Blueprint Medicines Reports (PIONEER) Trial Results of Ayvakit (avapritinib) for the Treatment of Non-Advanced Systemic Mastocytosis


Part 2 of the (PIONEER) trial evaluates Ayvakit (25mg, qd) + best available care vs PBO + best available care in 212 patients with non-advanced SM for 24wk.
The trial met its 1EPs & 2EPs i.e., 15.6 points vs 9.2 points reduction in TSS @24wks., improvements across all measures of mast cell burden, ≥50% reduction of serum tryptase (53.9% vs 0%), was well-tolerated & had a favorable safety profile, 96.5% vs 93.0% has completed 24wks. of therapy, 0.7% vs 0% discontinued due to TRAEs, rate of AEs (90.8% vs 93.0)
The company plans to submit an sNDA of Ayvakit to the US FDA in non-advanced SM in Q4’22 with an expected submission of a type II variation MAA to the EMA in 2023. The (PIONEER) trial results will be presented at an upcoming medical meeting

Ref: Blueprint Medicines | Image: Blueprint Medicines