Cyclophosphamide

The mechanism of action has not been fully characterized. However, cross-linking of tumor cell DNA may be involved.

• Cyclophosphamide for injection is an alkylating drug indicated for treatment of adults and pediatric patients with: Malignant Diseases: malignant lymphomas: Hodgkin’s disease, lymphocytic lymphoma, mixed-cell type lymphoma, histiocytic lymphoma, Burkitt’s lymphoma; multiple myeloma, leukemias, mycosis fungoides, neuroblastoma, adenocarcinoma of ovary, retinoblastoma, breast carcinoma ( 1.1 ) Minimal Change Nephrotic Syndrome in Pediatric Patients: biopsy proven minimal change nephrotic syndrome patients who failed to adequately respond to or are unable to tolerate adrenocorticosteroid therapy ( 1.2 ) Limitations of Use: The safety and effectiveness for the treatment of nephrotic syndrome in adults or other renal disease has not been established.ICD-10: C85.90, C90.00

• Hypersensitivity Cyclophosphamide for injection is contraindicated in patients who have a history of severe hypersensitivity reactions to cyclophosphamide, any of its metabolites, or to other components of the product. Anaphylactic reactions including death have been reported with cyclophosphamide.contraindicated

During or immediately after cyclophosphamide for injection administration, administer adequate amounts of fluid to reduce the risk of urinary tract toxicity ( 2.1 ). Malignant Diseases: Adult and Pediatric Patients ( 2.2 ) Intravenous: Initial course for patients with no hematologic deficiency: 40 mg per kg to 50 mg per kg in divided doses over 2 to 5 days. Other regimens include 10 mg per kg to 15 mg per kg given every 7 to 10 days or 3 mg per kg to 5 mg per kg twice weekly. Oral: 1 mg per kg per day to 5 mg per kg per day for both initial and maintenance dosing. Minimal Change Nephrotic Syndrome in Pediatric Patients ( 2.3 ) Oral: 2 mg per kg daily for 8 to 12 weeks (maximum cumulative dose 168 mg per kg). Treatment beyond 90 days increases the probability of sterility in males. ( 8.4 ) 2.1 Important Administration Instructions During or immediately after the administration of cyclophosphamide for injection, adequate amounts of fluid should be ingested or infused to force diuresis in order to reduce the risk of urinary tract toxicity. Therefore, cyclophosphamide for injection should be administered in the morning. 2.2 Recommended Dosage for Malignant Diseases Adults and Pediatric Patients Intravenous Use When used as the only oncolytic drug therapy, the recommended dosage for the initial course of cyclophosphamide for injection for patients with no hematologic deficiency is 40 mg per kg to 50 mg per kg given intravenously in divided doses over a period of 2 to 5 days.

Myelosuppression, Immunosuppression, Bone Marrow Failure and Infections: Severe immunosuppression may lead to serious and sometimes fatal infections. Close hematological monitoring is required. ( 5.1 ) Urinary Tract and Renal Toxicity: Hemorrhagic cystitis, pyelitis, ureteritis, and hematuria can occur. Urotoxicity can be fatal. Exclude or correct any urinary tract obstructions prior to treatment. ( 5.2 ) Cardiotoxicity: Myocarditis, myopericarditis, pericardial effusion, arrythmias and congestive heart failure, which may be fatal, have been reported. Monitor patients, especially those with risk factors for cardiotoxicity or pre-existing cardiac disease. ( 5.3 ) Pulmonary Toxicity: Pneumonitis, pulmonary fibrosis and pulmonary veno-occlusive disease leading to respiratory failure may occur. Monitor patients for signs and symptoms of pulmonary toxicity. ( 5.4 ) Secondary malignancies ( 5.5 ) Veno-occlusive Liver Disease: Fatal outcome can occur. ( 5.6 ) Embryo-Fetal Toxicity: Can cause fetal harm. Advise patients of reproductive potential of the portential risk to a fetus and to use effective contraception. ( 5.7 , 8.1 , 8.3 ) 5.1 Myelosuppression, Immunosuppression, Bone Marrow Failure and Infections Cyclophosphamide can cause myelosuppression (leukopenia, neutropenia, thrombocytopenia and anemia), bone marrow failure, and severe immunosuppression which may lead to serious and sometimes fatal infections, including sepsis and septic shock. Latent infections can be reactivated [see Adverse Reactions ( 6.2 )] .

