Currently, IV busulfan is the gold standard for use as a pre-treatment for stem cell transplantation in AML patients. According to the Center for International Blood and Marrow Transplant Research (CIBMTR), IV busulfan is used in 74% of the patients. Despite the high usage of IV busulfan in this patient population, there are no busulfan formulations approved by regulatory authorities to treat this deadly disease. In 2020, there were approximately 3,200 allogeneic AML transplants in the USA. The drug cost is a minor component of the transplantation as the total costs for the procedure is approximately $250,000 in the United States.
It has been demonstrated that some of the tyrosine kinase inhibitors (TKI) are effective for CML patients but due to the heterogenous nature of AML, the TKIs are ineffective for AML.