NCIt definition : 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. PDGFRa and c-Kit, protein
tyrosine kinases and tumor-associated antigens (TAAs), are mutated in various tumor
cell types; they play key roles in the regulation of cellular proliferation.;