-- In the European Union, patients with indolent systemic mastocytosis now have an approved medicine that treats the primary driver of disease --

--在欧盟，无痛性系统性肥大细胞增多症患者现在有一种经批准的药物来治疗疾病的主要驱动因素--

-- Approval based on data from PIONEER trial, in which AYVAKYT achieved significant improvements across a broad range of symptoms with a safety profile comparable to placebo1 --

--根据PIONEER试验的数据获得批准，在该试验中，AYVAKYT在广泛的症状中取得了显着改善，其安全性与安慰剂相当1--

CAMBRIDGE, Mass., Dec. 12, 2023 /PRNewswire/ -- Blueprint Medicines Corporation (Nasdaq: BPMC) today announced the European Commission has approved AYVAKYT® (avapritinib) for the treatment of adult patients with indolent systemic mastocytosis (ISM) with moderate to severe symptoms inadequately controlled on symptomatic treatment.

马萨诸塞州剑桥市，2023年12月12日/PRNewswire/--Blueprint Medicines Corporation （纳斯达克：BPMC）今天宣布，欧盟委员会已批准AYVAKYT®（avapritinib）用于 治疗成人无痛性全身性肥大细胞增多症（ISM），中度至重度症状对症治疗控制不足。

AYVAKYT is the first and only approved therapy for people living with ISM in Europe.  .

艾瓦基特 是欧洲第一个也是唯一一个被批准用于ISM患者的治疗方法。 。

Systemic mastocytosis (SM) is a rare hematologic disorder that can lead to a range of debilitating symptoms with a significant impact on patients' quality of life. The majority of patients living with SM have ISM, and there are approximately 40,000 people living with ISM in the European Union.2,3* AYVAKYT was designed to potently and selectively target KIT D816V, the primary underlying driver of the disease. .

系统性肥大细胞增多症（SM）是一种罕见的血液系统疾病，可导致一系列衰弱症状，对患者的生活质量产生重大影响。大多数SM患者患有ISM，欧盟约有40000人患有ISM。2,3*AYVAKYT旨在有效和选择性地靶向KIT D816V，这是该疾病的主要潜在驱动因素。。

'Today's approval represents an important step toward delivering a new global standard of care for patients with ISM and builds on years of collaboration with the SM community,' said Georg Pirmin Meyer, M.D., Senior Vice President, International at Blueprint Medicines. 'For the first time in Europe ISM patients have an approved therapy, marking a new era in the treatment of this disease.

Blueprint Medicines国际高级副总裁Georg Pirmin Meyer医学博士说：“今天的批准标志着向为ISM患者提供新的全球护理标准迈出了重要一步，并建立在与SM社区多年合作的基础上。”ISM患者首次在欧洲获得批准的治疗，标志着该疾病治疗的新时代。

AYVAKYT is the first approved medicine for both ISM and advanced SM, and our team is committed to bringing this transformative therapy to patients across the spectrum of disease.'  .

AYVAKYT是第一种被批准用于ISM和晚期SM的药物，我们的团队致力于将这种变革性疗法带给各种疾病的患者。”。

'Indolent systemic mastocytosis can be characterized by significant symptom burden across multiple organ systems, which can profoundly impact patients' ability to perform activities of daily living in a relevant proportion of patients,' said Jens Panse, M.D., Deputy Director of the Department of Hematology/Oncology of the University Hospital RWTH Aachen. 'AYVAKYT represents an important treatment breakthrough as the first medicine approved for patients living with ISM, and the only therapy designed to selectively target the primary genetic driver of the disease.

亚琛州立大学医院血液学/肿瘤学系副主任延斯·潘斯（Jens Panse，M.D.）说：“惰性系统性肥大细胞增多症的特征是多器官系统的症状负担严重，这会深刻影响患者在相关比例的患者中进行日常生活活动的能力。”。'AYVAKYT代表了一项重要的治疗突破，它是第一种被批准用于ISM患者的药物，也是唯一一种旨在选择性靶向该疾病主要遗传驱动因素的疗法。

In the PIONEER trial, AYVAKYT showed statistically significant and durable clinical benefits across all measured ISM symptoms with a well-tolerated safety profile. Based on these practice-changing data, AYVAKYT has the potential to advance treatment for a broad range of patients living with ISM.'   .

