Melphalan

Melphalan is an antitumoral agent belonging to the class of nitrogen mustard alkylating agents. Despite being one of the older molecules in the oncology arsenal, it continues to hold a significant role in clinical practice, particularly in the treatment of multiple myeloma.

Melphalan exerts its therapeutic action by interfering with DNA and RNA transcription. It alkylates the guanine base of DNA, ultimately inhibiting DNA replication and leading to programmed cell death.
Because of its mechanism of action and associated risks, Melphalan is classified as a high-potency active pharmaceutical ingredient (HP-API), being mutagenic, carcinogenic, and toxic to reproduction. As such, its handling and manufacturing require strict containment and safety protocols.

Melphalan has been approved for the treatment of a wide range of malignancies including multiple myeloma, malignant lymphomas, lymphoblastic and myeloblastic leukemias, childhood neuroblastoma, ovarian cancer, mammary adenocarcinoma, and uveal melanoma. First approved in Canada in 1963, it has since gained regulatory approval in over 100 countries and is listed in the World Health Organization’s List of Essential Medicines. Today, it is available as a generic drug.

Although newer therapies and targeted treatments have emerged over the decades, Melphalan remains a cornerstone in certain oncological protocols. In particular, it continues to be a reference treatment in the management of multiple myeloma, where its efficacy, especially in conditioning regimens for autologous stem cell transplantation, is well established and clinically validated.