FDA Approves Avapritinib for Advanced Systemic Mastocytosis

The FDA has approved avapritinib (Ayvakit, Blueprint Medicines Corp) for adult patients with advanced systemic mastocytosis (AdvSM), including patients with aggressive systemic mastocytosis, systemic mastocytosis with an associated hematological neoplasm, and mast cell leukemia.

The researchers anaylzed efficacy results from both the EXPLORER and PATHFINDER studies in patients with AdvSM. Duration of response (DoR), time to response, changes in individual measures of mast cell burden, and overall response rate (ORR) were looked at as additional efficacy measures. Further, 53 patients received daily doses of avapritinib up to 200 mg, according to an FDA press release.

In all evaluated patients in both trials, the ORR was 57%, with 28% complete remissions and 28% partial remissions. The median time to response was 2.1 months, whereas the median DoR was 38.3 months.

In patients with AdvSM, the most common adverse events were edema, diarrhea, nausea, and fatigue/asthenia. Because of this, avapritinib is not recommended for the treatment of patients with AdvSM with platelet counts of less than 50 X 109/L, according to the FDA.

REFERENCE

FDA approves avapritinib for advanced systemic mastocytosis. FDA. Published June 16, 2021. Accessed June 16, 2021. https://www.fda.gov/drugs/drug-approvals-and-databases/fda-approves-avapritinib-advanced-systemic-mastocytosis?utm_medium=email&utm_source=govdelivery