Drug intelligence / Profile preview

midostaurin

Development stage
Approved
Lead developer
Novartis
Modality
Small Molecules
Administration
Oral
01

Overview

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].

Brand names
Rydapt
Other names
midostaurin
02

Targets

PRKCA (Protein kinase C alpha)KIT (c-KIT proto-oncogene receptor tyrosine kinase)FLT3 (Fms related receptor tyrosine kinase 3)PDGFRA (Platelet-derived growth factor receptor alpha)VEGFR2 (Vascular endothelial growth factor receptor 2)

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