Blueprint Medicines Corporation announced data presentations at the 2025 European Hematology Association (EHA2025) Hybrid Congress and the European Academy of Allergy and Clinical Immunology (EAACI) Congress 2025. These presentations showcased over a decade of collaboration and research in systemic mastocytosis (SM).
Key results from the PIONEER, PATHFINDER, and EXPLORER trials, with follow-up extending up to five years in indolent SM (ISM) and 6.5 years in advanced SM, reinforced AYVAKIT's (avapritinib) position as a durable standard of care. The data demonstrated sustained disease control in ISM and prolonged survival in advanced SM.
The PIONEER three-year data specifically highlighted the durable clinical benefits and consistent safety profile of long-term AYVAKIT use in ISM. AYVAKIT is approved by the U.S. Food and Drug Administration (FDA) for advanced SM in June 2021 and ISM in May 2023, with global approvals in 16 countries.
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