16 June 2021 - On June 16, 2021, the FDA approved avapritinib (Ayvakit, Blueprint Medicines) for adult patients with advanced systemic mastocytosis, including patients with aggressive systemic mastocytosis, systemic mastocytosis with an associated hematological neoplasm and mast cell leukaemia.
Efficacy was evaluated in EXPLORER and PATHFINDER, two multi-center, single-arm, open-label clinical trials enrolling patients with advanced systemic mastocytosis.