Mechlorethamine
/api/v1/drug/mechlorethamineMechanism of action
Sourced from openFDAMechlorethamine, also known as nitrogen mustard, is an alkylating agent which inhibits rapidly proliferating cells.
Indications
Sourced from openFDA- VALCHLOR is indicated for the topical treatment of Stage IA and IB mycosis fungoides-type cutaneous T-cell lymphoma in patients who have received prior skin-directed therapy. VALCHLOR is an alkylating drug indicated for the topical treatment of Stage IA and IB mycosis fungoides-type cutaneous T-cell lymphoma in patients who have received prior skin-directed therapy ( 1 ).ICD-10: C85.90
Contraindications
Sourced from openFDA- The use of VALCHLOR is contraindicated in patients with known severe hypersensitivity to mechlorethamine. Hypersensitivity reactions, including anaphylaxis, have occurred with topical formulations of mechlorethamine.contraindicated
Dosage & administration
Sourced from openFDAFor topical dermatological use only ( 2.1 ). Apply a thin film once daily to affected areas of the skin ( 2.1 , 2.2 ). 2.1 Dosing and Dose Modification For Topical Dermatological Use Only Apply a thin film of VALCHLOR gel once daily to affected areas of the skin. Stop treatment with VALCHLOR for any grade of skin ulceration, blistering, or moderately-severe or severe dermatitis (i.e., marked skin redness with edema) [ see Warnings and Precautions ( 5.3 ) ]. Upon improvement, treatment with VALCHLOR can be restarted at a reduced frequency of once every 3 days. If reintroduction of treatment is tolerated for at least one week, the frequency of application can be increased to every other day for at least one week and then to once daily application if tolerated. 2.2 Application Instructions VALCHLOR is a cytotoxic drug. Follow applicable special handling and disposal procedures. 1 Patients must wash hands thoroughly with soap and water after handling or applying VALCHLOR. Caregivers must wear disposable nitrile gloves when applying VALCHLOR to patients and wash hands thoroughly with soap and water after removal of gloves. If there is accidental skin exposure to VALCHLOR, caregivers must immediately wash exposed areas thoroughly with soap and water for at least 15 minutes and remove contaminated clothing [ see Warnings and Precautions ( 5.2 ) ]. Patients or caregivers should follow these instructions when applying VALCHLOR: Apply immediately or within 30 minutes after removal from the refrigerator. Return VALCHLOR to the refrigerator immediately after each use.
Warnings & precautions
Sourced from openFDAMucosal or eye injury: VALCHLOR exposure to mucous membranes, especially of the eyes, can cause mucosal injury which may be severe. Eye injury may lead to blindness. Immediately irrigate for at least 15 minutes followed by immediate medical consultation ( 5.1 ). Secondary exposure to VALCHLOR: individuals other than the patient must avoid skin contact with VALCHLOR ( 2.2 , 5.2 ). Dermatitis: Monitor patients for redness, swelling, inflammation, itchiness, blisters, ulceration, and secondary skin infections. Stop treatment or reduce dose frequency ( 2.1 , 5.3 ). Non-melanoma skin cancer: Monitor patients during and after treatment ( 5.4 ). Embryo-fetal toxicity: May cause fetal harm ( 5.5 ). Flammable gel: VALCHLOR is an alcohol-based gel. Avoid fire, flame, and smoking until the gel has dried ( 2.2 , 5.6 ). 5.1 Mucosal or Eye Injury Exposure of the eyes to mechlorethamine causes pain, burns, inflammation, photophobia, and blurred vision. Blindness and severe irreversible anterior eye injury may occur. Advise patients that if eye exposure occurs, (1) immediately irrigate for at least 15 minutes with copious amounts of water, normal saline, or a balanced salt ophthalmic irrigating solution and (2) obtain immediate medical care (including ophthalmologic consultation). Exposure of mucous membranes such as the oral mucosa or nasal mucosa causes pain, redness, and ulceration, which may be severe. Should mucosal contact occur, immediately irrigate for at least 15 minutes with copious amounts of water, followed by immediate medical consultation.
