Midostaurin

DB06595ApprovedInvestigationalSmall moleculeKinase Inhibitor

Midostaurin is an antineoplastic agent used to treat 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). Midostaurin (as Rydapt) is a multitarget kinase inhibitor for the treatment for adult patients with newly diagnosed acute myeloid leukemia (AML) who have a specific genetic mutation called FLT3.

Mechanism: It potently inhibits multiple receptor tyrosine kinases. Midostaurin and its major active metabolites CGP62221 and CGP52421 inhibit the activity of protein kinase C alpha (PKCalpha), VEGFR2, KIT, PDGFR and WT and/or mutant FLT3 tyrosine kinases. Inhibition of FLT3 receptor signalling cascades induces apoptosis of target leukemia cells expressing target receptors and mast cells, in addition to its antiproliferative activity toward multiple cancer cell lines. Midostaurin also interacts with organic anion transporter (OATP) 1A1 and multidrug resistance protein (MRP)-2 according to preliminary in vitro studies.
Curator reviewed · 1 reference
Primary indication: Investigated for use/treatment in adult patients with high-risk acute myeloid leukemia (AML) who are FLT3 mutation-positive, agressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN), or mast cell leukemia (MCL).Curator reviewed · 4 structured indications
Formula / weight: C₃₅H₃₀N₄O₄ · 570.649 g/mol (avg)
First approval: Canada, 2017 · United States, 2017 · European Union, 2020
Also known as: 4'-N-benzoylstaurosporine
Code names: CGP-41251 · NVP-PKC412 · PKC-412
Brand names: Rydapt

Resolves to

Compound

UNIIID912S5VONCAS120685-11-2ChEMBLCHEMBL608533ChEBI63452PubChem9829523KEGGD05029

Clinical / RWD

RxNorm1919083ATCL01EX10

Structure

InChIKeyBMGQWWVMWDBQGC-IIFHNQTCSA-NSMILESCO[C@@H]1[C@@H](C[C@H]2O[C@]1(C)N1C3=C(C=CC=C3)C3=C1C1=C(C4=C(C=CC=C4)N21)C1=C3CNC1=O)N(C)C(=O)C1=CC=CC=C1

Targets

P17252PRKCAP35968KDRP16234PDGFRAP09619PDGFRBP36888FLT3P10721KITP05129PRKCG

What you can answer from here — as of June 15, 2026

8Protein targetsEach mapped to UniProt, with action and pharmacological action

Listed above

1,049Drug interactionsStructured to mechanism, not free text

44Clinical trialsPhase, status and sponsor resolved per trial

4Structured indicationsCondition, population, route and combination as fields, not prose

12Marketed productsAcross 9 countries and 7 labellers

1ATC codeIncluding every combination product, plus 57 drug categories

45+ReferencesStructured and connected to the statements they support

Which drugs share a target with Midostaurin, and which of those have an active Phase 3 trial?

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