Midostaurin, marketed as Rydapt, is an antineoplastic agent employed in the treatment of high-risk acute myeloid leukemia (AML) with specific mutations, aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematologic neoplasm (SM-AHN), or mast cell leukemia (MCL). Primarily, it serves as a multitarget kinase inhibitor, particularly for adult patients with newly diagnosed AML who possess the FLT3 genetic mutation. Approved in April 2017, Midostaurin has demonstrated its ability to improve overall survival rates when used as an adjunct therapy alongside chemotherapeutic agents in AML patients. Its mechanism of action involves targeting multiple receptor tyrosine kinases, including protein kinase C alpha (PKCalpha), VEGFR2, KIT, PDGFR, and WT and/or mutant FLT3 tyrosine kinases. By inhibiting these kinases, Midostaurin disrupts signaling cascades that lead to malignancies such as AML and ASM, ultimately inducing apoptosis in leukemia cells and mast cells. Moreover, Midostaurin has shown effectiveness as a combination therapy during chemotherapy, further enhancing its therapeutic value.

Midostaurin Pathway References