![]() ![]() In addition, the PIONEER trial met all key secondary endpoints, including significant improvements across all measures of mast cell burden. At 24 weeks, the control arm had a reduction of 9.2 points in mean TSS. The AYVAKIT arm had a reduction of 15.6 points in mean TSS at 24 weeks, which continued to deepen to 20.2 points at 48 weeks in patients who rolled over to the Part 3 open-label extension study. TSS was assessed by the Indolent SM Symptom Assessment Form (ISM-SAF). The trial, which was designed to assess AYVAKIT plus best available care versus placebo plus best available care (control arm), achieved its primary endpoint with a highly significant difference in the mean change in total symptom score (TSS) at 24 weeks (p=0.003). In addition, Blueprint Medicines plans to present detailed data from the PIONEER trial at an upcoming medical meeting. Food and Drug Administration (FDA) for AYVAKIT in non-advanced SM in the fourth quarter of 2022, with a subsequent submission of a type II variation marketing authorization application to the European Medicines Agency (EMA) anticipated in 2023. Based on these top-line data, Blueprint Medicines plans to submit a supplemental new drug application (sNDA) to the U.S. 17, 2022 /PRNewswire/ - Blueprint Medicines Corporation (NASDAQ: BPMC) today announced positive top-line results from the registrational Part 2 of the PIONEER clinical trial of AYVAKIT ® (avapritinib) in patients with non-advanced systemic mastocytosis (SM) demonstrating clinically meaningful and highly significant improvements across the primary and all key secondary endpoints, including patient-reported symptoms and objective measures of mast cell burden. Blueprint Medicines to host investor conference call and webcast today at 8:00 a.m. Plan to submit supplemental new drug application to FDA in fourth quarter of 2022. AYVAKIT had a favorable safety profile compared to the control arm, supporting potential for long-term treatment. Highly significant reductions across all objective measures of mast cell burden reinforce the disease modifying activity of AYVAKIT. AYVAKIT showed a superior mean change in total symptom score (p=0.003), compared to placebo plus best available care. ![]()
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