Topotecan

Topoisomerase I relieves torsional strain in DNA by inducing reversible single-strand breaks. Topotecan binds to the topoisomerase I-DNA complex and prevents re-ligation of these single-strand breaks.

• Topotecan Injection is indicated for the treatment of patients with small cell lung cancer (SCLC) with platinum-sensitive disease who progressed at least 60 days after initiation of first-line chemotherapy. Topotecan Injection is a topoisomerase inhibitor indicated for treatment of small cell lung cancer (SCLC) platinum-sensitive disease in patients who progressed at least 60 days after initiation of first-line chemotherapy.ICD-10: C34.90

• Topotecan Injection is contraindicated in patients who have a history of severe hypersensitivity reactions to topotecan. Reactions have included anaphylactoid reactions [see Adverse Reactions (6.2) ] .contraindicated

Recommended dosage : 1.5 mg/m 2 by intravenous infusion over 30 minutes daily for 5 consecutive days, starting on Day 1 of a 21-day cycle. ( 2.2 ) Renal impairment : Reduce dose for creatinine clearance (CLcr) of 20 to 39 mL/min. ( 2.4 ) 2.1 Important Safety Information Verify dosage using body surface area. Do not exceed a single dose of 4 mg intravenously. 2.2 Recommended Dosage for Small Cell Lung Cancer The recommended dosage of Topotecan Injection is 1.5 mg/m 2 by intravenous infusion over 30 minutes daily for 5 consecutive days, starting on Day 1 of a 21-day cycle. 2.3 Dosage Modifications for Adverse Reactions Hematologic Do not administer subsequent cycles of Topotecan Injection until neutrophils recover to greater than 1,000/mm 3 , platelets recover to greater than 100,000/mm 3 , and hemoglobin levels recover to greater than or equal to 9 g/dL (with transfusion if necessary). Reduce the dose of Topotecan Injection to 1.25 mg/m 2 /day for: neutrophil counts of less than 500/mm 3 or administer granulocyte-colony stimulating factor (G-CSF) starting no sooner than 24 hours following the last dose platelet counts less than 25,000/mm 3 during previous cycle 2.4 Dosage Modification for Renal Impairment Reduce the dose of Topotecan Injection to 0.75 mg/m 2 /day for patients with creatinine clearance (CLcr) of 20 to 39 mL/min (calculated with the Cockcroft-Gault method using ideal body weight) [see Clinical Pharmacology (12.3) ] . 2.5 Preparation and Administration Topotecan Injection is a cytotoxic drug. Follow applicable special handling and disposable procedures.

Interstitial lung disease (ILD): Fatal cases have occurred. Permanently discontinue if ILD confirmed. ( 5.2 ) Extravasation and tissue injury : Severe cases have occurred. If extravasation occurs, immediately stop administration and institute recommended management procedures. ( 5.3 ) Embryo-fetal toxicity : Can cause fetal harm. Advise women of potential risk to the fetus. ( 5.4 , 8.1 , 8.3 ) 5.1 Myelosuppression Topotecan can cause severe myelosuppression. Grade 4 neutropenia occurred in 78% of 879 patients, with a median duration of 7 days and was most common during Cycle 1 (58% of patients). Grade 4 neutropenia associated with infection occurred in 13% and febrile neutropenia occurred in 5%. Sepsis occurred in 4% and was fatal in 1%. Grade 4 thrombocytopenia occurred in 27%, with a median duration of 5 days. Grade 3 or 4 anemia occurred in 37% of patients. Topotecan can cause fatal typhlitis (neutropenic enterocolitis). Consider the possibility of typhlitis in patients presenting with fever, neutropenia, and abdominal pain. Administer the first cycle of Topotecan Injection only to patients with a baseline neutrophil count of greater than or equal to 1,500/mm 3 and a platelet count greater than or equal to 100,000/mm 3 . Monitor blood counts frequently during treatment. Withhold and reduce dose of Topotecan Injection based on neutrophil counts, platelet counts and hemoglobin levels [see Dosage and Administration (2.3) ] . 5.2 Interstitial Lung Disease Interstitial lung disease (ILD), including fatalities, has occurred with topotecan [see Adverse Reactions (6.2) ] .

