Health
Blueprint Medicines, a Sanofi Company, Highlights Long-Term Clinical Benefit of AYVAKIT® (avapritinib) Across Indolent and Advanced Systemic Mastocytosis at 2025 ASH Annual Meeting
Blueprint Medicines, a Sanofi company, today announced updated data reinforcing the clinical efficacy and safety of long-term AYVAKIT® (avapritinib) use across the spectrum of systemic mastocytosis (SM), including indolent and advanced SM. AYVAKIT led to sustained symptom and quality-of-life benefits in indolent SM (ISM) after a median follow-up of more than three years, and extended survival rates in advanced SM after a median follow-up of more than four years. Across both forms of SM, AYVAKIT
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[{"type":"text","content":"-- Durable disease control and quality-of-life improvements for patients with ISM in the PIONEER trial --","length":105,"tagName":"p","attribs":{}},{"type":"text","content":"-- Prolonged survival, including in the first-line treatment setting, for patients with advanced SM in the PATHFINDER trial --","length":126,"tagName":"p","attribs":{}},{"type":"text","content":"CAMBRIDGE, Mass., Dec. 5, 2025 /PRNewswire/ -- Blueprint Medicines, a Sanofi company, today announced updated data reinforcing the clinical efficacy and safety of long-term AYVAKIT® (avapritinib) use across the spectrum of systemic mastocytosis (SM), including indolent and advanced SM. AYVAKIT led to sustained symptom and quality-of-life benefits in indolent SM (ISM) after a median follow-up of more than three years, and extended survival rates in advanced SM after a median follow-up of more than four years. Across both forms of SM, AYVAKIT showed bone health improvements reflecting disease-modifying effects, and a safety and tolerability profile consistent with previously reported results. As part of its ongoing leadership to improve SM care, Blueprint Medicines will report one oral presentation and seven poster presentations at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition, December 6–9 in Orlando, Florida.","length":962,"tagName":"p"},{"type":"image","alt":"(PRNewsfoto/Blueprint Medicines)","displaySize":"","headline":null,"caption":"(PRNewsfoto/Blueprint Medicines)","className":"","disableSlideshowImg":false,"size":{"original":{"width":400,"height":186,"url":"https://media.zenfs.com/en/prnewswire.com/66d4d034940e0c5a6f4ac4ba630a14a8"},"resized":{"url":"https://s.yimg.com/ny/api/res/1.2/NpuYza_grizjsylUDnkf_Q--/YXBwaWQ9aGlnaGxhbmRlcjt3PTcwNTtoPTMyODtjZj13ZWJw/https://media.zenfs.com/en/prnewswire.com/66d4d034940e0c5a6f4ac4ba630a14a8","width":400,"height":186}},"href":"https://mma.prnewswire.com/media/2700885/Blueprint_Medicines_a_Sanofi_company_Logo_v2.html","hrefExternal":true,"rel":"nofollow"},{"type":"text","content":""Our ASH data add to the substantial body of evidence generated since we initiated clinical development for systemic mastocytosis about a decade ago, and reflect the transformative benefits AYVAKIT has delivered since its FDA approval for advanced SM in 2021 and ISM in 2023," said Mik Rinne, M.D., ...