Conditioning treatment prior to hematopoietic stem cell transplant (HSCT) for patients with multiple myeloma
Route of Administration
Mode of Action
Melphalan inhibits DNA replication and transcription causing cytotoxicity in dividing and non-dividing cells including multiple myeloma. Captisol-enabled™ melphalan is a new propylene glycol (PG)-free IV formulation of melphalan developed for patients with multiple myeloma undergoing HSCT. The formulation completely avoids the use of PG, which is used as a co-solvent in the current formulation of melphalan. The use of Captisol technology increases stability of reformulated melphalan potentially enabling clinicians to achieve a higher dose intensity of pre-transplant chemotherapy.
About Multiple Myeloma and Melphalan
Multiple myeloma is a cancer of plasma cells, a type of white blood cell present in the bone marrow that produces antibodies. In multiple myeloma, a group of plasma cells (myeloma cells) becomes cancerous and multiplies, raising the number of plasma cells to abnormally high levels, which can overcrowd bone marrow and interfere with the production of normal blood cells. Myeloma cells also produce abnormally high levels of proteins that may cause multiple organ failure.
There were an estimated 22,000 new cases of multiple myeloma in 2013 alone (per NCI and ACS), with the incidence of new cases continuing to increase by approximately 1.7% per year.
The current intravenous melphalan market is approximately $100 million annually, with predominant use in HSCT. The rate of autologous HSCT for patients with multiple myeloma is growing by approximately 3.3% annually.