The following serious adverse reactions are described elsewhere in the labeling: Myelosuppression [see Warnings and Precautions (5.1) ] Interstitial Lung Disease [see Warnings and Precautions (5.2) ] Extravasation and Tissue Injury [see Warnings and Precautions (5.3) ] The most common Grade 3 or 4 hematologic adverse reactions (incidence >5%) were: neutropenia , anemia , thrombocytopenia, and febrile neutropenia. ( 6.1 ) The most common non-hematologic adverse reactions (incidence >5%) (all grades) were asthenia, dyspnea, nausea, pneumonia, abdominal pain, and fatigue. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Hospira, Inc. at 1-800-441-4100 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared with rates in the clinical trials of another drug and may not reflect the rates observed in practice. The data in Warnings and Precautions reflect exposure to topotecan from 8 trials in which 879 patients with small cell lung cancer (SCLC) and other solid tumors received topotecan 1.5 mg/m 2 by intravenous infusion daily for 5 consecutive days, starting on Day 1 of a 21-day cycle. Small Cell Lung Cancer (SCLC) The safety of topotecan was evaluated in randomized, comparative trial in patients with recurrent or progressive SCLC (Study 090) [see Clinical Studies (14.1) ]. Table 1 shows the Grade 3 or 4 hematologic and non-hematologic adverse reactions in patients with SCLC. Table 1.

Lactation : Advise not to breastfeed. ( 8.2 ) 8.1 Pregnancy Risk Summary Based on animal data and its mechanism of action, Topotecan Injection can cause fetal harm when administered to a pregnant woman. There are no available clinical data on the use of topotecan in pregnancy. Topotecan caused embryolethality, fetotoxicity, and teratogenicity in rats and rabbits when administered during organogenesis at doses similar to the clinical dose (see Data ). Advise pregnant women of the potential risk to a fetus. In the U.S. general population, the background risk of major birth defects is 2% to 4% and of miscarriage is 15% to 20% of clinically recognized pregnancies. Data Animal Data In rabbits, an intravenous dose of 0.10 mg/kg/day [(about equal to the 1.5 mg/m 2 clinical dose based on body surface area (BSA)] given on Days 6 through 20 of gestation caused maternal toxicity, embryolethality, and reduced fetal body weight. In the rat, an intravenous dose of 0.23 mg/kg/day (about equal to the 1.5 mg/m 2 clinical dose based on BSA) given for 14 days before mating through gestation Day 6 caused fetal resorption, microphthalmia, pre-implant loss, and mild maternal toxicity. Administration of an intravenous dose of 0.10 mg/kg/day (about half the 1.5 mg/m 2 clinical dose based on BSA) given to rats on Days 6 through 17 of gestation caused an increase in post-implantation mortality.

Metabolism

Following administration of topotecan at doses of 0.5 to 1.5 mg/m 2 (0.3 to 1 times the recommended dose) administered as a 30-minute infusion, area under the curve (AUC) increases approximately proportional with dose. Distribution Protein binding of topotecan is approximately 35%.

Overdoses (up to 10-fold of the recommended dose) have occurred in patients receiving intravenous topotecan. The primary complication of overdosage is myelosuppression. Elevated hepatic enzymes, mucositis, gastrointestinal toxicity and skin toxicity have occurred with overdosages. If an overdose is suspected, monitor the patient closely for myelosuppression and institute supportive-care measures as appropriate.

