment Use Protocol for Subjects Continuing on From the Open-label Extension 0601
NCT01339689PHASE2COMPLETEDGanaxolone in Posttraumatic Stress Disorder (PTSD)
NCT01725152PHASE2COMPLETEDGanaxolone Treatment in Children With Fragile X Syndrome
NCT01857531PHASE2COMPLETEDProof-Of-Concept Investigation With a Neurosteroid Analog (Ganaxolone) as a Smoking Cessation Candidate
NCT02358538PHASE2COMPLETEDOpen-Label PoC Trial of Ganaxolone in Children With PCDH19 Female Pediatric Epilepsy and Other Rare Genetic Epilepsies
NCT03228394PHASE2COMPLETEDA Clinical Trial of Intravenous (IV) Ganaxolone in Women With Postpartum Depression
NCT03460756PHASE2COMPLETEDA Clinical Trial of Oral Ganaxolone in Women With Postpartum Depression
NCT03865732PHASE2COMPLETEDStudy of Adjunctive Ganaxolone Treatment in Female Children With Protocadherin 19 (PCDH19)-Related Epilepsy (Violet Study)
NCT04285346PHASE2COMPLETEDAdjunctive Ganaxolone Treatment (Part A) in TSC Followed by Long-term Treatment (Part B)
NCT02900092Not specifiedCOMPLETEDAugmentation of Treatment-Resistant Depression With An Analog of the Neuroactive Steroid Allopregnanolone
NCT04678479Not specifiedNO_LONGER_AVAILABLEGanaxolone Expanded Access Program Compassionate Use

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.

Molecules sharing ≥1 of this drug’s curated primary targets, merged from two biobtree sources and ranked by shared-target count, then clinical phase: ChEMBL clinical-stage candidates (development phase ≥2) and PubChem drug-class bioactivity (approved / known drugs acting on the target). Deduplicated by drug name; the drug’s own salt forms are excluded. Note: for a drug with few primary targets a shared-target match can reflect off-target / promiscuous binding rather than the same therapeutic mechanism — the phase ordering surfaces bona-fide therapeutics first.

70 molecules share ≥1 primary target. Top 70 by shared-target count:

MoleculeSourceStatusShared targets
ALPRAZOLAMChEMBLPhase 4 (approved)GABRA1
APALUTAMIDEChEMBLPhase 4 (approved)GABRA1
ASENAPINEChEMBLPhase 4 (approved)GABRA1
AZELASTINEChEMBLPhase 4 (approved)GABRA1
BENPERIDOLChEMBLPhase 4 (approved)GABRA1
BREXANOLONEChEMBLPhase 4 (approved)GABRA1
CANDESARTAN CILEXETILChEMBLPhase 4 (approved)GABRA1
CHLORAMBUCILChEMBLPhase 4 (approved)GABRA1
CHLORDIAZEPOXIDEChEMBLPhase 4 (approved)GABRA1
CLONAZEPAMChEMBLPhase 4 (approved)GABRA1
CLOZAPINEChEMBLPhase 4 (approved)GABRA1
CRIZOTINIBChEMBLPhase 4 (approved)GABRA1
DIAZEPAMChEMBLPhase 4 (approved)GABRA1
ENZALUTAMIDEChEMBLPhase 4 (approved)GABRA1
EPINASTINEChEMBLPhase 4 (approved)GABRA1
ESZOPICLONEChEMBLPhase 4 (approved)GABRA1
ETOMIDATEChEMBLPhase 4 (approved)GABRA1
FLUMAZENILChEMBLPhase 4 (approved)GABRA1
FLUNITRAZEPAMChEMBLPhase 4 (approved)GABRA1
GLAFENINEChEMBLPhase 4 (approved)GABRA1
LINDANEChEMBLPhase 4 (approved)GABRA1
LIOTHYRONINEChEMBLPhase 4 (approved)GABRA1
PROPOFOLChEMBLPhase 4 (approved)GABRA1
SIMVASTATINChEMBLPhase 4 (approved)GABRA1
STIRIPENTOLChEMBLPhase 4 (approved)GABRA1
TIPRANAVIRChEMBLPhase 4 (approved)GABRA1
TRIAZOLAMChEMBLPhase 4 (approved)GABRA1
TROGLITAZONEChEMBLPhase 4 (approved)GABRA1
ZALEPLONChEMBLPhase 4 (approved)GABRA1
ZOLPIDEMChEMBLPhase 4 (approved)GABRA1
ZURANOLONEChEMBLPhase 4 (approved)GABRA1
ALISERTIBChEMBLPhase 3GABRA1
GABOXADOLChEMBLPhase 3GABRA1
SURAMINChEMBLPhase 3GABRA1
TIRATRICOLChEMBLPhase 3GABRA1
ALFAXALONEChEMBL + PubChemPhase 2 (approved)GABRA1
ABECARNILChEMBLPhase 2GABRA1
ALOGABATChEMBLPhase 2GABRA1
AZD7325ChEMBLPhase 2GABRA1
BAICALEINChEMBLPhase 2GABRA1
BASMISANILChEMBLPhase 2GABRA1
BRETAZENILChEMBLPhase 2GABRA1
DARIGABATChEMBLPhase 2GABRA1
DELORAZEPAMChEMBLPhase 2GABRA1
FLAVONEChEMBLPhase 2GABRA1
LORECLEZOLEChEMBLPhase 2GABRA1
MK-0777ChEMBLPhase 2GABRA1
PANADIPLONChEMBLPhase 2GABRA1
PIPERINEChEMBLPhase 2GABRA1
PROGABIDEChEMBLPhase 2GABRA1
REDAFAMDASTATChEMBLPhase 2GABRA1
SARIPIDEMChEMBLPhase 2GABRA1
ZOTEPINEChEMBLPhase 2GABRA1
AfatinibPubChemApprovedGABRA1
ApixabanPubChemApprovedGABRA1
BinimetinibPubChemApprovedGABRA1
chenodiolPubChemApprovedGABRA1
FulvestrantPubChemApprovedGABRA1
gentian violetPubChemApprovedGABRA1
ImipenemPubChemApprovedGABRA1
LactulosePubChemApprovedGABRA1
levocarnitinePubChemApprovedGABRA1
podofiloxPubChemApprovedGABRA1
Propylene GlycolPubChemApprovedGABRA1
PyrazinamidePubChemApprovedGABRA1
PyridoxinePubChemApprovedGABRA1
RiociguatPubChemApprovedGABRA1
saxagliptinPubChemApprovedGABRA1
ThiaminePubChemApprovedGABRA1
VorapaxarPubChemApprovedGABRA1

About this page

Generated deterministically by Sugi Atlas from primary biomedical databases via BioBTree. Every record on this page traces to a primary source.

Generated
Atlas version
v1.1.4
BioBTree
v2.0.2

Sources 30

This page pulls from 30 datasets indexed by BioBTree:

chebichembl_activitychembl_moleculechembl_targetcivic_evidenceclingen_dosageclinical_trialsdepmapefoensemblentrezgogoparentgtopdbgtopdb_interactiongtopdb_ligandhgncmeshmondomsigdbpatent_compoundpharmgkbpharmgkb_guidelinepubchempubchem_activityreactomereactomeparentrefseqtranscriptuniprot