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Atlas / Drug / Topotecan

Topotecan

drug
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Also known as Ff-10850 (liposomal topotecan)HycamptamineHycamptinNogitecanNSC-609699NSC-641007SK&F-104864SKF-104864TopotecaneSID137275799SID174007309SID164339428NATopotecan (Hydrochloride)

Summary

Topotecan (CHEMBL84) is an approved small-molecule EC 5.99.1.2 (DNA topoisomerase) inhibitor (ATC L01CE01); indicated across 62 conditions including neoplasm and small cell lung carcinoma; with CIViC clinical evidence for 1 variant-indication association (e.g. TOP1 EXPRESSION in ovarian cancer).

At a glance

  • Status: Approved (max clinical phase 4)
  • Modality: Small molecule
  • ATC class: L01CE01
  • Indications: 62 conditions
  • Clinical trials: 249
  • Precision-oncology evidence (CIViC): 1 variant–indication association
  • Chemistry: 421.4 Da · C23H23N3O5

Identifiers

Drug identity and classification

FieldValue
ChEMBL IDCHEMBL84
NameTopotecan
TypeSmall molecule
Max phase4
FDA approvedyes
PubChem CID60700
ChEBICHEBI:63632
ATCL01CE01
Molecular formulaC23H23N3O5
Molecular weight421.4
InChIKeyUCFGDBYHRUNTLO-QHCPKHFHSA-N

SMILES: CC[C@@]1(C2=C(COC1=O)C(=O)N3CC4=CC5=C(C=CC(=C5CN(C)C)O)N=C4C3=C2)O

IUPAC name: (19S)-8-[(dimethylamino)methyl]-19-ethyl-7,19-dihydroxy-17-oxa-3,13-diazapentacyclo[11.8.0.02,11.04,9.015,20]henicosa-1(21),2,4(9),5,7,10,15(20)-heptaene-14,18-dione

ChEBI definition: A pyranoindolizinoquinoline used as an antineoplastic agent. It is a derivative of camptothecin and works by binding to the topoisomerase I-DNA complex and preventing religation of these 328 single strand breaks.

Pharmacological roles (ChEBI): EC 5.99.1.2 (DNA topoisomerase) inhibitor, antineoplastic agent.

Also known as: Ff-10850 (liposomal topotecan), Hycamptamine, Hycamptin, Nogitecan, NSC-609699, NSC-641007, SK&F-104864, SKF-104864, Topotecan, Topotecane, topotecan, SID137275799

Parent form; salt/anhydrous children: CHEMBL1607

Patent coverage: 34,554 distinct patent families (141,586 SureChEMBL compound mentions), from 3 matched compound structure(s). One matched structure accounts for 141,150 (100%) of the total. Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

Broader ChEMBL bioactivity targets: 9 (assay-derived). Sample: Solute carrier family 22 member 2, Multidrug and toxin extrusion protein 1, Multidrug and toxin extrusion protein 2, DNA topoisomerase 1, DNA topoisomerase II, Hypoxia inducible factors; HIF-1-alpha, HIF-2-alpha, DNA topoisomerase 1, Cytochrome P450 3A4, Hypoxia-inducible factor 1-alpha.

Bioactivity

ChEMBL activities: 23 potent at pChembl ≥ 5 of 26 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

TargetpChemblTypeValueUnitActivity ID
HIF1A7.92EC5012nMCHEMBL_ACT_15624813
TOP17.68IC5021nMCHEMBL_ACT_18991721
TOP17.68IC5021nMCHEMBL_ACT_22978569
TOP17.58Kd26.5nMCHEMBL_ACT_6318866
TOP17.3IC5050nMCHEMBL_ACT_1499313
TOP17.27EC5054nMCHEMBL_ACT_12177606
HIF1A7.22IC5060nMCHEMBL_ACT_3527980
TOP17.15EC5071.3nMCHEMBL_ACT_12177607
HIF1A7.15EC5071.3nMCHEMBL_ACT_2433568
TOP2A6.32IC50480nMCHEMBL_ACT_19240603
TOP2A6.14IC50730nMCHEMBL_ACT_19240593
TOP16IC501000nMCHEMBL_ACT_945893
TOP15.99IC501028nMCHEMBL_ACT_302645
TOP15.96IC501100nMCHEMBL_ACT_781582
SLC47A15.89IC501300nMCHEMBL_ACT_12637851
CYP3A45.82IC501521nMCHEMBL_ACT_7825751
TOP15.75IC501800nMCHEMBL_ACT_18991791
TOP15.75IC501800nMCHEMBL_ACT_22978580
TOP15.5IC503200nMCHEMBL_ACT_25031392
TOP15.28IC505250nMCHEMBL_ACT_12457531
TOP15.26IC505550nMCHEMBL_ACT_12457533
SLC47A25.07IC508600nMCHEMBL_ACT_12636221
TOP15.01IC509650nMCHEMBL_ACT_12457529