在PIONEER试验中，AYVAKYT在所有测量的ISM症状中均显示出统计学上显着且持久的临床益处，并且具有良好的耐受性安全性。根据这些改变实践的数据，AYVAKYT有可能为广泛的ISM患者推进治疗。”  。

The approval follows the positive opinion by the Committee for Medicinal Products for Human Use (CHMP), and this EC decision is based on data from the double-blind, placebo-controlled PIONEER trial – the largest study ever conducted in ISM. AYVAKYT showed clinically meaningful improvements versus placebo in the primary and all key secondary endpoints, including overall symptoms and measures of mast cell burden.

该批准是在人类使用药品委员会（CHMP）的积极意见之后做出的，欧盟委员会的这项决定是基于双盲、安慰剂对照的PIONEER试验的数据，该试验是ISM有史以来进行的最大规模的研究。与安慰剂相比，AYVAKYT在主要和所有关键次要终点（包括总体症状和肥大细胞负荷的测量）均显示出临床上有意义的改善。

AYVAKYT was well-tolerated with a favorable safety profile, and most adverse events (AEs) were reported as mild (Grade 1). The most common AEs were flushing, edema, increased blood alkaline phosphate and insomnia.1   .

AYVAKYT耐受性良好，安全性良好，大多数不良事件（AE）报告为轻度（1级）。最常见的不良反应是潮红、水肿、血碱性磷酸盐升高和失眠。1 。

'Many people living with indolent systemic mastocytosis face unpredictable and severe symptoms, which significantly impair their ability to work or spend quality time with their family, friends and communities,' said Patrizia Marcis, President of the Associazione Italiana Mastocitosi (ASIMAS) ODV. 'Today's approval offers a new sense of hope to the ISM community, and we are proud to collaborate with clinical researchers, patients and companies like Blueprint Medicines to advance care for all those living with the disease.'   .

意大利肥大细胞增多症协会（ASIMAS）ODV主席帕特里齐亚·马西斯（PatriziaMarcis）说：“许多患有惰性全身性肥大细胞增多症的人面临着不可预测的严重症状，这严重损害了他们的工作能力或与家人、朋友和社区共度美好时光。”今天的批准为ISM社区带来了新的希望，我们很自豪能与临床研究人员、患者和Blueprint Medicines等公司合作，为所有患有这种疾病的人提供更好的护理。” 。

In Europe, Blueprint Medicines plans to initiate its first commercial launch in Germany, followed by additional markets based on local healthcare technology assessment and reimbursement process timelines.

在欧洲，Blueprint Medicines计划在德国启动其首次商业推出，然后根据当地医疗保健技术评估和报销流程时间表扩大市场。

About AYVAKYT® (avapritinib)

艾伐基特®（阿普替尼）

AYVAKYT® (avapritinib) is a kinase inhibitor approved by the European Commission for the treatment of three indications: adults with indolent systemic mastocytosis (ISM), adults with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematological neoplasm (SM-AHN) or mast cell leukemia (MCL), after at least one systemic therapy, and adults with unresectable or metastatic gastrointestinal stromal tumors (GIST) harboring the PDGFRA D842V mutation.4 Under the brand name AYVAKIT®, the medicine is approved in the U.S.

AYVAKYT®（avapritinib）是一种激酶抑制剂，经欧盟委员会批准用于治疗三种适应症：惰性系统性肥大细胞增多症（ISM）的成年人，侵袭性系统性肥大细胞增多症（ASM）的成年人，伴有血液肿瘤（SM-AHN）或肥大细胞白血病（MCL）的系统性肥大细胞增多症，至少经过一次全身治疗，以及患有PDGFRA D842V突变的不可切除或转移性胃肠道间质瘤（GIST）的成年人。4该药以AYVAKIT®品牌在美国获得批准。

for the treatment of adults with ISM, adults with advanced SM, including ASM, SM-AHN and MCL, and adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations.5  .

对于ISM成人的治疗，包括ASM，SM-AHN和MCL在内的晚期SM成人，以及具有PDGFRA外显子18突变（包括PDGFRA D842V突变）的不可切除或转移性GIST的成人。

To learn about ongoing or planned clinical trials, contact Blueprint Medicines at [email protected] and +31 85 064 4001. Additional information is available at blueprintclinicaltrials.com and clinicaltrials.gov.

要了解正在进行或计划进行的临床试验，请联系 蓝图医药公司 在[受电子邮件保护] 和+31 85 064 4001。其他信息可在blueprintclinicaltrials.com和clinicaltrials.gov上获得。

Please click here to see the Summary of Product Characteristics for AYVAKYT.

