Ayvakyt
What Ayvakyt is used for
Unresectable or metastatic gastrointestinal stromal tumour (GIST) AYVAKYT is indicated as monotherapy for the treatment of adult patients with unresectable or metastatic gastrointestinal stromal tumours (GIST) harbouring the platelet-derived growth factor receptor alpha (PDGFRA) D842V mutation. Advanced systemic mastocytosis (AdvSM) AYVAKYT is indicated as monotherapy for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated haematological neoplasm (SM-AHN) or mast cell leukaemia (MCL), after at least one systemic therapy. Indolent systemic mastocytosis (ISM) AYVAKYT is indicated for the treatment of adult patients with indolent systemic mastocytosis (ISM) with moderate to severe symptoms inadequately controlled on symptomatic treatment (see section 5.1).
Approved use, as published by the European Medicines Agency.
What the labels mean
- Orphan medicine: for a rare disease (affecting no more than 5 in 10,000 people in the EU).
- Conditional approval: approved on less complete data because the need is high; more data must follow.
Key facts
- Brand
- Ayvakyt
- Active substance
- avapritinib
- Disease area
- Gastrointestinal Stromal Tumors
- Medicine group
- Other antineoplastic agents;Protein kinase inhibitors
- ATC code
- L01EX18
- Marketing authorisation holder
- Blueprint Medicines (Netherlands) B.V.
- Authorised in the EU since
- 2020
Official information
In the USA
Same active substance: Ayvakit
Specialist medicine: started and supervised by a specialist doctor or hospital. This page is a plain-language summary of public official information, not medical advice; follow your doctor and the official product information.
Common questions
What is Ayvakyt used for?
Unresectable or metastatic gastrointestinal stromal tumour (GIST) AYVAKYT is indicated as monotherapy for the treatment of adult patients with unresectable or metastatic gastrointestinal stromal tumours (GIST) harbouring the platelet-derived growth factor receptor alpha (PDGFRA) D842V mutation. Advanced systemic mastocytosis (AdvSM) AYVAKYT is indicated as monotherapy for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated haematological neoplasm (SM-AHN) or mast cell leukaemia (MCL), after at least one systemic therapy. Indolen…
What is the active substance in Ayvakyt?
Ayvakyt contains avapritinib.
Who makes Ayvakyt?
The EU marketing authorisation holder is Blueprint Medicines (Netherlands) B.V..
Is Ayvakyt an orphan medicine?
Yes. Ayvakyt is designated an orphan medicine in the EU, for a rare disease (Gastrointestinal Stromal Tumors).
Source: European Medicines Agency medicines data (ema.europa.eu), reproduced with acknowledgement of the source. Page made 5 November 2026 by Satya Babu.
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