OnCo
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Topotecan

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

Approved 1996 (IV) and 2007 (oral) for relapsed SCLC after platinum. ORR ~20%, median OS ~6-8 months; heavy myelosuppression. Control arm in DeLLphi-304 (beaten by tarlatamab) and IDeate-Lung02.

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Topotecan
PubChem
Mechanism, step by step
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1.Enters cell as active lactone

Modality
Cytotoxic (topoisomerase-I inhibitor)
Mechanism
Topoisomerase-I poison; stabilises the cleavage complex causing replication-associated DNA breaks.
Brand / code
Hycamtin
Dosing & schedule
Route
Intravenous or oral
Schedule
1.5 mg/m² IV days 1-5 every 21 days, or 2.3 mg/m² oral days 1-5
Dose modifications
Frequent dose reduction for neutropenia; renal dose adjustment
Monitoring
Weekly blood counts
Medicare
Part B or Part D

Oral anticancer drug with an injectable equivalent used for the same indication, so Medicare covers the oral form under Part B (the oral anticancer drug benefit) as well as under Part D; the pharmacy bills whichever applies. IV topotecan is Part B.

Commercial insurance
covered on label

Multi-source generic; covered under the medical benefit without prior authorisation in most plans, as part of standard regimens.

Sources: Medicare.gov: Chemotherapy · Medicare.gov: Prescription drugs (outpatient, Part B) · Medicare.gov: Drug coverage (Part D) · Medicare.gov: Costs for Medicare drug coverage (annual out-of-pocket cap). Not medical or financial advice; verify with your plan.

NICE recommendedNICE TA184 · 2009SMC: accepted
Appraised for
Relapsed small-cell lung cancer
Notes
Also recurrent cervical cancer with cisplatin (TA183, 2009). Generic.
NHS England
Routinely funded for the appraised indication (or via managed access)

Sources: NICE TA184 · SMC advice: topotecan. Funding decisions are indication-specific and change monthly; verify with NICE and your treating team.

Regulatory

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Approvals

RegionYearIndication
US1996Relapsed SCLC (IV)
US2007Relapsed SCLC (oral)

Safety

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Toxicity profile
Adverse eventAny gradeGrade 3+
Neutropenia (grade 4)
70%
Thrombocytopenia (grade 4)
27%
Anaemia
90%
Diarrhoea (oral)
30%

Events listed without rates were not read from a primary source; see the label. Blank cells mean the figure was not sourced, not that it is zero.

Trials

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ClinicalTrials.gov · phase 2/3
refreshed 2026-09-09
335 studies13 recruiting64 Phase 242 Phase 3
Search “Topotecan” on ClinicalTrials.gov →
Counts are from a name search and may include unrelated studies; up to 100 studies are summarised.

Recruiting now (live from ClinicalTrials.gov)

Recruiting trials near you · live from ClinicalTrials.gov
Topotecan
intervention: Topotecan
Open on ClinicalTrials.gov →

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Landmark trials in OnCo

Latest papers

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Literature trend104 papers in the last 12 months+7% vs prior 12How this is computed
Latest papers · live from Europe PMC
Open in Europe PMC

Query for this drug: (TITLE:"Topotecan" OR ABSTRACT:"Topotecan" OR TITLE:"Hycamtin" OR ABSTRACT:"Hycamtin") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about Topotecan, not a curated reading list.

Connected

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