# Topotecan

Source: https://onco.cc/drugs/topotecan/  
OnCo record `topotecan` (Treatment). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Topotecan is the long-standing second-line chemotherapy for relapsed small-cell lung cancer, and now the comparator that new drugs must beat.

## Summary

Topotecan is a topoisomerase-I poison that stabilises the enzyme-DNA cleavage complex so replication forks break the DNA. Approved 1996 (IV) and 2007 (oral) for relapsed SCLC after platinum, it is given on days 1 to 5 of a 21-day cycle and also has roles in ovarian and cervical cancer. Activity is modest, with ORR around 20% and median OS around 6 to 8 months, and myelosuppression is heavy: grade 4 neutropenia in 70% of patients, so weekly blood counts and dose reductions are routine. It was the control arm in DeLLphi-304, where tarlatamab beat it with OS 13.6 versus 8.3 months, and is the comparator again in IDeate-Lung02; in ATLANTIS, lurbinectedin plus doxorubicin did not outperform it. Topotecan defines the floor for relapsed small-cell lung cancer, and new agents are judged by how far they rise above it.

## Fields

- Kind: Treatment
- Status: approved
- Last checked: 2026-09-07
- Brand: Hycamtin
- Modality: Cytotoxic (topoisomerase-I inhibitor)
- Mechanism: Topoisomerase-I poison; stabilises the cleavage complex causing replication-associated DNA breaks.
- Approvals: US 1996: Relapsed SCLC (IV); US 2007: Relapsed SCLC (oral); England (NICE) 2009: Relapsed small-cell lung cancer, oral topotecan, only where re-treatment with the first-line regimen is inappropriate and cyclophosphamide, doxorubicin and vincristine is contraindicated
- Dosing: Intravenous or oral; 1.5 mg/m² IV days 1-5 every 21 days, or 2.3 mg/m² oral days 1-5
- Toxicity (grade 3+): Neutropenia (grade 4) 70%; Thrombocytopenia (grade 4) 27%

## Sources

- Wikipedia: https://en.wikipedia.org/wiki/Topotecan
- FDA label (DailyMed): https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Topotecan
- NICE TA184: topotecan for the treatment of relapsed small-cell lung cancer: https://www.nice.org.uk/guidance/ta184

