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midostaurin

Rydapt · ATC code L01XE

Key facts
Active substance
midostaurin
Drug class
ALK inhibitor
ATC code
L01XE
Authorisation
centrally authorised across the EU (EMA)
Dosage forms
Rydapt 25 mg soft capsules
Marketing authorisation holder
Novartis Europharm Ltd
Information as of
2026-10-29

Key facts

Midostaurin is a kinase inhibitor that inhibits FLT3 and KIT, among others. It is used for FLT3-mutated acute myeloid leukaemia (AML) and for advanced forms of systemic mastocytosis.

What it is used for

  • Adults with newly diagnosed FLT3 mutation-positive AML in combination with standard daunorubicin and cytarabine induction and high-dose cytarabine consolidation, followed by single-agent maintenance in complete response.
  • Monotherapy in adults with aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated haematological neoplasm (SM-AHN) or mast cell leukaemia (MCL).

Dosing

  • Rydapt (25 mg soft capsules): twice daily at approximately 12-hour intervals with food; give prophylactic antiemetics.
  • AML: 50 mg twice daily on days 8-21 of induction and consolidation cycles, then for patients in complete response daily as maintenance for up to 12 cycles of 28 days each. Discontinue 48 hours before conditioning for stem cell transplant.
  • ASM, SM-AHN and MCL: starting dose 100 mg twice daily, as long as clinical benefit is observed.
  • Dose modifications for QTc prolongation, neutropenia, pulmonary infiltrates and other toxicities – see the full text.
  • No adjustment in mild or moderate renal or hepatic impairment; caution in severe renal impairment.

Contraindications

  • Hypersensitivity to the active substance or to any of the excipients.
  • Concomitant potent CYP3A4 inducers, e.g. rifampicin, St John's wort, carbamazepine, enzalutamide, phenytoin.

Important warnings

  • Neutropenia and infections: monitor white blood cell counts regularly, especially at initiation; active serious infections should be controlled before starting.
  • Cardiac dysfunction (congestive heart failure, LVEF decrease) in mastocytosis patients, including some fatalities; use with caution in patients at risk.
  • QTc prolongation: caution with risk factors; consider ECG monitoring with QT-prolonging medicines.
  • Interstitial lung disease and pneumonitis, some fatal; discontinue for severe non-infectious pulmonary symptoms.
  • Strong CYP3A4 inhibitors (e.g. ketoconazole, ritonavir, clarithromycin) can increase levels – caution.

Side effects

  • AML (with chemotherapy): most frequent febrile neutropenia (83.4%), nausea (83.4%), exfoliative dermatitis (61.6%), vomiting (60.7%), headache (45.9%), petechiae (35.8%) and pyrexia (34.5%).
  • ASM, SM-AHN and MCL: most frequent nausea (82%), vomiting (68%), diarrhoea (51%), peripheral oedema (35%) and fatigue (31%); hyperglycaemia frequent among laboratory abnormalities.

Interactions

  • Strong CYP3A4 inducers markedly reduce midostaurin exposure and are contraindicated.
  • Strong CYP3A4 inhibitors increase exposure; monitor closely for toxicity when combined.

Pregnancy and breastfeeding

  • May cause foetal harm: pregnancy test within 7 days before starting; effective contraception during and for at least 4 months after treatment; not recommended during pregnancy.
  • Breast-feeding should be discontinued during and for at least 4 months after treatment.

Summary of the official product information, written with AI on 2026-10-29. Every number was checked automatically against the original text. Not reviewed by a physician – the full official text is authoritative.

Full official text →

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For patients

Package leaflet, verbatim

This product has no separate US patient information.

Authorisation and brand names by country

Medicines with exactly this active-substance composition, as listed in the national registers – alphabetical and without ranking. Whether a product is currently available is not shown here.

EU – centrally authorised (all member states)

  • RydaptNovartis Europharm Ltd · EMEA/H/C/004095

Source: European Medicines Agency (EMA), product information

Brand names in 3 more European countries
Iceland
Rydapt
Liechtenstein
Rydapt
Norway
Rydapt

EMA – Article 57 product data (EU/EEA), 2026-10-29

Medelix receives no money from pharmaceutical companies. Brand names are listed alphabetically and without ranking.

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Sources

  1. European Medicines Agency. Rydapt – EPAR, Product Information. 2026-10-29. https://www.ema.europa.eu/en/documents/product-information/rydapt-epar-product-information_en.pdf
  2. Wikidata (CC0): ATC L01XE.

Editorial: Medelix editorial team · last updated 2026-10-29 · How these pages are made · Report an error

Source: European Medicines Agency (EMA), product information. Not advertising, not a treatment recommendation. For questions about your own treatment, ask your doctor or pharmacist.