Ayvakin for glioblastome – pro
AYVAKIT® (avapritinib) is indicated for the treatment of adult patients with: Unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including PDGFRA D842V mutations, and Advanced SM (AdvSM) including patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematological neoplasm (SM-AHN), and mast cell leukemia (MCL). Avapritinib can inhibit PDGFRA signaling, penetrate the blood-brain barrier, and demonstrate clinical responses in a subset of patients with these tumors. It is in the early stages of investigation and considered E/I and not medically necessary.
Mayr, L., et al. (2025). Effective targeting of PDGFRA-altered high-grade glioma with avapritinib. Cancer Cell. doi.org/10.1016/j.ccell.2025.02.018.
Shen Y, Thng DKH, Wong ALA, Toh TB. Mechanistic insights and the clinical prospects of targeted therapies for glioblastoma: a comprehensive review. Exp Hematol Oncol. 2024 Apr 13;13(1):40. doi: 10.1186/s40164-024-00512-8. PMID: 38615034; PMCID: PMC11015656.
Ayvaknt, Prescribing Information 2025