Melphalan (melphalan)
Multiple manufacturers (generic); original brand: Alkeran by Aspen Pharmacare · Alkylating Agent / Nitrogen Mustard
What is Melphalan? Melphalan (brand: Alkeran) is a nitrogen mustard alkylating agent used to treat multiple myeloma and ovarian carcinoma. It is also used as a high-dose conditioning regimen before autologous stem cell transplantation (ASCT) in myeloma.
About Melphalan
| Brand Name | Melphalan |
| Generic / INN | melphalan |
| Drug Class | Alkylating Agent / Nitrogen Mustard |
| Manufacturer | Multiple manufacturers (generic); original brand: Alkeran by Aspen Pharmacare |
| Administration | Oral tablet or Intravenous (IV) infusion |
| FDA Approval | 1964 |
FDA-Approved Uses for Melphalan
Indications are based on FDA approval. Your healthcare provider determines appropriate use based on your specific diagnosis, stage, biomarker status, and overall clinical picture.
- Multiple myeloma — palliative treatment
- Non-resectable epithelial carcinoma of the ovary — palliative treatment
- High-dose melphalan — conditioning regimen before autologous stem cell transplantation (ASCT) in multiple myeloma (IV formulation: Evomela)
How Does Melphalan Work?
Melphalan is a bifunctional alkylating agent that forms cross-links within and between DNA strands, interfering with DNA replication and transcription. It is cell cycle non-specific and has particular activity in hematopoietic tumor cells.
Dosage & Administration
Oral (multiple myeloma): 6 mg/day for 2–3 weeks, then rest, repeated as response and blood counts allow. IV high-dose (ASCT conditioning): Evomela or generic formulation at doses of 140–200 mg/m² per the transplant protocol. Renal impairment dose adjustment required.
Dosing is individualized by the prescribing clinician. Never adjust your dose without consulting your healthcare provider. This information is educational only and does not replace the official prescribing information.
Warnings, Precautions & Side Effects
General Warning
SEVERE myelosuppression — dose-limiting and potentially fatal. Secondary malignancies (acute myeloid leukemia, myelodysplastic syndrome) — increased risk with prolonged use. Pulmonary fibrosis. Hypersensitivity reactions (IV formulation). Embryo-fetal toxicity. Impaired fertility.
- Severe myelosuppression
- Secondary malignancies (AML/MDS)
- Pulmonary fibrosis
- Severe hypersensitivity (IV)
- Embryo-fetal toxicity
Common Side Effects
- Nausea
- Vomiting
- Myelosuppression
- Mucositis (high-dose)
- Alopecia
- Diarrhea
This is not a complete list. Always report new or worsening symptoms to your healthcare provider promptly.
Storage & Handling
Oral tablets: Store at 15°C to 30°C (59°F to 86°F) in a dry place. IV (Evomela): reconstituted solution refrigerate; use within 1 hour at room temperature.
BiologicHealthPlus provides temperature-monitored cold-chain specialty delivery for medications requiring refrigeration. Never use a medication if its storage conditions have been compromised.
Insurance Coverage, Prior Authorization & Patient Assistance
🛡️ Commercial Insurance
Melphalan is covered by most major commercial insurance plans when medically necessary and prior authorized. Coverage tier and cost-sharing vary by plan.
✅ Prior Authorization
Prior authorization is required by virtually all insurance plans for Melphalan. BiologicHealthPlus handles the complete prior authorization process at no cost to patients or providers.
🏥 Medicare Coverage
Medicare Part B covers this IV medication when administered in a clinical setting. Medicaid coverage varies by state.
🆓 Patient Assistance
Manufacturer patient assistance programs may be available for eligible uninsured or underinsured patients. Contact BiologicHealthPlus for a confidential eligibility review.
Prior Authorization Patient Assistance Insurance & Savings Medication Costs
Frequently Asked Questions — Melphalan
Melphalan is used to treat multiple myeloma and ovarian carcinoma, and at high doses as a conditioning regimen before autologous stem cell transplantation (ASCT) in myeloma.
Oral melphalan (2 mg tablets) is used for standard-dose palliative treatment of myeloma. IV melphalan at high doses (Evomela) is used as conditioning before ASCT. The two formulations are not interchangeable.
Secondary malignancies — particularly acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) — are a significant long-term risk with cumulative melphalan exposure.
Related Medications & Conditions
Related Medications
Revlimid (lenalidomide) · Velcade (bortezomib) · Darzalex (daratumumab) · Abecma · Carvykti
Related Conditions
Multiple myeloma · Ovarian carcinoma · Autologous stem cell transplant conditioning