Xospata, the new drug, has been approved recently by the US Food and Drug Administration (FDA) for treating adults with relapsed or refractory acute myeloid leukemia (AML) having the FLT3 mutation.
AML is a rapidly progressing cancer that crowds out normal cells in the bone marrow and bloodstream, resulting in low numbers of normal blood cells and a continuous need for transfusions, reports Xinhua news agency.
‘Patients who are suffering from AML could benefit from the new drug Xospata which could reduce the need for frequent platelet transfusions’
"Approximately 25 to 30 per cent of patients with AML have a mutation in the FLT3 gene. These mutations are associated with a particularly aggressive form of the disease and a higher risk of relapse," Richard Pazdur, acting director of the Office of Haematology and Oncology Products in the FDA's Centre for Drug Evaluation and Research, said on Wednesday.
"Xospata targets this gene and is the first drug to be approved that can be used alone in treating patients with AML having a FLT3 mutation who have relapsed or who don't respond to initial treatment."
The efficiency of Xospata was studied in a clinical trial of 138 patients with relapsed or refractory AML having a confirmed FLT3 mutation.
Twenty-one per cent of patients achieved complete remission or complete remission with partial haematologic recovery with treatment, according to FDA.
Of the 106 patients who required red blood cell or platelet transfusions at the start of treatment with Xospata, 31 per cent became transfusion-free for at least 56 days, according to the FDA.