## Connected records

- cancers: [Cervical cancer](https://onco.cc/cancers/cervical/), [Ewing sarcoma](https://onco.cc/cancers/ewing-sarcoma/), [Extensive-stage small-cell lung cancer](https://onco.cc/cancers/extensive-stage-sclc/), [Extrapulmonary neuroendocrine carcinoma](https://onco.cc/cancers/extrapulmonary-nec/), [High-risk neuroblastoma](https://onco.cc/cancers/neuroblastoma-high-risk/), [Limited-stage small-cell lung cancer](https://onco.cc/cancers/limited-stage-sclc/), [Lung cancer (all types)](https://onco.cc/cancers/lung-cancer/), [Ovarian cancer](https://onco.cc/cancers/ovarian/), [Platinum-resistant ovarian cancer](https://onco.cc/cancers/platinum-resistant-ovarian-cancer/), [Recurrent or metastatic cervical cancer](https://onco.cc/cancers/recurrent-metastatic-cervical-cancer/), [Retinoblastoma](https://onco.cc/cancers/retinoblastoma/), [Small-cell lung cancer](https://onco.cc/cancers/sclc/)
- technologies: [Cytotoxic chemotherapy](https://onco.cc/technologies/cytotoxic-chemotherapy/), [Topoisomerase-I inhibitors (and ADC payloads)](https://onco.cc/technologies/topoisomerase-inhibitors/)
- companies: [GSK](https://onco.cc/companies/gsk/), [Novartis](https://onco.cc/companies/novartis/)
- trials: [A Study Comparing BL-B01D1 With Topotecan in Patients With Recurrent Small Cell Lung Cancer(PANKU-Lung03)](https://onco.cc/trials/nct06500026/), [A Study of GSK5764227 in Participants With Relapsed Small Cell Lung Cancer (SCLC)](https://onco.cc/trials/nct07099898/), [A Study of SHR-A1811 in Subjects With Ovarian Cancer](https://onco.cc/trials/nct06828354/), [A Study to Compare Sacituzumab Tirumotecan (MK-2870) Monotherapy Versus Treatment of Physician's Choice as Second-line Treatment for Participants With](https://onco.cc/trials/nct06459180/), [A Study to Investigate Mocertatug Rezetecan Compared With Standard of Care in Participants With Platinum-resistant Ovarian Cancer (BEHOLD-Ovarian01)](https://onco.cc/trials/nct07286266/), [An Extension Study to Assess the Efficacy of Rina-S Compared to Treatment of Investigator's Choice in Participants With Platinum Resistant Ovarian Can](https://onco.cc/trials/nct07604766/), [ANBL0531](https://onco.cc/trials/anbl0531/), [ANBL17P1](https://onco.cc/trials/anbl17p1/), [ATLANTIS](https://onco.cc/trials/atlantis/), [AURELIA](https://onco.cc/trials/aurelia/), [DeLLphi-304](https://onco.cc/trials/dellphi-304/), [IDeate-Lung02](https://onco.cc/trials/ideate-lung02/), [JK-1201I Compared with Topotecan in Patients with Relapsed Extensive Stage Small Cell Lung Cancer](https://onco.cc/trials/nct06581380/), [Lurbinectedin basket trial, small-cell lung cancer cohort](https://onco.cc/trials/lurbinectedin-basket-sclc/), [Lurbinectedin or in Combination with Irinotecan Versus Topotecan in Patients with Relapsed SCLC](https://onco.cc/trials/nct06496048/), [Organoid-guided vs Topotecan Therapy in Relapsed Extensive-Stage Small Cell Lung Cancer](https://onco.cc/trials/nct07106528/), [Pharmacogenomics ANDA SNP Clinical Study - Topotecan and Single Nucleotide Polymorphisms](https://onco.cc/trials/nct07145333/), [REFRaME-O1: A Study to Investigate the Efficacy and Safety of Luveltamab Tazevibulin Versus Investigator's Choice (IC) Chemotherapy in Women With Ovarian Cancer (Including Fallopian Tube or Primary Peritoneal Cancers) Expressing FOLR1](https://onco.cc/trials/nct05870748/), [Retinoblastoma Phase II Expanded Access Clinical Trial](https://onco.cc/trials/nct06679634/), [Safety, Preliminary Effectiveness of BNT327, an Investigational Therapy for Patients With Small-cell Lung Cancer in Combination With Chemotherapy](https://onco.cc/trials/nct06449209/), [Study Comparing Rovalpituzumab Tesirine Versus Topotecan in Subjects With Advanced or Metastatic Small Cell Lung Cancer With High Levels of Delta-like Protein 3 (DLL3) and Who Have First Disease Progression During or Following Front-line Platinum-based Chemotherapy (TAHOE)](https://onco.cc/trials/nct03061812/), [Study of LY01610 in Patients With Recurrent Small Cell Lung Cancer](https://onco.cc/trials/nct06128837/), [Study Of Palbociclib Combined With Chemotherapy In Pediatric Patients With Recurrent/Refractory Solid Tumors](https://onco.cc/trials/nct03709680/), [Study of Sacituzumab Govitecan Versus Standard of Care in Participants With Previously Treated Extensive Stage Small Cell Lung Cancer](https://onco.cc/trials/nct06801834/), [Study to Assess the Efficacy of Rina-S Compared to Treatment of Investigator's Choice in Participants With Platinum Resistant Ovarian Cancer](https://onco.cc/trials/nct06619236/)
- terms: [Lung cancer drugs in England: what NICE has recommended](https://onco.cc/terms/lung-uk-drug-access/)
- targets: [Topoisomerase I (TOP1)](https://onco.cc/targets/top1/)
- drugs: [Trilaciclib](https://onco.cc/drugs/trilaciclib/)
- pathways: [DNA replication & origin licensing](https://onco.cc/pathways/dna-replication-licensing/), [Small cell lung cancer (KEGG map)](https://onco.cc/pathways/sclc-signalling/)

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JSON: https://onco.cc/api/v1/entities/topotecan.json