Topotecan

Topoisomerase Inhibitor — ANTINEOPLASTIC AND IMMUNOMODULATING AGENTS

Key facts

Topotecan is an ofp drug in the Topoisomerase Inhibitor class catalogued in DrugCentral. It was first approved by the FDA in 1996. Its mechanism of action is classified as Topoisomerase Inhibitors. DrugCentral lists 15 molecular targets, including TOP1, HIF1A and SLC47A1. Recorded indications include Malignant tumor of cervix, Malignant tumor of ovary and Small cell carcinoma of lung.

• Approval status: ofp (first approved 1996 by the FDA).
• Drug class: Topoisomerase Inhibitor.
• Mechanism of action: Topoisomerase Inhibitors.
• Molecular targets: TOP1, HIF1A, SLC47A1 and CYP3A4 (15 listed in DrugCentral).
• Indications: Malignant tumor of cervix, Malignant tumor of ovary and Small cell carcinoma of lung.
• ATC code: L01CE01 (Topoisomerase 1 (TOP1) inhibitors).
• Molecular formula: C23H23N3O5; molecular weight 421.45 g/mol.

Indications & contraindications

Drug–disease relationships curated by DrugCentral (OMOP / SNOMED concepts).

Indications (3)

• Malignant tumor of cervix
• Malignant tumor of ovary
• Small cell carcinoma of lung

Off-label uses (2)

• Ewing's sarcoma
• Refractory Osteosarcoma

Contraindications (15)

• Acute infectious disease
• Anemia
• Bone marrow depression
• Breastfeeding (mother)
• Diarrhea
• Fibrosis of lung
• Impaired renal function disorder
• Interstitial lung disease
• Interstitial pneumonia
• Neutropenic colitis
• Neutropenic disorder
• Pregnancy, function
• Radiation Therapy Involving the Lungs
• Severe Bone Marrow Depression
• Thrombocytopenic disorder

Mechanism & targets

• Mechanism of action (MoA): Topoisomerase Inhibitors
• Established pharmacologic class: Topoisomerase Inhibitor

Molecular targets (15)

Target	Gene	Action	Activity
DNA topoisomerase 1	TOP1	unknown	Kd 7.58
Hypoxia-inducible factor 1-alpha	HIF1A	unknown	IC50 7.22
Multidrug and toxin extrusion protein 1	SLC47A1	unknown	IC50 5.89
Cytochrome P450 3A4	CYP3A4	unknown	IC50 5.82
ORF 73	—	unknown	AC50 5.67
UDP-galactopyranose mutase	glf	unknown	AC50 5.16
Multidrug and toxin extrusion protein 2	SLC47A2	unknown	IC50 5.07
DNA topoisomerase I	Top1	unknown	IC50 4.70
Solute carrier family 22 member 2	SLC22A2	unknown	IC50 4.21
Nuclear receptor subfamily 1 group I member 2	NR1I2	unknown	—
Canalicular multispecific organic anion transporter 1	ABCC2	unknown	—
DNA topoisomerase I, mitochondrial	TOP1MT	inhibitor	—
Multidrug resistance protein 1	ABCB1	unknown	—
Multidrug resistance-associated protein 4	ABCC4	unknown	—
ATP-binding cassette sub-family G member 2	ABCG2	unknown	—

Regulatory approvals (2)

Date	Agency	Marketed as	Applicant	Orphan
1996-11-12	EMA	—	SANDOZ PHARMACEUTICALS D.D.
1996-05-28	FDA	—	NOVARTIS PHARMS CORP

ATC classification

ATC code	Therapeutic hierarchy
L01CE01	ANTINEOPLASTIC AND IMMUNOMODULATING AGENTS › ANTINEOPLASTIC AGENTS › PLANT ALKALOIDS AND OTHER NATURAL PRODUCTS › Topoisomerase 1 (TOP1) inhibitors

Reported adverse-event signals

Strongest FDA FAERS disproportionality signals (log-likelihood ratio). A signal is a statistical association in spontaneous reports, not proof that the drug causes the event.

MedDRA term	LLR
Febrile neutropenia	729.5
Anaemia	457.7
Neutropenia	410.5
Thrombocytopenia	406.6
Malignant neoplasm progression	347.1
Disease progression	305.0
Product use in unapproved indication	293.8
Neoplasm progression	293.0
Pancytopenia	289.2
White blood cell count decreased	281.4
Neutrophil count decreased	234.3
Platelet count decreased	216.3

Chemical structure

• Molecular formula: C₂₃H₂₃N₃O₅
• Molecular weight: 421.45 g/mol
• CAS number: 123948-87-8
• InChIKey: UCFGDBYHRUNTLO-QHCPKHFHSA-N
• PubChem CID: 60700

CC[C@@]1(O)C(=O)OCC2=C1C=C1N(CC3=CC4=C(CN(C)C)C(O)=CC=C4N=C13)C2=O

Frequently asked questions

Answers are generated from the structured data on this page, not written by hand.

What is Topotecan used for?

DrugCentral lists the following indications for Topotecan: Malignant tumor of cervix, Malignant tumor of ovary and Small cell carcinoma of lung.

How does Topotecan work?

Topotecan is classified as Topoisomerase Inhibitors; its molecular targets in DrugCentral include TOP1, HIF1A, SLC47A1 and CYP3A4.

When was Topotecan first approved?

Topotecan was first approved by the FDA in 1996.

What drug class does Topotecan belong to?

Topotecan is in the Topoisomerase Inhibitor class.

What is the chemical formula of Topotecan?

Topotecan has the molecular formula C23H23N3O5 and a molecular weight of 421.45 g/mol.

Sources

• DrugCentral drug card 2707 — structure, indications, targets, approvals, pharmacologic classes (CC BY-SA 4.0)
• ChEMBL CHEMBL1607 — compound record and bioactivity
• PubChem CID 60700 — chemical identifiers
• DrugBank DB01030 — drug record
• FDA UNII 7M7YKX2N15 — substance identity
• DailyMed: FDA labels for Topotecan — current prescribing information (label search)
• UniProt P11387 (TOP1) — target protein
• UniProt Q16665 (HIF1A) — target protein
• UniProt Q96FL8 (SLC47A1) — target protein
• UniProt P08684 (CYP3A4) — target protein
• UniProt Q86VL8 (SLC47A2) — target protein
• UniProt O15244 (SLC22A2) — target protein
• FDA Adverse Event Reporting System (openFDA FAERS) — adverse-event signals, as aggregated by DrugCentral

How this page is built: methodology · editorial policy & corrections.