• May 28, 1996NDANDA020671Sandoz
• Oct 11, 2007NDANDA020981Sandoz
• Nov 29, 2010ANDAANDA091089Fresenius Kabi Usa
• Dec 2, 2010ANDAANDA090620Actavis Totowa
• Feb 2, 2011NDANDA200582Hospira Inc
• Jun 26, 2013ANDAANDA202351Accord Hlthcare
• Jul 6, 2017ANDAANDA204406Accord Hlthcare

1. 1Febrile Neutropenia5998.8%
2. 2Anaemia5768.5%
3. 3Death5638.3%
4. 4Off Label Use5598.2%
5. 5Neutropenia5578.2%
6. 6Disease Progression4496.6%
7. 7Nausea4496.6%
8. 8Pyrexia4456.6%
9. 9Thrombocytopenia4456.6%
10. 10Vomiting4296.3%
11. 11Product Use In Unapproved Indication3745.5%
12. 12Fatigue3294.8%
13. 13White Blood Cell Count Decreased3254.8%
14. 14Diarrhoea3204.7%
15. 15Malignant Neoplasm Progression3164.7%

Recent PubMed references pinned to Topotecan as a MeSH major topic. Citations link to pubmed.ncbi.nlm.nih.gov.

• Quercetin Enhances Topotecan Cytotoxicity in Retinoblastoma Cells Through ROS-Associated Stress and Apoptotic Signaling.
  Biomolecules · 2026 · Maçin A, Duman E, Özdemir İ, et al.
  PMID 42072718DOI 10.3390/biom16040597
• Design, Synthesis and Biological Evaluation of 6H-Benzimidazo[1',2':1,2]pyrido[3,4-b]indole Derivatives as TDP1 Inhibitors: Potent Synergistic Agents with Topotecan against Cervical Cancer.
  Journal of medicinal chemistry · 2026 · Zeng H, Qiu B, Yang J, et al.
  PMID 42012226DOI 10.1021/acs.jmedchem.6c00016
• Cyclophosphamide and Topotecan in Relapsed and Refractory Pediatric Extracranial Solid Tumors: A Retrospective Analysis.
  Indian pediatrics · 2026 · Thamaraiselvan P, Das G, Srinivasan P, et al.
  PMID 41499028DOI 10.1007/s13312-025-00248-6
• Loss of ELF2 drives topotecan resistance in retinoblastoma revealed by genome-wide CRISPR-Cas9 screening.
  Cell death & disease · 2025 · Jiang J, Jiang Z, Luo Q, et al.
  PMID 41436498DOI 10.1038/s41419-025-08335-z
• A phase II, multicenter, open-label, single-arm study of berzosertib plus topotecan in patients with relapsed platinum-resistant small-cell lung cancer (DDRiver SCLC 250).
  ESMO open · 2026 · Navarro A, Thomas A, Cheng Y, et al.
  PMID 41421295DOI 10.1016/j.esmoop.2025.105918
• [Amidothiazole Derivatives of (+)-Usnic Acid Effectively Inhibit TDP1 and Sensitize Tumor Cells to the Effects of Topotecan].
  Molekuliarnaia biologiia · 2025 · Chepanova AA, Zakharenko AL, Dyrkheeva NS, et al.
  PMID 41391025DOI 10.31857/S0026898425050069
• Results of a Pilot Trial of Infusion Rate Escalation During Convection-Enhanced Delivery of Topotecan with a Multiport Catheter.
  World neurosurgery · 2026 · Liu JKC, Tran N, Piteo C, et al.
  PMID 41344399DOI 10.1016/j.wneu.2025.124696
• Assessment of leachables in hospital pharmacy compounded topotecan conditioned in common off-label syringes for intravitreal use.
  Scientific reports · 2025 · Bello W, Hosotte C, Stampfli C, et al.
  PMID 41258448DOI 10.1038/s41598-025-24557-9

