FDA approval date:
AYVAKIT is a kinase inhibitor indicated for the treatment of adults with
unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a
platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation,
including PDGFRA D842V mutations.
Mechanism of Action:
Avapritinib is a tyrosine kinase inhibitor that targets PDGFRA and PDGFRA D842 mutants as well as
multiple KIT exon 11, 11/17 and 17 mutants with half maximal inhibitory concentrations (IC50s) less than
25 nM. Certain mutations in PDGFRA and KIT can result in the autophosphorylation and constitutive
activation of these receptors which can contribute to tumor cell proliferation. Other potential targets for
avapritinib include wild type KIT, PDGFRB, and CSFR1.
In in vitro cellular assays, avapritinib inhibited the autophosphorylation of KIT D816V and PDGFRA
D842V, mutants associated with resistance to approved kinase inhibitors, with IC50 of 4 nM and 30 nM,
respectively. Avapritinib also had anti-tumor activity in mice implanted with an imatinib-resistant patientderived xenograft model of human GIST with activating KIT exon 11/17 mutations.
• 100 mg, round, white film-coated, printed with blue ink “BLU” on one side and “100” on the other
• 200 mg, capsule shaped, white film-coated, printed with blue ink “BLU” on one side and “200” on
the other side.
• 300 mg, capsule shaped, white film-coated, printed with blue ink “BLU” on one side and “300” on
the other side.
The most common adverse reactions (incidence ≥ 20%) are edema, nausea,
fatigue/asthenia, cognitive impairment, vomiting, decreased appetite, diarrhea,
hair color changes, increased lacrimation, abdominal pain, constipation, rash