Avapritinib

Avapritinib (Ayvakyt, Blueprint Medicines (Netherlands) B.V.) has 3 EU-approved indications in 2 tumour types. 1 needs a biomarker result: PDGFR (1).
Approved indications of Avapritinib
Biomarker requirementRegimenSettingConditions
Gastrointestinal stromal tumour1
PDGFRA D842V
Monotherapy
Any line
advanced
None
Systemic mastocytosis2
None
Monotherapy
2L
Advanced systemic mastocytosis only (ASM, SM-AHN or MCL); After at least one systemic therapy
None
Monotherapy
Any line
Indolent systemic mastocytosis only; Moderate to severe symptoms inadequately controlled on symptomatic treatment
Full text of section 4.1 (therapeutic indications)

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).

Label history

12 changes to the Ayvakyt label since 2021, 2 of them to the indications. Newest first, in the words of EMA's record of each procedure.

Changes