Class/mechanism from the NCI Drug Dictionary: An orally bioavailable inhibitor of specific mutated forms of platelet-derived growth factor receptor alpha (PDGFR alpha; PDGFRa) and mast/stem cell factor receptor c-Kit (SCFR), with potential antineoplastic activity. Upon oral administration, avapritinib specifically binds to and inhibits specific mutant forms of PDGFRa and c-Kit, including the PDGFRa D842V mutant and various KIT exon 17 mutants. This results in the inhibition of PDGFRa- and c-Kit-mediated signal transduction pathways and the inhibition of proliferation in tumor cells that express these PDGFRa and c-Kit mutants.
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Diseases for which it is established (work in progress)
Patient Drug Information
History of changes in FDA indication
- 2020-01-09: Initial approval for adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including D842V mutations. (Based on NAVIGATOR)
- 2021-06-16: Approved for adult patients with advanced systemic mastocytosis (AdvSM), including patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematological neoplasm (SM-AHN), and mast cell leukemia (MCL). (Based on EXPLORER and PATHFINDER)
- 2023-05-22: Approved for the treatment of adults with indolent systemic mastocytosis. (Based on PIONEER)
History of changes in EMA indication
- 2020-09-24: Initial conditional authorization as Ayvakyt. 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. (Based on NAVIGATOR)
- Uncertain date: 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.
- 2023-11-09: CHMP recommendation - Ayvakyt is indicated for the treatment of adult patients with indolent systemic mastocytosis (ISM) with moderate to severe symptoms inadequately controlled on symptomatic treatment.
Also known as
- Code name: BLU-285
- Brand name: Ayvakit, Ayvakyt