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].

撰文丨王聪 编辑丨王多鱼 排版丨水成文 2025 年 6 月 2 日，法国制药巨头 赛诺菲 （Sanofi） 宣布收购美国生物制药上市公司 Blueprint Medicines ， 以扩大自身在罕见免疫疾病领域的产品组合，并增添免疫学领域的早期研发管线， 交易总金 额高达 95 亿美元 。 根据收购条款，赛诺菲将在交易完成时以每股 129.00 美元的现金支付，股权价值约为 91 亿 美元，此 外，Blueprint 股东还将获得一份不可交易的或有价值权 （CVR） ，该权利持有人将有权在 BLU-808 未 来达到研发里程碑和监管里程碑时分别获得额外里程碑付款。因此，此次收购的总股权价值在完全稀释的 基础上约为 95 亿 美元。该收购预计在 2025 年第三季度完成。 此次收购对于赛诺菲的战略意义在于： 1、强化免疫学布局 ： 2、商业化协同 ： Blueprint 在过敏、皮肤和免疫领域的渠道资源，可加速赛诺菲免疫学产品落地。 3、财务影响 ： 赛诺菲将获得 Blueprint 在 系统性肥大细胞增多症 （S ystemic Mastocytosis ） 领域的独家药物 Ayvakit/Ayvakyt ...

法国制药巨头赛诺菲(SNY.US)将以91亿美元股权价值收购美国生物制药公司Blueprint Medicines Corp. (BPMC.US)，此举将显著增强其在罕见免疫疾病领域的治疗管线。 Blueprint目前主营治疗系统性肥大细胞增多症的靶向药物(美国商品名Ayvakit/欧洲商品名Ayvakyt)，该 罕见病会导致免疫细胞异常积聚引发荨麻疹、腹痛等症状。据公司预测，该药物2025年销售额预计达7- 7.2亿美元。 该交易彰显了赛诺菲打造免疫学领域领导地位的雄心。今年早些时候，该公司已宣布拟以最高19亿美元 收购Dren Bio,Inc.的自身免疫疾病药物。 赛诺菲首席执行官Paul Hudson表示："此次收购是我们罕见病和免疫学产品组合的战略性突破，不仅丰 富了我们的研发管线，更将加速我们转型为全球领先免疫学企业的进程。" Hudson补充道："赛诺菲仍保持可观的并购能力。" 赛诺菲指出，Blueprint还拥有包括多款在研免疫药物在内的丰富产品管线，并在专科医生群体中建立了 稳固的临床网络。 根据双方周一发布的联合声明，赛诺菲将以每股129美元的现金价格进行收购，较Blueprint上周五收盘 ...

  Sanofi to acquire Blueprint Medicines, expanding portfolio in rare immunological disease and adding early-stage pipeline in immunology Adds fast-growing and only approved medicine for advanced and indolent systemic mastocytosis to the Sanofi portfolio Paris and Cambridge, MA. June 2, 2025. Sanofi and Blueprint Medicines Corporation (Blueprint), a US-based, publicly traded biopharmaceutical company specializing in systemic mastocytosis (SM), a rare immunological disease, and other KIT-driven diseases, have ...