Prof. Erhabor Osaro / Wikimedia Commons
The US Food and Drug Administration (FDA) has approved midostaurin (Rydapt; Novartis) for use in combination with chemotherapy by newly diagnosed acute myeloid leukaemia (AML) patients who have the specific gene mutation, FLT3.
Midostaurin is a kinase inhibitor that works by blocking several enzymes that promote cell growth; it is the first targeted therapy to be made available in the United States to treat newly diagnosed patients with AML in combination with chemotherapy.
It will be offered only in cases where the FLT3 mutation has been confirmed by a test carried out by a LeukoStrat CDx FLT3 Mutation Assay device which was also given FDA approval last week (28 April 2017).
The FDA’s decision to approve the drug followed the results of a randomised trial involving 717 patients who had not been treated previously for AML. Patients who received the drug in combination with chemotherapy lived longer than patients who received chemotherapy alone. Patients who received the drug in combination with chemotherapy went just over eight months longer without experiencing certain complications compared to those given chemotherapy alone.
Common side effects of the drug include low levels of white blood cells with fever, nausea, inflammation of the mucous membranes, vomiting, headache, nosebleeds and upper respiratory tract infection.
The FDA also approved midostaurin as a treatment for adults with certain types of rare blood disorders, such as aggressive systemic mastocytosis; systemic mastocytosis with associated haematological neoplasm; or mast cell leukaemia.