The following adverse reactions are discussed in more detail in other sections of the labeling. Hypersensitivity [see Contraindications ( 4 )] Myelosuppression, Immunosuppression, Bone Marrow Failure, and Infections [see Warnings and Precautions ( 5.1 )] Urinary Tract and Renal Toxicity [see Warnings and Precautions ( 5.2 )] Cardiotoxicity [see Warnings and Precautions ( 5.3 )] Pulmonary Toxicity [see Warnings and Precautions ( 5.4 )] Secondary Malignancies [see Warnings and Precautions ( 5.5 )] Veno-occlusive Liver Disease [see Warnings and Precautions ( 5.6 )] Infertility [ see Warnings and Precautions ( 5.8 ) and Use in Specific Populations ( 8.3 , 8.4 )] Impaired Wound Healing [see Warnings and Precautions ( 5.9 )] Hyponatremia [see Warnings and Precautions ( 5.10 )] Adverse reactions reported most often include neutropenia, febrile neutropenia, fever, alopecia, nausea, vomiting, and diarrhea. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact XGen Pharmaceuticals DJB, Inc. at 1-866-390-4411 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . 6.1 Clinical Trials and Postmarketing Experience The following adverse reactions associated with the use of cyclophosphamide were identified in clinical studies or postmarketing reports. Because some of these reactions were reported voluntarily from a population of uncertain size, it is not always possible to reliably estimate their frequency or establish a causal relationship to drug exposure. The most common adverse reactions were neutropenia, febrile neutropenia, fever, alopecia, nausea, vomiting, and diarrhea.

Lactation: Advise not to breastfeed. ( 8.2 ) Renal Impairment: Monitor for toxicity in patients with moderate and severe renal impairment. ( 8.6 , 12.3 ) See 17 for PATIENT COUNSELING INFORMATION 8.1 Pregnancy Risk Summary Based on its mechanism of action and published reports of effects in pregnant patients or animals, cyclophosphamide for injection can cause fetal harm when administered to a pregnant woman [see Clinical Pharmacology ( 12.1 ) and Nonclinical Toxicology ( 13.1 )]. Exposure to cyclophosphamide during pregnancy may cause fetal malformations, miscarriage, fetal growth retardation, and toxic effects in the newborn [see Data] . Cyclophosphamide is teratogenic and embryo-fetal toxic in mice, rats, rabbits and monkeys [see Data] . Advise pregnant women and females of reproductive potential of the potential risk to the fetus. The estimated background risk of major birth defects and miscarriage for the indicated population is unknown. In the U.S. general population, the estimated background risk of major birth defects is 2%-4% and miscarriage is 15%-20% of clinically recognized pregnancies. Data Human Data Malformations of the skeleton, palate, limbs and eyes as well as miscarriage have been reported after exposure to cyclophosphamide in the first trimester.

Metabolism

Cyclophosphamide is a prodrug. Cyclophosphamide pharmacokinetics are linear over the approved recommended dose range.

No specific antidote for cyclophosphamide is known. Overdosage should be managed with supportive measures, including appropriate treatment for any concurrent infection, myelosuppression, or cardiac toxicity should it occur. Serious consequences of overdosage include manifestations of dose dependent toxicities such as myelosuppression, urotoxicity, cardiotoxicity (including cardiac failure), veno-occlusive hepatic disease, and stomatitis [see Warnings and Precautions ( 5.1 , 5.2 , 5.3 , and 5.6 )] . Patients who received an overdose should be closely monitored for the development of toxicities, and hematologic toxicity in particular. Cyclophosphamide and its metabolites are dialyzable. Therefore, rapid hemodialysis is indicated when treating any suicidal or accidental overdose or intoxication [ see Clinical Pharmacology ( 12.3 )]. Cystitis prophylaxis with mesna may be helpful in preventing or limiting urotoxic effects with cyclophosphamide overdose.