请单击此处查看AYVAKYT的产品特性摘要。

About Systemic Mastocytosis

关于系统性肥大细胞增多症

Systemic mastocytosis (SM) is a rare disease driven by the KIT D816V mutation in about 95 percent of cases.6 Uncontrolled proliferation and activation of mast cells result in chronic, severe and often unpredictable symptoms for patients across the spectrum of SM. In the European Union, approximately 40,000 people live with indolent systemic mastocytosis.2,3* A broad range of symptoms, including anaphylaxis, maculopapular rash, pruritis, diarrhea, brain fog, fatigue and bone pain, frequently persist in patients with ISM despite treatment with multiple symptom-directed therapies. This burden of disease can lead to a profound, negative impact on quality of life.  Patients often live in fear of severe, unexpected symptoms, have limited ability to work or perform daily activities, and isolate themselves to protect against unpredictable triggers.  .

在大约95%的病例中，系统性肥大细胞增多症（SM）是由KIT D816V突变驱动的罕见疾病。肥大细胞的不受控制的增殖和活化会导致SM患者出现慢性，严重且通常不可预测的症状。在欧盟，约有40000人患有惰性系统性肥大细胞增多症。2,3*广泛的症状，包括过敏反应，斑丘疹，瘙痒，腹泻，脑雾，疲劳和骨痛在内的ISM患者经常持续存在，尽管采用多种症状导向疗法进行治疗。这种疾病负担可能会对生活质量产生深远的负面影响。患者通常生活在对严重，意外症状的恐惧中，工作或日常活动的能力有限，并将自己隔离以防止不可预测的触发因素。 。

About Blueprint Medicines

关于 蓝图医药公司

Blueprint Medicines is a global precision therapy company that invents life-changing therapies for people with cancer and blood disorders. Applying an approach that is both precise and agile, we create medicines that selectively target genetic drivers, with the goal of staying one step ahead across stages of disease.

Blueprint Medicines是一家全球精准治疗公司，为癌症和血液疾病患者发明改变生命的疗法。应用一种既精确又灵活的方法，我们创造了选择性靶向遗传驱动因素的药物，目标是在疾病的各个阶段保持领先一步。

Since 2011, we have leveraged our research platform, including expertise in molecular targeting and world-class drug design capabilities, to rapidly and reproducibly translate science into a broad pipeline of precision therapies. Today, we have brought our approved medicines to patients in the United States and Europe, and we are globally advancing multiple programs for mast cell disorders, including systemic mastocytosis and chronic urticaria, breast cancer and other cancers vulnerable to CDK2 inhibition, as well as EGFR-mutant lung cancer.

自2011年以来，我们利用我们的研究平台，包括分子靶向方面的专业知识和世界一流的药物设计能力，将科学快速且可重复地转化为广泛的精确疗法。今天，我们已经将批准的药物带给了美国和欧洲的患者，我们正在全球范围内推进肥大细胞疾病的多项计划，包括全身性肥大细胞增多症和慢性荨麻疹，乳腺癌和其他易受CDK2抑制的癌症，以及EGFR突变型肺癌。

For more information, visit www.BlueprintMedicines.com and follow us on Twitter (@BlueprintMeds) and LinkedIn.  .

有关更多信息，请访问www.BlueprintMedicines.com，并在Twitter（@BlueprintMeds）和LinkedIn上关注我们。 。

Cautionary Note Regarding Forward-Looking Statements

关于前瞻性声明的警示说明

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding Blueprint Medicines' views with respect to the implications of the approval of AYVAKYT for people living with ISM in Europe; plans for Blueprint Medicines' first commercial launch in Germany, followed by additional markets; plans, strategies, timelines and expectations for Blueprint Medicines' current or future approved drugs and drug candidates; the potential benefits of any of Blueprint Medicines' current or future approved drugs or drug candidates in treating patients; and Blueprint Medicines' financial performance, strategy, goals and anticipated milestones, business plans and focus.

