New Ayvakit data will ease wider label, but market impact is unclear

17 August 2022

Blueprint Medicines (Nasdaq: BPMC) has announced positive top-line results from the registrational Part 2 of the PIONEER clinical trial of Ayvakit (avapritinib).

The study is examining the impact of Ayvakit, a kinase inhibitor, on people with non-advanced systemic mastocytosis (SM).

Blueprint said that top-line data show clinically-meaningful and highly-significant improvements across the primary and all key secondary endpoints, including patient-reported symptoms as well as objective measures of mast cell burden.

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