Midostaurin

FLT3 inhibitors
FLT3 inhibitors
High alert
An error with this medicine can cause serious harm: double checks and protocols are used.
Pregnancy: avoid
Avoid in pregnancy. Midostaurin may harm the fetus: a pregnancy test is done first, women and men use contraception during and for months after treatment, and breastfeeding is…

An oral multikinase inhibitor of FLT3 and KIT, added to chemotherapy in FLT3-mutated AML and used in advanced systemic mastocytosis.

Updated Nov 12, 2026

Also called: PKC412

Quick facts

Group: FLT3 inhibitors
Target: FLT3 receptor tyrosine kinase (also KIT and other kinases)
Used for: Aggressive systemic mastocytosis
Key effects: Nausea and vomiting · Diarrhoea · Headache
Monitor: FLT3 mutation test before starting
ATC: L01EX10

No doses: the clinician sets them by the current guideline.

Mechanism of action

FLT3 receptor tyrosine kinase (also KIT and other kinases)Midostaurin

No structure with midostaurin bound to FLT3 was found. The figure shows gilteritinib, another FLT3 inhibitor, in the ATP site of human FLT3.

Target in 3D: FLT3 receptor tyrosine kinase (also KIT and other kinases). PDB structure 6JQR (RCSB PDB, public domain). View the structure at RCSB PDB

Midostaurin is a multikinase inhibitor active against mutant FLT3, wild-type KIT and the KIT D816V mutant of mastocytosis, PDGFR, SRC and protein kinase C. In AML it adds to chemotherapy by cutting off FLT3 growth signals; in mastocytosis it stops KIT-driven mast cell growth and mediator release.

Midostaurin blocks mutant FLT3, KIT and other kinases
Mutant FLT3 and KIT D816V stop signalling
Leukaemic blasts and mast cells stop growing

Indications

Newly diagnosed FLT3-mutated acute myeloid leukaemia, with cytarabine and daunorubicin induction and cytarabine consolidation. Acute leukaemia (AML and ALL) →
Aggressive systemic mastocytosis, systemic mastocytosis with an associated haematological neoplasm, and mast cell leukaemia. Mastocytosis (cutaneous and systemic) →

Adverse effects

Common

Nausea and vomiting
Diarrhoea, abdominal pain, mouth sores
Headache, nosebleeds, sweating
Limb swelling, musculoskeletal pain
Fatigue, insomnia

Contraindications

Hypersensitivity to midostaurin

Key interactions

St John's wort: Can lower midostaurin levels; avoided.
Grapefruit and its juice: Can raise midostaurin levels; discussed with the treating team.
Cytarabine and daunorubicin (given together): The approved AML combination; low blood counts are expected and watched.

What is monitored

FLT3 mutation test before starting (AML)
Complete blood count
New cough or breathlessness
ECG in patients at risk of QT prolongation

Administration and safety

Oral capsules with food, swallowed whole, not opened or crushed.
An antiemetic is usually given before each dose.
If vomiting follows a dose, no extra dose is taken.

Pregnancy and breastfeeding

Avoid in pregnancy. Midostaurin may harm the fetus: a pregnancy test is done first, women and men use contraception during and for months after treatment, and breastfeeding is stopped.

Nursing care

Give antiemetics before each dose and monitor intake.
Teach to report fever, cough, wheeze or breathlessness.
Monitor blood counts and teach infection precautions.
Pregnancy test before starting; contraception during and for months after treatment.

Pharmacokinetics

Absorption: Oral capsules twice a day with food.

Test yourself

Think of the answer, then tap the question to see it.

Which two kinases explain midostaurin's two indications?

Mutant FLT3 (AML) and KIT D816V (systemic mastocytosis).

With which chemotherapy is midostaurin combined in AML?

Cytarabine and daunorubicin induction, then cytarabine consolidation.

Why is an antiemetic given before each dose?

Nausea and vomiting are very common.

Add to my cards (3)

Connections

← Back to the class: FLT3 inhibitors

Educational content on how drugs work; not a prescribing guide and no substitute for a health professional's advice. No doses are given.

Sources: Midostaurin: MedlinePlus Drug Information (MedlinePlus (U.S. National Library of Medicine), 2017); Midostaurin - NCI (National Cancer Institute, 2022); ATCDDD - ATC/DDD Index (WHO Collaborating Centre for Drug Statistics Methodology, 2026).

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Representative model, for educational purposes only.