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

62 indications (0 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

IndicationTrial phaseMONDOEFO
neoplasm3MONDO:0005070EFO:0000616
small cell lung carcinoma3MONDO:0008433EFO:0000702
non-small cell lung carcinoma3MONDO:0005233EFO:0003060
ovarian neoplasm3MONDO:0021068EFO:0003893
neuroblastoma3MONDO:0005072EFO:0000621
ovarian cancer3MONDO:0008170MONDO:0008170
acute lymphoblastic leukemia3MONDO:0004967EFO:0000220
ganglioneuroblastoma3MONDO:0005035EFO:0000502
cervical carcinoma3MONDO:0005131EFO:0001061
ovarian carcinoma3MONDO:0005140EFO:0001075
cervical adenocarcinoma3MONDO:0005153EFO:0001416
retinoblastoma3MONDO:0008380MONDO:0008380
lung neoplasm3MONDO:0021117MONDO:0008903
tumor of uterus2MONDO:0021353EFO:0003859
brain neoplasm2MONDO:0021211EFO:0003833
Ewing sarcoma2MONDO:0012817EFO:0000174
acute myeloid leukemia2MONDO:0018874EFO:0000222
glioblastoma2MONDO:0018177EFO:0000519
leukemia2MONDO:0005059EFO:0000565
sarcoma2MONDO:0005089EFO:0000691
plasma cell myeloma2MONDO:0009693EFO:0001378
rhabdomyosarcoma2MONDO:0005212EFO:0002918
medulloblastoma2MONDO:0007959EFO:0002939
breast neoplasm2MONDO:0021100EFO:0003869
non-Hodgkin lymphoma2MONDO:0018908EFO:0005952
mediastinal cancer2MONDO:0005843EFO:0007362
central nervous system neoplasm2MONDO:0006130EFO:1000158
fallopian tube carcinoma2MONDO:0006206EFO:1000251
peritoneal neoplasm2MONDO:0006901EFO:1001100
gliosarcoma2MONDO:0016681EFO:1001465
fallopian tube neoplasm2MONDO:0021092MONDO:0002158
Wilms tumor2MONDO:0006058MONDO:0006058
endometrium neoplasm2MONDO:0021251MONDO:0011962
colonic neoplasm2MONDO:0005401MONDO:0021063
plasma cell neoplasm1MONDO:0004959EFO:0000200
lymphoma1MONDO:0005062EFO:0000574
myxoid liposarcoma1MONDO:0013280EFO:0000613
oligoastrocytoma1MONDO:0016702EFO:0000630
renal cell carcinoma1MONDO:0005086EFO:0000681
fibrosarcoma1MONDO:0005164EFO:0002087
anaplastic astrocytoma1MONDO:0016684EFO:0002499
anaplastic oligodendroglioma1MONDO:0016696EFO:0002501
metastatic melanoma1MONDO:0005191EFO:0002617
tumor of meninges1MONDO:0016743EFO:0003851
triple-negative breast carcinoma1MONDO:0005494EFO:0005537
histiocytoma1MONDO:0005509EFO:0005561
clear cell sarcoma1MONDO:0002926EFO:0008498
small cell carcinoma1MONDO:0000402EFO:0008524
paraganglioma1MONDO:0000448EFO:1000453
rectal cancer1MONDO:0006519EFO:1000657
aseptic meningitis1MONDO:0006662EFO:1000823
desmoplastic small round cell tumor1MONDO:0019373EFO:1000895
female reproductive organ cancer1MONDO:0001416EFO:1001331
myoepithelial tumor1MONDO:0002380MONDO:0002380
fibroma1MONDO:0005167MONDO:0037745
severe acute respiratory syndrome1MONDO:0005091MONDO:0100096
glioma0MONDO:0021042MONDO:0100342

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

Clinical trials

Total trials: 249.

