Blueprint Medicines Announces Data Reinforcing Sustained Clinical Efficacy and Well-Tolerated Safety Profile of Long-Term AYVAKIT®/AYVAKYT® (avapritinib) Treatment at 2025 EHA and EAACI Congresses

Core Insights - Blueprint Medicines Corporation has showcased its leadership in advancing care for patients with systemic mastocytosis (SM) through data presentations at major congresses, emphasizing the effectiveness of AYVAKIT®/AYVAKYT® (avapritinib) as a standard treatment for both indolent and advanced forms of SM [1][2][9]. Data Presentation Highlights - The presentations included large patient populations from the PIONEER, PATHFINDER, and EXPLORER trials, demonstrating long-term benefits of AYVAKIT with follow-up periods of up to five years for indolent systemic mastocytosis (ISM) and 6.5 years for advanced SM [2][5]. - AYVAKIT has shown transformative clinical outcomes, including sustained disease control in ISM and prolonged survival in advanced SM, leading to an expanded view among clinicians regarding appropriate candidates for disease-modifying therapy [2][7]. Clinical Efficacy and Safety - AYVAKIT demonstrated robust improvements in overall symptoms and quality of life measures over 144 weeks, with a low treatment-related adverse event discontinuation rate of 3% and common adverse events including low-grade edemas, headache, and nausea [7][23]. - The Revised Mutation-Adjusted Risk Score (MARS-R) tool was validated for assessing overall survival risks in advanced SM patients treated with AYVAKIT, showing meaningful survival benefits across all prognostic categories [7][12]. Disease Burden and Patient Impact - The PRISM study highlighted the substantial disease burden faced by patients with ISM, including physical, social, and emotional challenges that significantly disrupt daily life [6][12]. - Patients reported various disease-related impacts, such as limitations in physical activities and work, as well as issues with pain, anxiety, and adjustments in daily routines to avoid triggers [8][12]. Regulatory and Market Position - AYVAKIT is the first and only FDA-approved treatment targeting the root cause of SM, with approvals for both advanced SM and ISM, and is marketed in 16 countries globally [14][26]. - The company aims to expand its impact by advancing a broad pipeline of programs in mast cell diseases and solid tumors, leveraging its established research and commercial capabilities [26].