Drug · Small Molecule

Midostaurin

CI-DRUG-00000378Explore in graph →NCIt C1872 CHEMBL608533 CIViC Approved

Regulatory

Approvals (7)

Each record names the authority, jurisdiction, indication text and status. A drug approved in one jurisdiction for one indication is not 'approved' in general.

openfdaRegulatory5 approval records · authority, jurisdiction and indication text as published.

Jurisdiction · authority	Cancer	Indication	Status	Approval date	Source
US FDA	—	Efficacy supplement 2020-11-24 (see label)	approved	Nov 24, 2020	openfda
US FDA	Acute Myeloid Leukemia	Newly diagnosed acute myeloid leukemia (AML) that is FLT3 mutation-positive as detected by an FDA-approved test, in combination with standard cytarabine and daunorubicin induction and cytarabine consolidation.	approved	Apr 28, 2017	openfda
US FDA	—	Limitations of Use: RYDAPT is not indicated as a single-agent induction therapy for the treatment of patients with AML. Aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN), or mast cell leukemia (MCL).	approved	Apr 28, 2017	openfda
US FDA	Acute Myeloid Leukemia	Newly diagnosed acute myeloid leukemia (AML) that is FLT3 mutation-positive as detected by an FDA-approved test, in combination with standard cytarabine and daunorubicin induction and cytarabine consolidation.

Health Canada records are DIN-level: one row per marketed product (brand, strength, form). The Drug Product Database does not publish indications, so no cancer is stated for these rows, and a cancelled or dormant DIN is the status of that one product — not a withdrawal of the molecule.

Data updated 6 hours agoSource updated unknown

Derived

Development pipeline

Most advanced stage across all cancers, then per top-level cancer reached through trial conditions or approval indications. Approval in any ingested jurisdiction outranks trial phase; counts are interventional studies.

Scope	Stage	Max phase	Active	Recruiting	Phase 3	Trials	Approvals	Jurisdictions	First approval	First trial
All cancers	Approved	Phase 3	6	2	7	39	7	CA, EU, US	Apr 28, 2017	Jan 30, 2002
Leukemia	Approved	Phase 3	5	2	6	35	3	EU, US	Apr 28, 2017	Jan 30, 2002
Malignant Rectal Neoplasm	Phase1	Phase 1	0	0	0	1	0	—	—	2011-08

Computedclinicaltrials

Curated evidence

Clinical evidence (33)

CIViC items in which this therapy appears, grouped by cancer context, then molecular profile. 50 items per page.

civicCuratedShowing 1–33 of 33 evidence items · levels, directions and significance as curated at the source; each row links to its CIViC record.

Therapy	Cancer	Type	Level	Direction · significance	Rating (1–5)	Status	Evidence	Source
FLT3 D593del1
Crenolanib +

Clinical trials

Trials with this intervention (43)

Most recently updated first, 50 per page.

clinicaltrialsPublishedShowing 1–43 of 43 studies · status and phase as posted by the registrant; each row links to the study record.

NCT	Title	Status	Phase	Enrollment (n)	Sponsor	Countries	Last update	Source
NCT03836209	Gilteritinib vs Midostaurin in FLT3 Mutated Acute Myeloid Leukemia	completed	Phase 2	181	PrECOG, LLC.OTHER	1	Jul 23, 2026	clinicaltrials
NCT03591510	A Global Study of Midostaurin in Combination With Chemotherapy to Evaluate Safety, Efficacy and Pharmacokinetics in Newly Diagnosed Pediatric Patients With FLT3 Mutated AML	active not recruiting

Molecule

Record

Kind: Small Molecule
Mechanism: Tyrosine-protein kinase receptor FLT3 inhibitor; Stem cell growth factor receptor inhibitor; Platelet-derived growth factor receptor inhibitor; Protein kinase C (PKC) inhibitor; Vascular endothelial growth factor receptor 2 inhibitor
Targets: 16 genes
UNII: ID912S5VON
Aliases: CGP 41251, CGP41251, Midostaurin, Midostaurina, Midostaurine, N-Benzoylstaurosporine, N-Benzoyl-Staurosporine, NSC-656576, NVP-PKC412, PKC 412, PKC412, Rydapt, RYDAPT

Data updated 9 hours agoSource updated unknown

Identifiers

Codes (5)

Upstream identifiers kept as first-class codes; each links to its registry where a stable public URL exists.

