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The first study of avapritinib, a drug designed to hit the KIT D816V mutation that drives most mastocytosis, produced responses in three quarters of patients with advanced disease and set the dose for later trials.
Phase 1 dose-escalation and expansion study of 86 patients with advanced systemic mastocytosis treated with avapritinib, a selective inhibitor of KIT D816V.
The overall response rate in response-evaluable patients was 75 percent with deep and durable responses, falls in serum tryptase and KIT D816V allele burden, and a recommended dose of 200 mg daily; cognitive effects and intracranial bleeding (mainly with severe thrombocytopenia) were the notable toxicities. The results, with PATHFINDER, led to approval of avapritinib for advanced systemic mastocytosis in 2021.
Avapritinib is the most active drug in advanced systemic mastocytosis; platelet counts must be checked before and during treatment.
Shares Avapritinib, Advanced systemic mastocytosis (aggressive SM, SM with an associated haematological neoplasm, mast cell leukaemia), Nature Medicine.
Shares Avapritinib, Advanced systemic mastocytosis (aggressive SM, SM with an associated haematological neoplasm, mast cell leukaemia).
Shares EXPLORER, Avapritinib, Advanced systemic mastocytosis (aggressive SM, SM with an associated haematological neoplasm, mast cell leukaemia).