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Melphalan (Mel)
Melphalan is an alkylating agent that cross-links DNA strands, preventing cell division and causing cancer cell death.
Melphalan is an alkylating agent that cross-links DNA strands, preventing cell division and inducing apoptosis in rapidly dividing cancer cells. Used for Multiple myeloma, Light chain amyloidosis, Waldenström macroglobulinemia.
At a glance
| Generic name | Melphalan (Mel) |
|---|---|
| Also known as | L-PAM |
| Sponsor | Institute of Hematology & Blood Diseases Hospital, China |
| Drug class | Alkylating agent |
| Target | DNA |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | FDA-approved |
Mechanism of action
Melphalan belongs to the nitrogen mustard class of alkylating agents. It forms covalent bonds with DNA bases, creating interstrand and intrastrand cross-links that interfere with DNA replication and transcription. This leads to cell cycle arrest and apoptosis, particularly affecting rapidly dividing cancer cells.
Approved indications
- Multiple myeloma
- Light chain amyloidosis
- Waldenström macroglobulinemia
Common side effects
- Myelosuppression (neutropenia, thrombocytopenia, anemia)
- Nausea and vomiting
- Mucositis
- Secondary malignancy
- Infertility
- Diarrhea
Key clinical trials
- Fludarabine Plus Melphalan Versus Addition of Venetoclax to Fludarabine/Melphalan Conditioning Regimen for Allogeneic Hematopoietic Stem Cell Transplantation in AML/MDS Patients Aged > 50 Years: a Multicenter, Randomized, Phase 3 Trial (PHASE3)
- A Platform Protocol to Investigate Post-Transplant Cyclophosphamide-Based Graft-Versus-Host Disease Prophylaxis in Patients With Hematologic Malignancies Undergoing Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation (PHASE2)
- Reduced Intensity Haploidentical BMT for High Risk Solid Tumors (PHASE2)
- MMulti-Immune HR; Multi-Target Immunotherapy for High-Risk Multiple Myeloma (PHASE2)
- Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders (PHASE2)
- Propylene Glycol-Free Melphalan HCl (EVOMELA®) in Combination With Fludarabine and Total Body Irradiation Based Reduced Intensity Conditioning for Haploidentical Transplantation (PHASE2)
- Induction Therapy Including 131 I-MIBG and Chemotherapy in Treating Patients With Newly Diagnosed High-Risk Neuroblastoma Undergoing Stem Cell Transplant, Radiation Therapy, and Maintenance Therapy With Isotretinoin (PHASE1)
- Trial Evaluating MGTA-456 in Patients With High-Risk Malignancy (PHASE2)
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:
- Melphalan (Mel) CI brief — competitive landscape report
- Melphalan (Mel) updates RSS · CI watch RSS
- Institute of Hematology & Blood Diseases Hospital, China portfolio CI