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Cyclophosphamide (CTX)
Cyclophosphamide is an alkylating agent that cross-links DNA strands, preventing cell division and triggering apoptosis in rapidly dividing cells.
Cyclophosphamide is an alkylating agent that cross-links DNA strands, preventing cell division and triggering apoptosis in rapidly dividing cells. Used for Lymphomas (Hodgkin and non-Hodgkin), Breast cancer, Ovarian cancer.
At a glance
| Generic name | Cyclophosphamide (CTX) |
|---|---|
| Also known as | Cytoxan, Neosar |
| Sponsor | Asahi Kasei Pharma Corporation |
| Drug class | Alkylating agent |
| Target | DNA (non-specific alkylation) |
| Modality | Small molecule |
| Therapeutic area | Oncology, Immunology |
| Phase | Phase 3 |
Mechanism of action
Cyclophosphamide is a nitrogen mustard derivative that requires hepatic activation to form active metabolites. These metabolites covalently bind to DNA, creating inter- and intra-strand cross-links that impair DNA replication and transcription. This leads to cell cycle arrest and apoptosis, making it effective against both malignant cells and rapidly proliferating immune cells in autoimmune conditions.
Approved indications
- Lymphomas (Hodgkin and non-Hodgkin)
- Breast cancer
- Ovarian cancer
- Lung cancer
- Severe systemic lupus erythematosus (SLE)
- Vasculitis
- Multiple myeloma
Common side effects
- Myelosuppression (neutropenia, thrombocytopenia)
- Nausea and vomiting
- Alopecia
- Hemorrhagic cystitis
- Infertility
- Secondary malignancies
- Immunosuppression/infections
- Cardiotoxicity (at high doses)
Key clinical trials
- Nivolumab in Combination With Chemo-Immunotherapy for the Treatment of Newly Diagnosed Primary Mediastinal B-Cell Lymphoma (PHASE3)
- Combination Chemotherapy With or Without Ganitumab in Treating Patients With Newly Diagnosed Metastatic Ewing Sarcoma (PHASE3)
- Novel Combination Therapy in the Treatment of Relapsed and Refractory Aggressive B-Cell Lymphoma (PHASE2)
- Pilot Trial of Allogeneic Blood or Marrow Transplantation for Primary Immunodeficiencies (PHASE2)
- Testing the Addition of the Anti-cancer Drug Venetoclax and/or the Anti-cancer Immunotherapy Blinatumomab to the Usual Chemotherapy Treatment for Infants With Newly Diagnosed KMT2A-rearranged or KMT2A-non-rearranged Leukemia (PHASE2)
- Metabolically Fit CD19 CAR T-cell Therapy With CD34 Selection in Patients With CD19+ Relapsed/Refractory NHL, CLL/SLL (PHASE1, PHASE2)
- A Clinical Study of Sacituzumab Tirumotecan (Sac-TMT, MK-2870) in People With Breast Cancer (MK-2870-032) (PHASE3)
- A Study to Investigate Blinatumomab in Combination With Chemotherapy in Patients With Newly Diagnosed B-Lymphoblastic Leukemia (PHASE3)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Cyclophosphamide (CTX) CI brief — competitive landscape report
- Cyclophosphamide (CTX) updates RSS · CI watch RSS
- Asahi Kasei Pharma Corporation portfolio CI