• Nov 16, 1959NDANDA012141Baxter Hlthcare
• May 21, 2008ANDAANDA040745Baxter Hlthcare
• Sep 16, 2013NDANDA203856Hikma
• Jul 30, 2020NDANDA212501Dr Reddys
• Aug 25, 2021NDANDA210735Eugia Pharma Speclts
• Jun 7, 2023NDANDA210852Avyxa Holdings
• Jun 27, 2023NDANDA217651Baxter Hlthcare Corp
• Sep 12, 2023NDANDA217150Sandoz

1. 1Off Label Use15,8669.2%
2. 2Febrile Neutropenia14,2188.3%
3. 3Neutropenia10,3386.0%
4. 4Drug Ineffective10,3336.0%
5. 5Pyrexia9,0645.3%
6. 6Disease Progression7,9254.6%
7. 7Pneumonia6,5123.8%
8. 8Product Use In Unapproved Indication6,4463.7%
9. 9Thrombocytopenia6,3493.7%
10. 10Nausea6,1033.5%
11. 11Anaemia5,9813.5%
12. 12Sepsis5,6103.3%
13. 13Diarrhoea5,3863.1%
14. 14Death5,1523.0%
15. 15Vomiting4,7992.8%

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

• Ellagic Acid Protects Multiple Organ Injury Induced by Cyclophosphamide-Mediated Oxidative Stress: Biochemical and Histological Insights.
  Journal of biochemical and molecular toxicology · 2026 · Sahu AA, Nirala SK, Bhadauria M, et al.
  PMID 42231620DOI 10.1002/jbt.70925
• Post-Transplant Cyclophosphamide Cardiac Toxicity: RN-APP Collaborative Assessment and Decision-Making.
  Clinical journal of oncology nursing · 2026 · Wheatley TT
  PMID 42227790DOI 10.1188/26.CJON.187-191
• [The Application of Human Lung Organoids for Assessing the Cyclophosphamide-Induced Pulmonary Toxicity and Potential Mechanisms].
  Zhongguo shi yan xue ye xue za zhi · 2026 · Wang YD, Li XT, Zhu H, et al.
  PMID 42227460DOI 10.19746/j.cnki.issn1009-2137.2026.02.041
• [Efficacy of Low-Dose Post-Transplant Cyclophosphamide for GVHD Prophylaxis after Allogeneic Stem Cell Transplantation].
  Zhongguo shi yan xue ye xue za zhi · 2026 · Lu L, Yu XQ, Wang LX, et al.
  PMID 42227454DOI 10.19746/j.cnki.issn1009-2137.2026.02.035
• Comparison of the efficacy of pre-transplant and post-transplant antithymocyte globulin combined with post-transplant cyclophosphamide for graft-versus-host disease prophylaxis in myeloablative haploidentical peripheral blood stem cell transplantation.
  Cell transplantation · 2026 · Jin X, Dong T, Ji J, et al.
  PMID 42223061DOI 10.1177/09636897261454024
• Soluble Dietary Fiber from Polygonatum cyrtonema Hua Attenuates Cyclophosphamide-Induced Intestinal Injury in Mice.
  International journal of molecular sciences · 2026 · Zeng L, Cui S, Peng T, et al.
  PMID 42196515DOI 10.3390/ijms27104537
• A New Food Supplement Product Containing Ergothioneine Protects Against Cyclophosphamide-Induced Primary Ovarian Insufficiency in Rats.
  Molecular nutrition & food research · 2026 · Jin C, Shi M, Liu H, et al.
  PMID 42165092DOI 10.1002/mnfr.70509
• Cytokine release syndrome after allogeneic hematopoietic stem cell transplantation using posttransplant cyclophosphamide: current understanding and management.
  Frontiers in immunology · 2026 · Wang J, Arora K, de Lima M, et al.
  PMID 42158862DOI 10.3389/fimmu.2026.1642583

