Avapritinib

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.

Also known as (S)-1-(4-fluorophenyl)-1-(2-(4-(6-(1-methyl-1Hpyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl)piperazin-yl)pyrimidin-5-yl)ethan-1-amine, AVAPRITINIB, AYVAKIT, Ayvakyt, BLU 285, BLU-285, BLU285, CS3007, PDGFR alpha/KIT Mutant-specific Inhibitor BLU-285 — per NCIT

Also identified as

• ATC L01EX18 per RXNORM
• NCIT NCIT:C123827 per NCIT
• SCTID 838409003 per RXNORM
• SCTID 838462005 per RXNORM
• UNII 513P80B4YJ per RXNORM

Indications