The 10 most recently updated of 487 ClinicalTrials.gov registrations naming Topotecan as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• Eflornithine (DFMO) for Ewing Sarcoma and Osteosarcoma
  Recruiting · Phase 2 · Interventional · 406 enrolled · Milton S. Hershey Medical Center
  NCT07321912updated 2026-06-12
• Dinutuximab With Chemotherapy, Surgery and Stem Cell Transplantation for the Treatment of Children With Newly Diagnosed High Risk Neuroblastoma
  Recruiting · Phase 3 · Interventional · 478 enrolled · National Cancer Institute (NCI)
  NCT06172296updated 2026-06-12
• Targeting Pediatric Brain Tumors and Relapsed/Refractory Solid Tumors With Sodium Glucose Cotransporter 2 Inhibitors (SGLT2i)
  Recruiting · Phase 1 · Interventional · 20 enrolled · Washington University School of Medicine
  NCT05521984updated 2026-06-10
• Study to Assess the Efficacy of Rina-S Compared to Treatment of Investigator's Choice in Participants With Platinum Resistant Ovarian Cancer
  Active not recruiting · Phase 3 · Interventional · 530 enrolled · Genmab
  NCT06619236updated 2026-06-08
• Pilot Study of IT Topotecan and Maintenance Chemotherapy for HR-EBTs in Children < 6 Years, Post Consolidation
  Recruiting · Early phase 1 · Interventional · 15 enrolled · C17 Council
  NCT06942039updated 2026-06-08
• Intra-Arterial Chemotherapy for Newly Diagnosed, Residual, or Recurrent Atypical Choroid Plexus Papilloma and Choroid Plexus Carcinoma Prior to Second-Look Surgery
  Terminated · Phase 1 · Interventional · 1 enrolled · Weill Medical College of Cornell University
  NCT04994977updated 2026-06-08
• An 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
  Not yet recruiting · Phase 3 · Interventional · 82 enrolled · Genmab
  NCT07604766updated 2026-06-02
• A Study of Ifinatamab Deruxtecan Versus Treatment of Physician's Choice in Subjects With Relapsed Small Cell Lung Cancer
  Recruiting · Phase 3 · Interventional · 540 enrolled · Daiichi Sankyo
  NCT06203210updated 2026-06-02
• Dinutuximab, Sargramostim, and Combination Chemotherapy in Treating Patients With Newly Diagnosed High-Risk Neuroblastoma
  Completed · Phase 2 · Interventional · 42 enrolled · National Cancer Institute (NCI)
  NCT03786783updated 2026-06-02
• Testing the Addition of an Anti-cancer Drug, BAY 1895344, to Usual Chemotherapy for Advanced Stage Solid Tumors, With a Specific Focus on Patients With Small Cell Lung Cancer, Poorly Differentiated Neuroendocrine Cancer, and Pancreatic Cancer
  Active not recruiting · Phase 1 · Interventional · 29 enrolled · National Cancer Institute (NCI)
  NCT04514497updated 2026-06-02

Frequently asked questions

How does Topotecan work?

Topoisomerase I relieves torsional strain in DNA by inducing reversible single-strand breaks. Topotecan binds to the topoisomerase I-DNA complex and prevents re-ligation of these single-strand breaks.

What is Topotecan used for?

According to FDA labeling, Topotecan carries indications including: Topotecan Injection is indicated for the treatment of patients with small cell lung cancer (SCLC) with platinum-sensitive disease who progressed at least 60 days after initiation of first-line chemotherapy. Topotecan Injection is a topoisomerase inhibitor indicated for treatment of small cell lung cancer (SCLC) platinum-sensitive disease in patients who progressed at least 60 days after initiation of first-line chemotherapy.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Topotecan?

Topotecan is classified as Topoisomerase 1 (TOP1) inhibitors, Topoisomerase Inhibitor, Topoisomerase Inhibitors, Decreased DNA Replication.

What are the brand names for Topotecan?

Topotecan is marketed under brand names including Hycamtin.

What are the contraindications for Topotecan?

Topotecan labeling lists contraindications including: Topotecan Injection is contraindicated in patients who have a history of severe hypersensitivity reactions to topotecan. Reactions have included anaphylactoid reactions [see Adverse Reactions (6.2) ] .. Always consult the full prescribing information and a clinician.