Blueprint Medicines presents registrational data from PIONEER trial AYVAKIT

Blueprint Medicines announced detailed results from the PIONEER trial of AYVAKIT in patients with indolent systemic mastocytosis. As previously reported, AYVAKIT achieved statistically significant and clinically meaningful improvements on the primary and all key secondary endpoints. New results further highlight the benefits of AYVAKIT on pathological mast cell burden, disease symptoms – including total symptom score, most severe symptom and all individual symptoms – and quality of life. Across clinical measures, improvements continued to deepen over time in patients treated with AYVAKIT through 48 weeks. AYVAKIT was well-tolerated with a safety profile favorable to placebo, and 96 percent of patients in the AYVAKIT arm opted to continue treatment in the open-label extension study. Based on these data, Blueprint Medicines submitted a supplemental new drug application for AYVAKIT to the U.S. Food and Drug Administration and a type II variation marketing authorization application for AYVAKYT to the European Medicines Agency for the treatment of patients with indolent SM. The FDA has granted priority review to the sNDA for AYVAKIT with a Prescription Drug User Fee Act action date of May 22, 2023, and the EMA has validated the MAA for AYVAKYT.