System	Code	Label
ATC (WHO)	L01EX10	MIDOSTAURIN
ATC (WHO)	L01XE	Antineoplastic agents

FLT3 ITD3
Midostaurin	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	B	Supports Sensitivity Response	5	submitted	EID7061 In a study of 717 newly diagnosed acute myeloid leukemia patients harboring FLT3 mutations (555 FLT3-ITD, 162 FLT3-TKD), patients were randomized to receive placebo or midostaurin. Patients receiving … (full text at CIViC) PMID 28644114 · Stone et al., 2017 · Open in CIViC	civic
〃	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	B	Supports Sensitivity Response	4	submitted	EID8658 Patients in the Beat AML published dataset (n= 214 (193 primary; 21 relapse AML)) samples were studied with an ex vivo drug sensitivity screen to test whether concomitant genomic alterations alter mid… (full text at CIViC) PMID 32754299 · Rosenberg et al., 2020 · Open in CIViC	civic
AG1295 + AGS324 + Crenolanib + FLT3 Tyrosine Kinase Inhibitor TTT-3002 + KW2449 + Lestaurtinib + Linifanib + Midostaurin + Ponatinib + Quizartinib + R406 + Sorafenib + SunitinibSubstitutes	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	D	Supports Sensitivity Response	3	submitted	EID11084 A panel of FLT3 tyrosine kinase inhibitors was tested against Ba/F3 cells transfected with FLT3-ITD using MTT growth/survival assay. Each of the 13 TKIs inhibited proliferation of FLT3-ITD cells in a … (full text at CIViC) PMID 28077790 · Nguyen et al., 2017 · Open in CIViC	civic

FLT3 N841I2
Midostaurin	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	D	Supports Sensitivity Response	3	accepted	EID8858 Expression of the N841I variant in murine BaF3 cell line resulted in constitutive autophosphorylation of FLT3 and the amount of autophosphorylated FLT3 was decreased with exposure to the FLT3 inhibito… (full text at CIViC) PMID 15178581 · Jiang et al., 2004 · Open in CIViC	civic
〃	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	D	Supports Sensitivity Response	2	submitted	EID12512 Ba/F3 cells were manipulated to express FLT3-ITD or the FLT3 Tyrosine Kinase Domain point mutation N841I. These cells were treated with the FLT3 inhibitor, midostaurin, for 2 days and subsequently cel… (full text at CIViC) PMID 40196870 · Nakazawa et al., 2025 · Open in CIViC	civic

FLT3 Y572C2
Midostaurin	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	D	Supports Sensitivity Response	3	submitted	EID12787 Ba/F3 cell lines engineered to express Y572C showed reduced cell viability when exposed to different concentrations of midostaurin [IC50 (nm) 0.84] (no Figure available, data presented in supplemental… (full text at CIViC) PMID 39806020 · Anabtawi et al., 2025 · Open in CIViC	civic
〃	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	D	Supports Sensitivity Response	5	submitted	EID12793 Ba/F3 cell lines engineered to express Y572C showed reduced cell growth when exposed to different concentrations of PKC412 (also known as midostaurin )[IC50 (nm) 2.5] (Figure 4A) when compared to a Y5… (full text at CIViC) PMID 18068628 · Fröhling et al., 2007 · Open in CIViC	civic

FLT3 Y572del2
Midostaurin	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	D	Supports Sensitivity Response	4	submitted	EID12859 Ba/F3 cell lines expressing the variant FLT3 Y572del showed sensitivity to midostaurin and was 5 to 10-fold more sensitive than the FLT3-ITD construct. PMID 33914060 · Young et al., 2021 · Open in CIViC	civic
Crenolanib + Lestaurtinib + Midostaurin + Ponatinib + Quizartinib + SorafenibSubstitutes	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	D	Supports Sensitivity Response	5	submitted	EID9306 The construct supported IL-3 independent growth of Ba/F3 cells. It showed FLT-3 autophosphorylation and downstream signaling via STAT5, AKT, and ERK1/2 pathways. With IL-3 deprivation, the cells showe… (full text at CIViC) PMID 33914060 · Young et al., 2021 · Open in CIViC	civic

FLT3 Y842C2
Midostaurin	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	D	Supports Sensitivity Response	3	accepted	EID8340 A screen using saturation mutagenesis of FLT3/ITD followed by selection of transfected TF1 cells in FLT3 TKIs identified Y842C. Cytotoxicity assessed by MTT demonstrated Ba/F3 cells expressing FLT3/IT… (full text at CIViC) PMID 22858906 · Williams et al., 2013 · Open in CIViC	civic
〃	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	D	Supports Sensitivity Response	3	submitted	EID8934 A novel mutation (FLT3 Y842C) was detected in 2 AML patients (n = 110). Ex vivo PKC412 treatment of primary blasts resulted in suppression of constitutive FLT3 and STAT-5 activation (Figure 1D). Cell … (full text at CIViC) PMID 15345593 · Kindler et al., 2005 · Open in CIViC	civic