Melphalan
Pronunciation: (MEL-fa-lan).
- Generic name
- melphalan
- Brand names
- Alkeran, Evomela
- Drug class
- Alkylating agent (antineoplastic, chemotherapy)
Availability and supply rules vary by country
Trade names by country: United States · Canada
Australia: · Full directory
What melphalan is and how it works
Uses, formulations, monitoring, and supply rules can differ by patient, product, and country. Follow the exact local product information and professional advice.
Mechanism of action: how melphalan works
Melphalan is a bifunctional alkylating agent that forms covalent bonds with DNA, leading to cross-linking of DNA strands. This inhibits DNA and RNA synthesis, resulting in cell cycle arrest and apoptosis, particularly in rapidly dividing malignant cells. (1)
What melphalan is used for
Palliative treatment of multiple myeloma (oral tablets).
This is a palliative use, not a cure, and is given as part of a chemotherapy regimen managed by an oncology team. (2) (3) (4)
Palliative treatment of non-resectable epithelial carcinoma of the ovary (oral tablets).
Palliative treatment of non-resectable epithelial carcinoma of the ovary (oral tablets). (2) (3) (4)
High-dose conditioning treatment before autologous hematopoietic (stem) cell transplantation in patients with multiple myeloma (IV formulation, e.g., Evomela) — a different, transplant-specific use from the palliative oral indications above.
High-dose conditioning treatment before autologous hematopoietic (stem) cell transplantation in patients with multiple myeloma (IV formulation, e.g., Evomela) — a different, transplant-specific use from the palliative oral indications above. (2) (3) (4)
Before taking melphalan
Do not take / not appropriate for
- Contraindicated in patients with hypersensitivity to melphalan or other alkylating agents, and in those with severe bone marrow suppression. (Contraindication) (2)
- Pregnancy and breastfeeding are contraindications due to teratogenicity. (Contraindication) (2)
Tell your clinician about
(2) (3) (4)How to take melphalan
- General Instructions: Take melphalan exactly as prescribed by your doctor. Do not change your dose or schedule on your own — the oncology team decides all dosing, pausing, and stopping decisions based on your blood counts and response.. Adult Dosing: Regimens differ by indication and formulation: oral tablets for multiple myeloma commonly use 6 mg/day (adjusted by weekly blood counts) or 0.15-0.25 mg/kg/day for 4-7 days repeated every 4-6 weeks; oral tablets for ovarian cancer commonly use 0.2 mg/kg/day for 5 days, repeated roughly every 4-5 weeks; the older IV-for-injection formulation for multiple myeloma uses 16 mg/m2 by infusion every 2 weeks for 4 doses then every 4 weeks; the high-dose IV conditioning regimen before stem cell transplant (a distinct product, e.g. Evomela) uses 100 mg/m2/day for 2 days. These regimens are not interchangeable and must follow the specific product's label and the oncology team's protocol.. Pediatric Dosing: Use in children is less common; safety and effectiveness have not been formally established, and dosing must be individualized by a specialist based on body surface area and the specific cancer protocol.. Administration: Tablets should be swallowed whole with water, preferably on an empty stomach. Intravenous melphalan is administered by a healthcare professional.. When To Stop: Treatment may be paused or adjusted by your oncology team if you develop severe side effects or low blood counts; do not change or stop your regimen without their guidance.. Storage: Oral tablets are refrigerated at 2-8°C (36-46°F) and protected from light; injectable forms have separate storage instructions specific to the product used, provided by your healthcare provider.
- This page does not provide a dose calculator. Dosing must follow the prescriber, pharmacist, or product label.
Procedures and treatment changes
Tell the treating team before surgery, procedures, or any medicine change. Do not alter treatment without the responsible clinician. (2) (3) (4)
Missed dose
Contact your oncology team for guidance if you miss a dose of melphalan, since chemotherapy schedules are individualized and timing matters for both safety and effectiveness. Do not take an extra dose on your own to make up for a missed one.
Overdose
Overdose can cause severe bone marrow suppression, infection, or bleeding. Seek emergency medical help or contact a poison control center immediately if an overdose is suspected. (2) (3) (4)
Side effects
Side effects and their urgency vary; report severe, persistent, or concerning symptoms to a healthcare professional.
