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avapritinib

Ayvakyt · ATC code L01EX18

Key facts
Active substance
avapritinib
ATC code
L01EX18
Authorisation
centrally authorised across the EU (EMA)
Dosage forms
AYVAKYT 25 mg film-coated tablets / AYVAKYT 50 mg film-coated tablets / AYVAKYT 100 mg film-coated tablets
Marketing authorisation holder
Blueprint Medicines (Netherlands) B.V.
Information as of
2026-10-28

Key facts

Avapritinib is a kinase inhibitor active against mutant KIT and PDGFRA kinases. It is used for certain gastrointestinal stromal tumours (GIST) and for systemic mastocytosis.

What it is used for

  • Monotherapy in adults with unresectable or metastatic GIST harbouring the PDGFRA D842V mutation.
  • Monotherapy in adults with advanced systemic mastocytosis (aggressive SM, SM with an associated haematological neoplasm, or mast cell leukaemia) after at least one systemic therapy.
  • Adults with indolent systemic mastocytosis (ISM) with moderate to severe symptoms inadequately controlled on symptomatic treatment.

Dosing

  • Ayvakyt, GIST: 300 mg once daily on an empty stomach; if a moderate CYP3A inhibitor cannot be avoided, reduce the starting dose to 100 mg once daily.
  • Ayvakyt, advanced systemic mastocytosis: 200 mg once daily (also the maximum dose); not recommended with platelets < 50 x 10⁹/L; with a moderate CYP3A inhibitor 50 mg once daily.
  • Ayvakyt, indolent systemic mastocytosis: 25 mg once daily (also the maximum dose); strong or moderate CYP3A inhibitors must be avoided.
  • For adverse reactions, interrupt and/or reduce the dose stepwise; permanently discontinue for intracranial haemorrhage – details in the full text.
  • Severe hepatic impairment (Child-Pugh C): reduced starting dose (GIST 200 mg, advanced SM 100 mg daily, ISM 25 mg every other day). Not recommended in severe renal impairment (not studied).

Contraindications

  • Hypersensitivity to the active substance or to any of the excipients.

Important warnings

  • Haemorrhages, including serious gastrointestinal and intracranial haemorrhage; monitor blood counts including platelets and coagulation, especially with anticoagulants.
  • Before starting, weigh the risk of intracranial haemorrhage (e.g. aneurysm, prior intracranial haemorrhage, stroke within the prior year, thrombocytopenia).
  • Cognitive effects such as memory impairment, confusion and encephalopathy can occur.
  • Fluid retention with oedema and pleural effusion; QT prolongation in GIST and advanced SM.
  • Elevations of bilirubin and transaminases; photosensitivity – avoid direct sunlight.

Side effects

  • GIST: most common nausea (45%), fatigue (40%), anaemia (39%), periorbital oedema (33%), face oedema (27%), hyperbilirubinaemia (28%), diarrhoea (26%), vomiting (24%) and memory impairment (20%).
  • Advanced systemic mastocytosis: most common periorbital oedema (38%), thrombocytopenia (37%), peripheral oedema (33%) and anaemia (22%).
  • Serious: intracranial haemorrhage, encephalopathy, pleural effusion.

Interactions

  • Strong or moderate CYP3A inhibitors increase avapritinib levels and should be avoided.
  • Strong or moderate CYP3A inducers (e.g. rifampicin, carbamazepine, phenytoin, St John's wort) decrease levels and should be avoided.

Pregnancy and breastfeeding

  • May cause foetal harm; verify pregnancy status before starting. Women must use effective contraception during treatment and for 6 weeks after the last dose, men with female partners of childbearing potential for 2 weeks after.

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)

  • AyvakytBlueprint Medicines (Netherlands) B.V. · EMEA/H/C/005208

Source: European Medicines Agency (EMA), product information

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

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

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Sources

  1. European Medicines Agency. Ayvakyt – EPAR, Product Information. 2026-10-28. https://www.ema.europa.eu/en/documents/product-information/ayvakyt-epar-product-information_en.pdf

Editorial: Medelix editorial team · last updated 2026-10-28 · 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.