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Melphalan Injection
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 causing cancer cell death. Used for Multiple myeloma, Light chain myeloma, Waldenström macroglobulinemia.
At a glance
| Generic name | Melphalan Injection |
|---|---|
| Also known as | Intra-arterial chemotherapy, Chemoreduction |
| Sponsor | Hospital das Clínicas de Ribeirão Preto |
| 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 myeloma
- Waldenström macroglobulinemia
- Ovarian cancer
Common side effects
- Myelosuppression (neutropenia, thrombocytopenia, anemia)
- Nausea and vomiting
- Mucositis
- Secondary malignancy
- Infertility
Key clinical trials
- Dinutuximab With Chemotherapy, Surgery and Stem Cell Transplantation for the Treatment of Children With Newly Diagnosed High Risk Neuroblastoma (PHASE3)
- Ibrutinib Before and After Stem Cell Transplant in Treating Patients With Relapsed or Refractory Diffuse Large B-cell Lymphoma (PHASE3)
- Venetoclax or Placebo in Combination With Reduced-Intensity Conditioning Hematopoietic Cell (Bone Marrow/Blood Stem Cell) Transplant and as Maintenance Therapy After Transplant in Patients With Acute Myeloid Leukemia (A MyeloMATCH Treatment Trial) (PHASE2)
- MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial) (PHASE2)
- Melphalan, Prednisone, and Thalidomide or Lenalidomide in Treating Patients With Newly Diagnosed Multiple Myeloma (PHASE3)
- CD34+ Enriched Transplants to Treat Myelodysplastic Syndrome (PHASE2)
- Ruxolitinib Before, During and After Hematopoietic Cell Transplant in Older Patients With Myelofibrosis and Myelodysplastic Syndrome/Myeloproliferative Neoplasm Overlap Syndromes (PHASE2)
- A Study to Give Treatment Inside the Eye to Treat Retinoblastoma (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 Injection CI brief — competitive landscape report
- Melphalan Injection updates RSS · CI watch RSS
- Hospital das Clínicas de Ribeirão Preto portfolio CI