Nausea, vomiting, or diarrhea
Incidence: Common
Mouth sores or temporary hair loss
Incidence: Common
Low blood cell counts (white cells, platelets, or red cells) found on routine blood tests
Incidence: Very common — expected with this medicine and monitored by routine blood tests
Fever, chills, or other signs of infection
Incidence: Common; can be serious due to bone marrow suppression
Unusual bruising or bleeding
Incidence: Common; related to low platelet counts
Hives, swelling, wheezing, or difficulty breathing during or after an IV infusion (hypersensitivity/anaphylaxis)
Incidence: About 2% of IV recipients
New shortness of breath or persistent cough, which could reflect rare pulmonary fibrosis, or yellowing of the skin/eyes suggesting a liver problem
Incidence: Rare
Serious warnings
BOXED WARNING — serious
Symptoms: BOXED WARNING: melphalan must be given only under the supervision of a physician experienced in cancer chemotherapy. It can cause severe, dose-related bone marrow suppression (low white cells, platelets, and red cells) that can lead to serious infection or bleeding — regular blood-count monitoring is required. Melphalan is leukemogenic in humans, meaning it carries a risk of causing secondary leukemia, with the risk increasing with cumulative dose. The IV form additionally carries a boxed warning for hypersensitivity reactions, including anaphylaxis, in about 2% of recipients.
Action: Follow the urgency stated and seek prompt or emergency care when indicated. (2) (3) (4)
Melphalan is teratogenic (can cause birth defects) and mutagenic (can cause chromosomal changes) — serious
Symptoms: Melphalan is teratogenic (can cause birth defects) and mutagenic (can cause chromosomal changes); it is avoided during pregnancy, and reliable contraception is used during treatment. Can increase the risk of developing secondary cancers over time.
Action: Follow the urgency stated and seek prompt or emergency care when indicated. (2) (3) (4)
Contraindications on the current FDA label — serious
Symptoms: Contraindications on the current FDA label: known hypersensitivity to melphalan; documented prior resistance to melphalan. Precautions recorded in the clinician database: extra caution in people with renal or hepatic impairment or active infections.
Action: Follow the urgency stated and seek prompt or emergency care when indicated. (2) (3) (4)
Interactions
| Medicine or test | Effect | Action |
|---|---|---|
| Other bone-marrow-suppressing chemotherapy drugs or immunosuppressants | Increased risk of infection or additive toxicity when combined with other agents that suppress the bone marrow. Always inform the oncology team about all medicines and supplements being taken. | (1) |
| Live vaccines | Avoid live vaccines during melphalan treatment because of the weakened immune response. | (1) |
| Cyclosporine (IV melphalan) | Concurrent use has been associated with severe kidney impairment. | (3) |
| Carmustine (BCNU) (IV melphalan) | Concurrent use may lower the threshold for BCNU-related lung toxicity. | (3) |
Pregnancy, breastfeeding, kidney/liver function, age
Pregnancy
Melphalan causes fetal harm and is teratogenic; it is avoided during pregnancy, and effective contraception is recommended during treatment. Anyone who becomes pregnant during treatment should contact their oncology team right away. (2) (3) (4)
Breastfeeding
It is not known whether melphalan passes into human breast milk; because of the potential for serious harm to a nursing infant, melphalan should not be given to a breastfeeding parent, or breastfeeding should be discontinued during treatment — this is a decision made with the oncology team. (2) (3) (4)
Children
Safety and effectiveness of oral melphalan have not been formally established in pediatric patients. When melphalan is used in children (e.g., in specialized transplant-conditioning protocols), dosing is individualized by a pediatric oncology specialist based on body surface area and the specific protocol. (2) (3) (4)
Renal_impairment
Renal impairment: dosage reduction of up to 50% is considered for the IV-for-injection formulation in patients with renal insufficiency; melphalan clearance is measurably reduced as kidney function declines, so dosing and monitoring are individualized by the oncology team. Hepatic impairment: used with added caution; a specific dose-adjustment table was not found in the sources reviewed for this pass. (2) (3) (4)
Hepatic_impairment
Renal impairment: dosage reduction of up to 50% is considered for the IV-for-injection formulation in patients with renal insufficiency; melphalan clearance is measurably reduced as kidney function declines, so dosing and monitoring are individualized by the oncology team. Hepatic impairment: used with added caution; a specific dose-adjustment table was not found in the sources reviewed for this pass. (2) (3) (4)
Monitoring
(2) (3) (4)Storage
Oral tablets: refrigerate at 2-8 degC (36-46 degF), protected from light. IV formulations have separate, product-specific storage instructions (for example, one IV product is stored at room temperature, light-protected, in original packaging) — always follow the instructions for the specific product dispensed.
Professional information
Clinician-facing context only. This section does not replace current product labeling.
General (US label)
Indication: Multiple myeloma (oral) / ovarian cancer (oral) / transplant conditioning (IV, separate product)
Dose summary: Oral MM: 6 mg/day adjusted by weekly counts, or 0.15-0.25 mg/kg/day x4-7 days q4-6wk. Oral ovarian: 0.2 mg/kg/day x5 days, repeated ~q4-5wk. Older IV-for-injection (MM): 16 mg/m2 q2wk x4 doses then q4wk. High-dose IV conditioning before stem cell transplant (separate product, e.g. Evomela): 100 mg/m2/day x2 days.
