FDA D.I.S.C.O. Burst Edition: FDA approval of Ayvakit (avapritinib) for adult patients with advanced systemic mastocytosis, including patients with aggressive systemic mastocytosis, systemic mastocytosis with an associated hematological neoplasm, and mast cell leukemia
Welcome back to the D.I.S.C.O., FDA’s Drug Information Soundcast in Clinical Oncology, Burst Edition, brought to you by FDA’s Division of Drug Information in partnership with FDA’s Oncology Center of Excellence. Today we have another quick update on a recent FDA cancer drug approval.
On June 16, 2021, the FDA approved avapritinib (brand name Ayvakit) for adult patients with advanced systemic mastocytosis, including patients with aggressive systemic mastocytosis, systemic mastocytosis with an associated hematological neoplasm, and mast cell leukemia.
Efficacy was evaluated in EXPLORER and PATHFINDER, two multi-center, single-arm, open-label clinical trials enrolling patients with advanced systemic mastocytosis. The main efficacy outcome measure was overall response rate per modified IWG-MRT-ECNM criteria, as adjudicated by a central committee. Additional efficacy measures were duration of response, time to response, and changes in individual measures of mast cell burden.
The overall response rate in all patients evaluated in both trials combined was 57%, with 28% complete remissions and 28% partial remissions. The median duration of response was 38.3 months and the median time to response was 2.1 months.
The most common adverse reactions reported in more than 20% of patients with advanced systemic mastocytosis were edema, diarrhea, nausea, and fatigue or asthenia.
Avapritinib is not recommended for the treatment of patients with advanced systemic mastocytosis with platelet counts of less than 50 X 109/L.
Full prescribing information for this approval can be found on the web at www.fda.gov/drugsatFDA.
Health care professionals should report serious adverse events to FDA’s MedWatch Reporting Program at www.fda.gov/medwatch.
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