Phase distribution

PhaseTrials
PHASE280
PHASE168
PHASE358
PHASE1/PHASE222
PHASE2/PHASE37
EARLY_PHASE16
Not specified6
PHASE42

Top trials by phase / activity

NCTPhaseStatusTitle
NCT05874401PHASE4RECRUITINGTrilaciclib vs Placebo in Patients With Extensive Stage Small Cell Lung Cancer (ES-SCLC) Receiving Topotecan
NCT00488709PHASE4COMPLETEDFludarabine, Cytarabine, Topotecan in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia
NCT02502266PHASE2/PHASE3ACTIVE_NOT_RECRUITINGTesting the Combination of Cediranib and Olaparib in Comparison to Each Drug Alone or Other Chemotherapy in Recurrent Platinum-Resistant Ovarian Cancer
NCT04681417PHASE2/PHASE3RECRUITINGOcular Conservative Treatment for Retinoblastoma : Efficacy of the New Management Strategies and Visual Outcome
NCT04799002PHASE3RECRUITINGTopotecan and Melphalan for Retinoblastoma
NCT05740566PHASE3ACTIVE_NOT_RECRUITINGStudy Comparing Tarlatamab With Standard of Care Chemotherapy in Relapsed Small Cell Lung Cancer
NCT06128837PHASE3RECRUITINGStudy of LY01610 in Patients With Recurrent Small Cell Lung Cancer
NCT06161025PHASE2/PHASE3RECRUITINGA Study of Raludotatug Deruxtecan (R-DXd) in Subjects With Platinum-resistant, High-grade Ovarian, Primary Peritoneal, or Fallopian Tube Cancer
NCT06172296PHASE3RECRUITINGDinutuximab With Chemotherapy, Surgery and Stem Cell Transplantation for the Treatment of Children With Newly Diagnosed High Risk Neuroblastoma
NCT06203210PHASE3RECRUITINGA Study of Ifinatamab Deruxtecan Versus Treatment of Physician’s Choice in Subjects With Relapsed Small Cell Lung Cancer
NCT06394492PHASE3RECRUITINGSHR-A1921 for Injection in Patients With Platinum-Resistant Recurrent Epithelial Ovarian Cancer
NCT06459180PHASE3ACTIVE_NOT_RECRUITINGA Study to Compare Sacituzumab Tirumotecan (MK-2870) Monotherapy Versus Treatment of Physician’s Choice as Second-line Treatment for Participants With Recurrent or Metastatic Cervical Cancer (MK-2870-020/TroFuse-020/Gog-3101/ENGOT-cx20)
NCT06496048PHASE3RECRUITINGLurbinectedin or in Combination with Irinotecan Versus Topotecan in Patients with Relapsed SCLC
NCT06498479PHASE3RECRUITINGARTEMIS-008：HS-20093 Compared With Topotecan in Subjects With Relapsed Small Cell Lung Cancer
NCT06500026PHASE3ACTIVE_NOT_RECRUITINGA Study Comparing BL-B01D1 With Topotecan in Patients With Recurrent Small Cell Lung Cancer(PANKU-Lung03)
NCT06581380PHASE3NOT_YET_RECRUITINGJK-1201I Compared with Topotecan in Patients with Relapsed Extensive Stage Small Cell Lung Cancer
NCT06616532PHASE3ACTIVE_NOT_RECRUITINGPM8002 in Combination With Paclitaxel Compared With Chemotherapy as Second-line Treatment in Small Cell Lung Cancer
NCT06619236PHASE3ACTIVE_NOT_RECRUITINGStudy to Assess the Efficacy of Rina-S Compared to Treatment of Investigator’s Choice in Participants With Platinum Resistant Ovarian Cancer
NCT06647953PHASE3RECRUITINGTesting a Standardized Approach to Surgery and Chemotherapy for Type I Pleuropulmonary Blastoma or the Addition of an Anti-cancer Drug, Topotecan, to the Usual Treatment for Types II and III Pleuropulmonary Blastoma
NCT06751485PHASE3NOT_YET_RECRUITINGJSKN003 in Platinum-Resistant, Relapsed Epithelial Ovarian Cancer
NCT06801834PHASE3RECRUITINGStudy of Sacituzumab Govitecan Versus Standard of Care in Participants With Previously Treated Extensive Stage Small Cell Lung Cancer
NCT06855069PHASE3RECRUITINGHS-20089 for Injection in Patients With Platinum-Resistant Recurrent Epithelial Ovarian Cancer