本新闻稿包含1995年《私人证券诉讼改革法案》修订版所指的前瞻性声明，包括但不限于关于Blueprint Medicines对批准AYVAKYT对欧洲ISM患者的影响的观点的声明；Blueprint Medicines在德国首次商业推出的计划，随后是其他市场；蓝图药物目前或未来批准的药物和候选药物的计划，策略，时间表和期望；蓝图药物目前或未来批准的任何药物或候选药物在治疗患者方面的潜在益处；以及蓝图药物的财务业绩，战略，目标和预期里程碑，业务计划和重点。

The words 'aim,' 'may,' 'will,' 'could,' 'would,' 'should,' 'expect,' 'plan,' 'anticipate,' 'intend,' 'believe,' 'estimate,' 'predict,' 'project,' 'potential,' 'continue,' 'target' and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.

“目标”、“可能”、“将”、“可能”、“将”、“应该”、“期望”、“计划”、“预期”、“打算”、“相信”、“估计”、“预测”、“项目”、“潜力”、“继续”、“目标”和类似的表达都是为了识别前瞻性陈述，尽管并非所有的前瞻性陈述都包含这些识别词。

Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation: preliminary activity and safety data may not be representative of more mature data; the risk of delay of any current or planned clinical trials or the development of Blueprint Medicines' current or future drug candidates; risks related to Blueprint Medicines' ability to successfully demonstrate th.

本新闻稿中的任何前瞻性声明均基于管理层当前的期望和信念，并受到许多风险、不确定性和重要因素的影响，这些风险、不确定性和重要因素可能导致实际事件或结果与本新闻稿中任何前瞻性声明所明示或暗示的结果产生重大差异，包括，但不限于：初步活动和安全数据可能无法代表更成熟的数据；任何当前或计划中的临床试验或蓝图药物当前或未来候选药物开发延迟的风险；与蓝图药物成功证明th的能力有关的风险。

References

参考文献

*Based on Cohen 2014 study of 548 adults with SM diagnosed from 1997 to 2010 in linked Danish national health registries, with a 14-year limited-duration prevalence estimated at 9.59 per 100,000 as of 1 January 2011

*根据科恩2014年对1997年至2010年在丹麦国家卫生登记处诊断出的548名SM成年人的研究，截至2011年1月1日，14年有限持续时间的患病率估计为每100000人9.59

Gotlib J, et al. Avapritinib versus Placebo in Indolent Systemic Mastocytosis. NEJM Evid 2023;2(6) May 23, 2023.

Gotlib J等人。阿伐他尼与安慰剂在惰性系统性肥大细胞增多症中的比较。NEJM Evid 2023；2023年5月23日，第2（6）条。

Cohen SS et al. Epidemiology of systemic mastocytosis in Denmark. Br J Haematol. 2014;166(4):521-8.

Cohen SS等人。丹麦系统性肥大细胞增多症的流行病学。Br J血液学。2014年；166（4）：521-8。

OrphaNet. Indolent systemic mastocytosis. Available at: https://www.orpha.net/consor/cgi-bin/OC_Exp.php?lng=en&Expert=98848. Accessed December 2023.

孤儿网。无痛性全身肥大细胞增多症。网址： https://www.orpha.net/consor/cgi-bin/oc_exp.php?lng=en&expert=98848.2023年12月访问。

AYVAKYT SMPC. Available at: https://www.ema.europa.eu/en/documents/ product-information/ayvakyt-epar-product-information_en.pdf. Accessed December 2023.

AYVAKYT SMPC。可在以下位置获取：https://www.ema.europa.eu/en/documents/产品信息/ayvakyt-depart-product-information_en.pdf。访问日期：2023年12月。

Blueprint Medicines. AYVAKIT (avapritinib) Prescribing Information. Available at: https://www.blueprintmedicines.com/wp-content/uploads/uspi/AYVAKIT.pdf. Accessed December 2023

蓝图药物。AYVAKIT（阿普替尼）处方信息。可在以下位置获取：https://www.blueprintmedicines.com/wp-content/uploads/uspi/ayvakit.pdf.访问日期：2023年12月

Mesa R.A et al. Perceptions of patient disease burden and management approaches in systemic mastocytosis: Results of the TouchStone Healthcare Provider Survey. Cancer. 2022;128(20):3700-3708

Mesa R.A等人。对系统性肥大细胞增多症患者疾病负担和管理方法的看法：试金石医疗保健提供者调查的结果。癌症。2022年；128（20）：3700-3708

Trademarks

商标

Blueprint Medicines, AYVAKIT, AYVAKYT and associated logos are trademarks of Blueprint Medicines Corporation.

Blueprint Medicines、AYVAKIT、AYVAKYT和相关徽标是Blueprint Medicines Corporation的商标。

SOURCE Blueprint Medicines Corporation

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