Midostaurin

Kinase Inhibitor — ANTINEOPLASTIC AND IMMUNOMODULATING AGENTS

Key facts

Midostaurin is an ofp drug in the Kinase Inhibitor class catalogued in DrugCentral. It was first approved by the FDA in 2017. Its mechanism of action is classified as Receptor Tyrosine Kinase Inhibitors. DrugCentral lists 214 molecular targets, including KIT, EGFR and PKN1. Recorded indications include Acute myeloid leukemia, disease, Aggressive systemic mastocytosis and Mast cell leukemia.

• Approval status: ofp (first approved 2017 by the FDA).
• Drug class: Kinase Inhibitor.
• Mechanism of action: Receptor Tyrosine Kinase Inhibitors.
• Molecular targets: KIT, EGFR, PKN1 and TBK1 (214 listed in DrugCentral).
• Indications: Acute myeloid leukemia, disease, Aggressive systemic mastocytosis and Mast cell leukemia.
• ATC code: L01EX10 (Other protein kinase inhibitors).
• Molecular formula: C35H30N4O4; molecular weight 570.65 g/mol.

Indications & contraindications

Drug–disease relationships curated by DrugCentral (OMOP / SNOMED concepts).

Indications (3)

• Acute myeloid leukemia, disease
• Aggressive systemic mastocytosis
• Mast cell leukemia

Mechanism & targets

• Mechanism of action (MoA): Receptor Tyrosine Kinase Inhibitors
• Established pharmacologic class: Kinase Inhibitor

Molecular targets (214)

Target	Gene	Action	Activity
Mast/stem cell growth factor receptor Kit	KIT	unknown	Kd 8.11
Epidermal growth factor receptor	EGFR	unknown	Kd 8.06
Serine/threonine-protein kinase N1	PKN1	unknown	Kd 8.03
Serine/threonine-protein kinase TBK1	TBK1	unknown	Kd 8.03
Receptor-type tyrosine-protein kinase FLT3	FLT3	inhibitor	Kd 7.96
Tyrosine-protein kinase JAK3	JAK3	unknown	Kd 7.92
Serine/threonine-protein kinase N2	PKN2	unknown	Kd 7.82
Mitogen-activated protein kinase kinase kinase 19	MAP3K19	unknown	Kd 7.82
Mitogen-activated protein kinase kinase kinase 9	MAP3K9	unknown	Kd 7.82
Mitogen-activated protein kinase kinase kinase 11	MAP3K11	unknown	Kd 7.77
Serine/threonine-protein kinase 4	STK4	unknown	Kd 7.77
Proto-oncogene tyrosine-protein kinase receptor Ret	RET	unknown	Kd 7.70
Calcium/calmodulin-dependent protein kinase type II subunit alpha	CAMK2A	unknown	Kd 7.70
MAP/microtubule affinity-regulating kinase 3	MARK3	unknown	Kd 7.68
Calcium/calmodulin-dependent protein kinase type II subunit delta	CAMK2D	unknown	Kd 7.44
Eukaryotic translation initiation factor 2-alpha kinase 4	EIF2AK4	unknown	Kd 7.41
Serine/threonine-protein kinase 3	STK3	unknown	Kd 7.40
NUAK family SNF1-like kinase 1	NUAK1	unknown	Kd 7.39
SRSF protein kinase 1	SRPK1	unknown	Kd 7.38
G protein-coupled receptor kinase 7	GRK7	unknown	Kd 7.32

Regulatory approvals (2)

Date	Agency	Marketed as	Applicant	Orphan
2017-09-18	EMA	—	Novartis Europharm Ltd	Yes
2017-04-28	FDA	—	NOVARTIS PHARMS CORP

ATC classification

ATC code	Therapeutic hierarchy
L01EX10	ANTINEOPLASTIC AND IMMUNOMODULATING AGENTS › ANTINEOPLASTIC AGENTS › PROTEIN KINASE INHIBITORS › Other protein kinase inhibitors

Pharmacogenomics

Drug-label and guideline annotations from PharmGKB and CPIC.

• EMA label: FLT3 · Testing Required
• FDA label: KIT, NPM1
• FDA label: FLT3 · Testing Required
• HCSC label: FLT3 · Testing Required

Reported adverse-event signals

Strongest FDA FAERS disproportionality signals (log-likelihood ratio). A signal is a statistical association in spontaneous reports, not proof that the drug causes the event.

MedDRA term	LLR
Acute myeloid leukaemia recurrent	761.3
Febrile neutropenia	392.0
Pancytopenia	239.9
Bone marrow failure	232.1
Skin toxicity	183.3
Neutropenic colitis	170.5
Thrombocytopenia	150.0
Death	144.2
Graft versus host disease	121.1
Neutropenia	120.4
Lymphadenopathy	107.9
Cytomegalovirus infection reactivation	102.0

Chemical structure

• Molecular formula: C₃₅H₃₀N₄O₄
• Molecular weight: 570.65 g/mol
• CAS number: —
• InChIKey: BMGQWWVMWDBQGC-IIFHNQTCSA-N
• PubChem CID: 9829523

CO[C@@H]1[C@@H](C[C@H]2O[C@]1(C)N1C3=CC=CC=C3C3=C4CNC(=O)C4=C4C5=CC=CC=C5N2C4=C13)N(C)C(=O)C1=CC=CC=C1

Frequently asked questions

Answers are generated from the structured data on this page, not written by hand.

What is Midostaurin used for?

DrugCentral lists the following indications for Midostaurin: Acute myeloid leukemia, disease, Aggressive systemic mastocytosis and Mast cell leukemia.

How does Midostaurin work?

Midostaurin is classified as Receptor Tyrosine Kinase Inhibitors; its molecular targets in DrugCentral include KIT, EGFR, PKN1 and TBK1.

When was Midostaurin first approved?

Midostaurin was first approved by the FDA in 2017.

What drug class does Midostaurin belong to?

Midostaurin is in the Kinase Inhibitor class.

What is the chemical formula of Midostaurin?

Midostaurin has the molecular formula C35H30N4O4 and a molecular weight of 570.65 g/mol.

Sources

• DrugCentral drug card 5231 — structure, indications, targets, approvals, pharmacologic classes (CC BY-SA 4.0)
• ChEMBL CHEMBL608533 — compound record and bioactivity
• PubChem CID 9829523 — chemical identifiers
• DrugBank DB06595 — drug record
• FDA UNII ID912S5VON — substance identity
• DailyMed: FDA labels for Midostaurin — current prescribing information (label search)
• UniProt P10721 (KIT) — target protein
• UniProt P00533 (EGFR) — target protein
• UniProt Q16512 (PKN1) — target protein
• UniProt Q9UHD2 (TBK1) — target protein
• UniProt P36888 (FLT3) — target protein
• UniProt P52333 (JAK3) — target protein
• FDA Adverse Event Reporting System (openFDA FAERS) — adverse-event signals, as aggregated by DrugCentral
• PharmGKB / CPIC — pharmacogenomic label annotations

How this page is built: methodology · editorial policy & corrections.