NCT06954246PHASE3RECRUITINGA Study of MHB088C Injection Versus Treatment of Physician’s Choice in Subjects With Relapsed Small Cell Lung Cancer
NCT07099898PHASE3RECRUITINGA Study of GSK5764227 in Participants With Relapsed Small Cell Lung Cancer (SCLC)
NCT07106528PHASE2/PHASE3NOT_YET_RECRUITINGOrganoid-guided vs Topotecan Therapy in Relapsed Extensive-Stage Small Cell Lung Cancer
NCT07189455PHASE3RECRUITINGStudy Comparing ZG006 With Investigator-Selected Chemotherapy in Participants With Relapsed Small Cell Lung Cancer
NCT07213804PHASE3RECRUITINGA Two-Part Phase 3 Study of Sofetabart Mipitecan (LY4170156) in Participants With Platinum-Resistant (Part A) and Platinum-Sensitive (Part B) Ovarian Cancer
NCT07218809PHASE3RECRUITINGAZD5335 vs. Mirvetuximab Soravtansine in FRα-high and AZD5335 vs. Chemotherapy in FRα-low Platinum-resistant Ovarian Cancer
NCT07286266PHASE3NOT_YET_RECRUITINGA Study to Investigate Mocertatug Rezetecan Compared With Standard of Care in Participants With Platinum-resistant Ovarian Cancer (BEHOLD-Ovarian01)
NCT07365241PHASE3NOT_YET_RECRUITINGA Study to Evaluate Adverse Events and Change in Disease Activity of Intravenous ABBV-706 Versus Standard of Care in Adult Participants With Relapsed/Refractory Small Cell Lung Cancer
NCT07545460PHASE3NOT_YET_RECRUITINGA Study Comparing BL-M07D1 With Physician’s Choice of Chemotherapy in Patients With HER2-Expressing Platinum-Resistant Recurrent Epithelial Ovarian Cancer, Fallopian Tube Cancer, and Primary Peritoneal Cancer
NCT07604766PHASE3NOT_YET_RECRUITINGAn Extension Study to Assess the Efficacy of Rina-S Compared to Treatment of Investigator’s Choice in Participants With Platinum Resistant Ovarian Cancer in China
NCT00043927PHASE3COMPLETEDExtensive Small Cell Lung Cancer Treatment Using An Investigational Drug Plus Chemotherapy In Chemotherapy-Naive Adults
NCT00049998PHASE3COMPLETEDOral Topotecan Versus Intravenous Docetaxel In Pretreated, Advanced Non-Small Cell Lung Cancer
NCT00102375PHASE3COMPLETEDPaclitaxel, Carboplatin, and Topotecan in Patients With Ovarian Cancer
NCT00186888PHASE3COMPLETEDStudy of Treatment for Patients With Cancer of the Eye -Retinoblastoma
NCT00187083PHASE3COMPLETEDA Study of Children With Refractory or Relapsed ALL
NCT00276276PHASE3COMPLETEDActive Symptom Control Alone Or In Combination With Oral Topotecan In Patients With Relapsed Resistant Small Cell Lung Cancer
NCT00320359PHASE3COMPLETEDTopotecan Plus Cisplatin Versus Etoposide Plus Cisplatin In 1st Line Extensive Disease Small Cell Lung Cancer
NCT00437307PHASE3COMPLETEDHycamtin Plus Carboplatin Versus Established Regimens for the Treatment of Ovarian Cancer Relapse

Clinical evidence (CIViC)

Variant × indication × effect (1 predictive associations from 1 curated evidence items):

VariantIndicationEffectTherapyLevelCIViC
TOP1 EXPRESSIONOvarian CancerSensitivity/ResponseCarboplatin + Cyclophosphamide + TopotecanCIViC BEID910

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline, but PharmGKB curates 1 clinical and 1 variant annotation(s) for this drug (gene-keyed; see PharmGKB).

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).

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
v1.1.2
BioBTree
v2.0.2

Sources 17

This page pulls from 17 datasets indexed by BioBTree:

chebichembl_activitychembl_moleculechembl_targetcivic_evidenceclinical_trialsefogtopdb_interactiongtopdb_ligandhgncmeshmondopatent_compoundpharmgkbpharmgkb_guidelinepubchemuniprot