Adverse reactions
Sourced from openFDAThe following clinically significant adverse reactions are discussed in greater detail in other sections of the prescribing information: Mucosal or eye injury [ see Warnings and Precautions ( 5.1 ) ] Secondary exposure to VALCHLOR [ see Warnings and Precautions ( 5.2 ) ] Dermatitis [ see Warnings and Precautions ( 5.3 ) ] Non-melanoma skin cancer [ see Warnings and Precautions ( 5.4 ) ] The most common adverse reactions (≥5%) are dermatitis, pruritus, bacterial skin infection, skin ulceration or blistering, and hyperpigmentation ( 6.1 ). To report SUSPECTED ADVERSE REACTIONS, contact Helsinn Therapeutics (U.S.), Inc., at 1-855-482-5245 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 clinical practice. In a randomized, observer-blinded, controlled trial, VALCHLOR 0.016% (equivalent to 0.02% mechlorethamine HCl) was compared to an Aquaphor ® -based mechlorethamine HCl 0.02% ointment (Comparator) [ see Clinical Studies ( 14 ) ]. The maximum duration of treatment was 12 months. Sixty-three percent (63%) of patients in the VALCHLOR arm and 67% in the comparator arm completed 12 months of treatment. The body system associated with the most frequent adverse reactions was skin and subcutaneous tissue disorders.
Use in specific populations
Sourced from openFDALactation: Advise not to breastfeed ( 8.2 ). 8.1 Pregnancy Risk Summary Based on case reports in humans, findings in animal reproduction studies, its mechanism of action, and genotoxicity findings, mechlorethamine may cause fetal harm. Available published case reports in pregnant women receiving intravenous mechlorethamine demonstrate that mechlorethamine can cause major birth defects when a pregnant woman is systemically exposed. In animal reproduction studies, subcutaneous administration of mechlorethamine to pregnant rats and ferrets during organogenesis resulted in embryo‐fetal mortality, alterations to growth, and structural abnormalities. Based on limited available data with VALCHLOR use in pregnant women, if VALCHLOR is used during pregnancy or if the patient becomes pregnant while taking this drug, patient should be advised of the potential risk to the fetus. The estimated background risk of major birth defects and miscarriage for the indicated population is unknown. All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2-4% and 15-20%, respectively. Data Human Data The limited available data with VALCHLOR use in pregnant women does not show evidence of congenital malformation in newborns.
Pharmacokinetics
Sourced from openFDA- Metabolism
- Systemic exposure was undetectable after topical administration of VALCHLOR to patients. Blood samples were analyzed from 16 and 15 patients following treatment with VALCHLOR (mechlorethamine gel 0.016%) and an identical formulation consisting of mechlorethamine 0.032% w/w, respectively.
Approval history
Sourced from openFDA- Aug 23, 2013NDANDA202317Helsinn
FAERS reports
- 1Pruritus32114%
- 2Application Site Pruritus22410%
- 3Application Site Erythema2209.8%
- 4Application Site Pain2179.7%
- 5Erythema2109.4%
- 6Condition Aggravated1938.6%
- 7Off Label Use1767.9%
- 8Skin Discolouration1476.6%
- 9Rash1325.9%
- 10Skin Irritation1125.0%
- 11Blister1074.8%
- 12Application Site Discolouration1024.6%
- 13Application Site Irritation1014.5%
- 14Disease Progression994.4%
- 15Death984.4%
Literature
Recent PubMed references pinned to Mechlorethamine as a MeSH major topic. Citations link to pubmed.ncbi.nlm.nih.gov.
- P97 deficiency interferes with the recovery from acute nitrogen mustard-induced respiratory tract injury.International immunopharmacology · 2026 · Cheng J, Yang Y, Liu H, et al.PMID 41997052DOI 10.1016/j.intimp.2026.116643
- Salt form and phosphate modification of an SN-38-nitrogen mustard conjugate: Overcoming water solubility limitations, combined efficacy, and broadened antitumor Spectrum.Bioorganic chemistry · 2026 · Wang X, He M, Han X, et al.PMID 41865567DOI 10.1016/j.bioorg.2026.109765
- Fenofibrate microemulsion eyedrops for treating nitrogen mustard induced corneal injury.Journal of controlled release : official journal of the Controlled Release Society · 2026 · Kaffash E, Pangeni R, Liang W, et al.PMID 41580131DOI 10.1016/j.jconrel.2026.114656
- Tumor-Selective and Chemical Activation Strategies for Nitrogen Mustard Prodrugs.Chemistry (Weinheim an der Bergstrasse, Germany) · 2026 · Ponnamperumage TNF, Mahendran M, Awoyera WO, et al.PMID 41574478DOI 10.1002/chem.202503344
- Mechanisms, pathological features, and intervention strategies for nitrogen mustard-induced skin toxicity.Toxicology letters · 2026 · Zhu B, Mao G, Meng Q, et al.PMID 41490600DOI 10.1016/j.toxlet.2025.111815
- Phase 1 Dose Escalation of Single-Agent Mechlorethamine in Dogs With Lymphoma.Veterinary and comparative oncology · 2026 · Chadsey LE, Hess PR, Intile JL, et al.PMID 41198583DOI 10.1111/vco.70025
- Hecogenin‑nitrogen mustard hybrids with improved anti-breast cancer activity: Design, synthesis, and biological evaluation.Bioorganic chemistry · 2025 · Su W, Huang X, Ji X, et al.PMID 41056651DOI 10.1016/j.bioorg.2025.109032
- Zeb1 Facilitates Nitrogen Mustard-Induced Corneal Epithelial Wound Healing by Maintaining Epithelial Renewability.Investigative ophthalmology & visual science · 2025 · Wang F, Sun X, Dong Y, et al.PMID 40956023DOI 10.1167/iovs.66.12.33
Clinical trials
The 10 most recently updated of 4,977 ClinicalTrials.gov registrations naming Mechlorethamine as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.