Renal context: Dosage reduction of up to 50% is considered for the IV-for-injection formulation in renal insufficiency; melphalan clearance falls measurably as kidney function declines. Dosing is individualized by the oncology team. (1) (2) (3) (4)
Frequently asked questions
What is melphalan used for?
Melphalan is primarily used to treat multiple myeloma and ovarian cancer. It may also be used in certain conditioning regimens before stem cell transplantation.
How does melphalan work?
Melphalan works by damaging the DNA of cancer cells, preventing them from dividing and growing. This helps slow or stop the spread of cancer.
What are the most common side effects?
Common side effects include nausea, vomiting, diarrhea, mouth sores, and temporary hair loss. Some people may also experience low blood cell counts.
Can I take melphalan if I am pregnant?
No. Melphalan can cause birth defects and should not be used during pregnancy. Use effective contraception during treatment and inform your doctor if you become pregnant.
How is melphalan given?
Melphalan can be taken orally as tablets or given as an intravenous injection by a healthcare professional. The method depends on your specific treatment plan.
What should I do if I miss a dose?
Contact your oncology team for guidance if you miss a dose — chemotherapy schedules are individualized, and timing affects both safety and effectiveness. Do not take an extra dose on your own.
Are there foods or medicines I should avoid while taking melphalan?
Avoid live vaccines and contact with people who have infections. Always inform your doctor about all medicines and supplements you are taking.
What happens if I take too much Melphalan?
Taking too much Melphalan can lead to severe and life-threatening side effects, including profound myelosuppression, gastrointestinal toxicity, and neurological symptoms. If an overdose is suspected, it is crucial to seek immediate emergency medical attention. Prompt intervention is essential to manage symptoms and mitigate potential harm.
Can I drink alcohol while taking Melphalan?
It is generally advised to avoid or significantly limit alcohol consumption while undergoing treatment with Melphalan. As an alkylating agent, Melphalan can cause liver toxicity, and alcohol can exacerbate this risk. Additionally, alcohol may worsen common side effects like nausea and fatigue, potentially interfering with treatment efficacy and patient well-being.
Is Melphalan safe during pregnancy?
Melphalan is generally considered unsafe during pregnancy due to its potential to cause severe harm to the developing fetus. It falls under a pregnancy category that strongly advises against its use. If you are pregnant or planning to become pregnant, it is imperative to discuss this with your healthcare provider to explore alternative treatment options or manage risks appropriately.
What is the mechanism of action of melphalan?
Melphalan is a bifunctional alkylating agent that forms covalent bonds with DNA, leading to cross-linking of DNA strands. This inhibits DNA and RNA synthesis, resulting in cell cycle arrest and apoptosis, particularly in rapidly dividing malignant cells. (1)
What is the generic name of Melphalan?
The generic name is melphalan. It is sold under brand names including Alkeran, Evomela.
What class of drug is melphalan?
Melphalan is classed as: Alkylating agent (antineoplastic, chemotherapy).
References
- Melphalan — clinician-authored medicine recordMedGuideGlobal · Global (internal database) · accessed 2026-08-06 · source 1
- ALKERAN (melphalan) tablets — prescribing informationU.S. Food and Drug Administration · United States · accessed 2026-08-06 · source 2
- EVOMELA (melphalan) injection — prescribing informationU.S. Food and Drug Administration · United States · accessed 2026-08-06 · source 3
- ALKERAN (melphalan hydrochloride) for Injection — prescribing information (2011)U.S. Food and Drug Administration · United States · accessed 2026-08-06 · source 4
- MELPHALAN HYDROCHLORIDE (MELPHALAN HYDROCHLORIDE) KIT [HERITAGE PHARMACEUTICALS INC. D/B/A AVET PHARMACEUTICALS INC.] — US prescribing information (DailyMed SPL)U.S. National Library of Medicine (DailyMed) · US · accessed 2026-08-14 · source 5
- Melphalan Hydrochloride — FDA approval record (Drugs@FDA ANDA211463)U.S. Food and Drug Administration · US · accessed 2026-08-14 · source 6
- melphalan — RxNorm normalized drug concept (RXCUI 235857)U.S. National Library of Medicine (RxNorm) · US · accessed 2026-08-14 · source 7
Brand names by country
Single-ingredient melphalan names are shown separately by country. Product availability, strengths, and supply rules can change; combination products are not included here.
Priority countries
United States
- IVRA90 mg/mL · apotex corp
- EVOMELA50 mg0mL · acrotech biopharma inc
Canada
- ALKERAN