Midostaurin: First Global Approval
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Updated: Feb 28, 2026

Author Spotlight: Developing Multiplexed Kinetic Assays for Organoid-Based Drug Response Analysis
Published on: January 5, 2024
1Springer, Private Bag 65901, Mairangi Bay, 0754, Auckland, New Zealand. dru@adis.com.
Abstract:
Midostaurin (Rydapt®) is a multikinase inhibitor being developed by Novartis Pharmaceuticals. In April 2017, midostaurin was approved in the USA for the treatment of adult patients with newly diagnosed, FMS-like tyrosine kinase 3 (FLT3) mutation-positive acute myeloid leukaemia (AML) [in combination with standard cytarabine and daunorubicin induction, and cytarabine consolidation], or aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated haematological neoplasm (SM-AHN) or mast cell leukaemia (MCL) [collectively known as advanced SM]. The article summarizes the milestones in the development of midostaurin leading to this first global approval.
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