- Eflornithine (DFMO) for Ewing Sarcoma and OsteosarcomaRecruiting · Phase 2 · Interventional · 406 enrolled · Milton S. Hershey Medical CenterNCT07321912updated 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 MalignanciesRecruiting · Phase 1 · Phase 2 · Interventional · 44 enrolled · M.D. Anderson Cancer CenterNCT06383572updated 2026-06-12
- Efficacy and Safety of the CloB2M (Clofarabine Combined With Busulfan and Melphalan) Conditioning Regimen in Allogeneic Hematopoietic Stem Cell Transplantation for Adult Patients With Acute Myeloid Leukemia in First Complete RemissionNot yet recruiting · Interventional · 30 enrolled · Institute of Hematology & Blood Diseases Hospital, ChinaNCT07644481updated 2026-06-12
- CD30 CAR for CD30+ NSGCTCompleted · Phase 2 · Interventional · 2 enrolled · UNC Lineberger Comprehensive Cancer CenterNCT05634785updated 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 LeukemiaRecruiting · 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 DiseaseNot yet recruiting · Phase 1 · Interventional · 40 enrolled · National Cancer Institute (NCI)NCT07509034updated 2026-06-12
- Dinutuximab With Chemotherapy, Surgery and Stem Cell Transplantation for the Treatment of Children With Newly Diagnosed High Risk NeuroblastomaRecruiting · Phase 3 · Interventional · 478 enrolled · National Cancer Institute (NCI)NCT06172296updated 2026-06-12
- Stem Cell Transplantation for Participants With Germline RUNX1 Associated Blood CancersNot 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 LeukemiaRecruiting · Phase 3 · Interventional · 130 enrolled · Memorial Sloan Kettering Cancer CenterNCT07223021updated 2026-06-12
- Study of CAR-T Cells Expressing CD30 and CCR4 for r/r CD30+ HL and CTCLActive not recruiting · Phase 1 · Interventional · 43 enrolled · UNC Lineberger Comprehensive Cancer CenterNCT03602157updated 2026-06-12
Frequently asked questions
- How does Mechlorethamine work?
- Mechlorethamine, also known as nitrogen mustard, is an alkylating agent which inhibits rapidly proliferating cells.
- What is Mechlorethamine used for?
- According to FDA labeling, Mechlorethamine carries indications including: VALCHLOR is indicated for the topical treatment of Stage IA and IB mycosis fungoides-type cutaneous T-cell lymphoma in patients who have received prior skin-directed therapy. VALCHLOR is an alkylating drug indicated for the topical treatment of Stage IA and IB mycosis fungoides-type cutaneous T-cell lymphoma in patients who have received prior skin-directed therapy ( 1 ).. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.
- What class of drug is Mechlorethamine?
- Mechlorethamine is classified as Nitrogen mustard analogues, Alkylating Drug, Alkylating Activity, Decreased DNA Integrity, Decreased DNA Replication.
- What are the brand names for Mechlorethamine?
- Mechlorethamine is marketed under brand names including Valchlor.
- What are the contraindications for Mechlorethamine?
- Mechlorethamine labeling lists contraindications including: The use of VALCHLOR is contraindicated in patients with known severe hypersensitivity to mechlorethamine. Hypersensitivity reactions, including anaphylaxis, have occurred with topical formulations of mechlorethamine.. Always consult the full prescribing information and a clinician.
mechlorethamine is illustrative MVP content compiled from public sources. pharmacopeia is for educational and informational use only and is not a substitute for professional medical advice.