The 10 most recently updated of 5,668 ClinicalTrials.gov registrations naming Cyclophosphamide 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
• Phase I/II Study of Engineered T Cell Receptor-Modified NK Cells Targeting PRAME in Conjunction With Lymphodepleting Chemotherapy for the Management of Relapse/Refractory Myeloid Malignancies
  Recruiting · Phase 1 · Phase 2 · Interventional · 44 enrolled · M.D. Anderson Cancer Center
  NCT06383572updated 2026-06-12
• CD30 CAR for CD30+ NSGCT
  Completed · Phase 2 · Interventional · 2 enrolled · UNC Lineberger Comprehensive Cancer Center
  NCT05634785updated 2026-06-12
• In Vitro Expanded Allogeneic Epstein-Barr Virus Specific Cytotoxic T-Lymphocytes (EBV-CTLs) Genetically Targeted to the CD19 Antigen in B-cell Malignancies
  Active not recruiting · Phase 1 · Interventional · 19 enrolled · Memorial Sloan Kettering Cancer Center
  NCT01430390updated 2026-06-12
• CD22 CAR T-cells to Extend Remission Following Commercial CD19 CAR T-cells in Children, Adolescents, and Adults With Relapsed/Refractory B-cell Acute Lymphoblastic Leukemia
  Recruiting · Phase 2 · Interventional · 20 enrolled · National Cancer Institute (NCI)
  NCT07328503updated 2026-06-12
• Autologous B7-H3 Chimeric Antigen Receptor T Cells in Previously Treated Extensive-Stage Small Cell Lung Cancer With Recurrent or Refractory Disease
  Not yet recruiting · Phase 1 · Interventional · 40 enrolled · National Cancer Institute (NCI)
  NCT07509034updated 2026-06-12
• E7 TCR-T Cell Immunotherapy for Human Papillomavirus (HPV) Associated Cancers
  Recruiting · Phase 2 · Interventional · 20 enrolled · Christian Hinrichs
  NCT05686226updated 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
• Stem Cell Transplantation for Participants With Germline RUNX1 Associated Blood Cancers
  Not yet recruiting · Phase 2 · Interventional · 98 enrolled · National Cancer Institute (NCI)
  NCT07524530updated 2026-06-12
• A Study of Fludarabine Dosing in Children and Young Adults With B-cell Acute Lymphoblastic Leukemia
  Recruiting · Phase 3 · Interventional · 130 enrolled · Memorial Sloan Kettering Cancer Center
  NCT07223021updated 2026-06-12

CPIC-curated drug–gene pairs for Cyclophosphamide. Levels describe the strength of curated evidence and guideline status — never a recommendation to test or to adjust therapy.

• GSTP1CPIC D (provisional)ClinPGx 3
• NQO1CPIC D (provisional)ClinPGx 3
• SOD2CPIC D (provisional)ClinPGx 3

Frequently asked questions

How does Cyclophosphamide work?

The mechanism of action has not been fully characterized. However, cross-linking of tumor cell DNA may be involved.

What is Cyclophosphamide used for?

According to FDA labeling, Cyclophosphamide carries indications including: Cyclophosphamide for injection is an alkylating drug indicated for treatment of adults and pediatric patients with: Malignant Diseases: malignant lymphomas: Hodgkin’s disease, lymphocytic lymphoma, mixed-cell type lymphoma, histiocytic lymphoma, Burkitt’s lymphoma; multiple myeloma, leukemias, mycosis fungoides, neuroblastoma, adenocarcinoma of ovary, retinoblastoma, breast carcinoma ( 1.1 ) Minimal Change Nephrotic Syndrome in Pediatric Patients: biopsy proven minimal change nephrotic syndrome patients who failed to adequately respond to or are unable to tolerate adrenocorticosteroid therapy ( 1.2 ) Limitations of Use: The safety and effectiveness for the treatment of nephrotic syndrome in adults or other renal disease has not been established.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Cyclophosphamide?

Cyclophosphamide is classified as Nitrogen mustard analogues, Alkylating Activity, Decreased DNA Integrity, Decreased DNA Replication.

What are the brand names for Cyclophosphamide?

Cyclophosphamide is marketed under brand names including Frindovyx.

What are the contraindications for Cyclophosphamide?

Cyclophosphamide labeling lists contraindications including: Hypersensitivity Cyclophosphamide for injection is contraindicated in patients who have a history of severe hypersensitivity reactions to cyclophosphamide, any of its metabolites, or to other components of the product. Anaphylactic reactions including death have been reported with cyclophosphamide.. Always consult the full prescribing information and a clinician.