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Midostaurin is an orally administered small molecule multikinase inhibitor used in the treatment of certain hematologic malignancies. It is primarily indicated for use in combination with standard cytarabine and daunorubicin induction and cytarabine consolidation chemotherapy for newly diagnosed acute myeloid leukemia (AML) that is FLT3 mutation-positive. Midostaurin is also approved for the treatment of aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematologic neoplasm (SM-AHN), and mast cell leukemia (MCL). The drug works by inhibiting multiple receptor tyrosine kinases, including FLT3, KIT, PDGFR, VEGFR2, and protein kinase C alpha. By blocking these kinases—especially mutant FLT3—it induces apoptosis in leukemic cells and inhibits proliferation of malignant mast cells. Midostaurin was developed by Novartis and was first approved in 2017[1][2][3][5][6][8].
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