Novel drug for treating acute AML with FLT3 mutation gets USFDA nod

The US Food and Drug Administration (FDA) has approved a new drug for the treatment of adult patients who have relapsed or refractory acute myeloid leukemia (AML) with a 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.

"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.

(With inputs from agencies.)