What is the role of midostaurin in the treatment of acute myeloid leukemia (AML)?

Updated: May 26, 2020
  • Author: Karen Seiter, MD; Chief Editor: Emmanuel C Besa, MD  more...
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Midostaurin (Rydapt), an orally administered multitargeted kinase inhibitor, was approved by the US Food & Drug Administration (FDA) in April 2017 for adults with newly diagnosed AML that is FLT3 mutation positive. The FLT3 mutation is observed in approximately 30-35% of patients with AML.

Midostaurin is used in combination with standard cytarabine and daunorubicin induction and cytarabine consolidation chemotherapy. Midostaurin and its major active metabolites inhibit the activity of wild-type FLT3, FLT3 mutant kinases (ITD and TKD), KIT (wild type and D816V mutant), PDGFR-alpha/beta, VEGFR2, and members of the serine/threonine kinase protein kinase C (PKC) family.

Approval of midostaurin was based on the CALGB 10603 (RATIFY) study, conducted in 717 adults younger than 60 years with newly-diagnosed FLT3-mutated AML. Patients who received midostaurin plus standard induction and consolidation chemotherapy had significantly longer overall survival than patients who received standard treatment plus placebo (hazard ratio [HR] for death, 0.78; one-sided P=0.009), as well as longer event-free survival (HR for event or death, 0.78; one-sided P=0.002). [47]

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