AYVAKIT
AYVAKIT (avapritinib) is a highly selective oral kinase inhibitor developed by Blueprint Medicines that targets activating mutations in KIT and PDGFRα — the dominant oncogenic drivers in gastrointestinal stromal tumors (GIST) and systemic mastocytosis. The drug fits precisely into the ATP-binding pocket of these mutant kinases to block the aberrant signaling driving tumor growth and mast cell accumulation. AYVAKIT is approved for PDGFRA Exon 18 D842V-mutant GIST — a subset previously insensitive to imatinib — and for advanced and indolent systemic mastocytosis; Blueprint is studying avapritinib versus sunitinib in second-line GIST in an ongoing Phase 3 trial.
Upcoming catalysts
Programs
GIST
GIST is the most common GI mesenchymal tumor; imatinib is first-line but patients inevitably progress, and second-line sunitinib has limited efficacy across mutation subtypes. AYVAKIT is approved for the PDGFRA D842V mutation subset and systemic mastocytosis; this Phase 3 program tests avapritinib versus sunitinib in a broader second-line GIST population regardless of mutation type, measuring progression-free survival as the primary endpoint.