NEW YORK--(BUSINESS WIRE)--Pfizer Inc. (NYSE: PFE) announced today that the European Commission (EC) has granted marketing authorization for HYMPAVZI™ (marstacimab) for the routine prophylaxis of bleeding episodes in patients 12 years of age and older weighing at least 35 kg with severe hemophilia A (congenital factor VIII [FVIII] deficiency, FVIII <1%) without FVIII inhibitors or severe hemophilia B (congenital factor IX [FIX] deficiency, FIX <1%) without FIX inhibitors..

纽约--（商业新闻短讯）--辉瑞公司（纽约证券交易所代码：PFE）今天宣布，欧盟委员会（EC）已授予HYMPAVZI™（marstacimab）上市许可，用于常规预防12岁及以上体重至少35公斤的严重血友病A（先天性VIII因子缺乏症，FVIII<1%），无FVIII抑制剂或严重血友病B（先天性IX因子缺乏症，FIX<1%），无FIX抑制剂的患者出血发作。。

HYMPAVZI is the first and only anti-tissue factor pathway inhibitor (anti-TFPI) approved in the European Union (EU) for the treatment of hemophilia A or B and the first hemophilia medicine approved in the EU to be administered via a pre-filled, auto-injector pen. HYMPAVZI offers a subcutaneous treatment option with a once-weekly dosing schedule and minimal preparation required for each individual administration..

HYMPAVZI是欧盟（EU）批准用于治疗血友病A或B的第一种也是唯一一种抗组织因子途径抑制剂（anti-TFPI），也是欧盟批准的第一种通过预填充自动注射器笔给药的血友病药物。HYMPAVZI提供了一种皮下治疗选择，每周给药一次，每次给药所需的准备最少。。

“There is a considerable treatment burden associated with the standard-of-care options for hemophilia A and B, including time-consuming preparation and administration of infusions and injections potentially causing missed doses and an increased risk of bleeding,” said Dr. Laurent Frenzel, Head of the Hemophilia Treatment and Research Center at the Necker-Enfants malades Hospital (Paris Cité).

内克尔儿童梅拉德斯医院（巴黎市）血友病治疗和研究中心主任劳伦特·弗伦泽尔博士说：“与a型和B型血友病的标准治疗选择相关的治疗负担相当大，包括耗时的输液和注射准备和管理，可能导致剂量漏服和出血风险增加。”。

“HYMPAVZI is a significant advancement for eligible patients in that it may provide bleed prevention as well as once-weekly subcutaneous administration via a pre-filled pen.”.

“HYMPAVZI对符合条件的患者来说是一项重大进步，因为它可以通过预先填充的笔提供出血预防以及每周一次的皮下给药。”。

Hemophilia is a family of rare genetic blood diseases caused by a clotting factor deficiency (FVIII in hemophilia A, FIX in hemophilia B), impacting more than 800,000 people globally.1 Diagnosed in early childhood, hemophilia inhibits the blood’s ability to clot properly, increasing the risk of repeated bleeding inside the joints, which can lead to permanent joint damage.2,3 Despite significant progress in hemophilia treatment in recent years, many people living with the disease continue to experience bleeding episodes and manage their condition with frequent intravenous infusions that may need to be administered multiple times a week.4.

血友病是由凝血因子缺乏症（血友病a中的FVIII，血友病B中的FIX）引起的罕见遗传性血液病家族，影响全球80多万人。1在儿童早期诊断出血友病，会抑制血液正确凝结的能力，增加关节内反复出血的风险，从而可能导致永久性关节损伤。2,3尽管近年来血友病治疗取得了重大进展，但许多患有该病的人继续经历出血发作，并通过频繁的静脉输注来控制病情，可能需要每周多次给药。

“HYMPAVZI offers a first-in-class treatment option for people living with hemophilia, a disease that often leads to recurring joint bleeds and can impact daily activities as simple as climbing stairs,” said Alexandre de Germay, Chief International Commercial Officer and Executive Vice President, Pfizer.

辉瑞首席国际商务官兼执行副总裁亚历山大德日尔曼（AlexandreDeGermay）表示：“HYMPAVZI为血友病患者提供了一流的治疗选择，血友病通常会导致关节反复出血，并可能影响日常活动，就像爬楼梯一样。”。

“This approval builds on Pfizer’s more than four-decade commitment to improve the standard of care in hemophilia, and we look forward to delivering this medicine that reduced bleeds as compared to factor prophylaxis and, importantly, requires limited preparation, meeting a key need for eligible patients.”.

“这项批准建立在辉瑞40多年来致力于改善血友病护理标准的基础上，我们期待着提供这种与因子预防相比减少出血的药物，重要的是，需要有限的准备，以满足符合条件的患者的关键需求。”。

The marketing authorization is based on results from the pivotal Phase 3 BASIS study (NCT03938792) that evaluated the efficacy and safety of marstacimab in adults and adolescents 12 years and older with severe hemophilia A or B without inhibitors. In the study, HYMPAVZI significantly reduced the annualized bleeding rate (ABR) for treated bleeds by 35% (ABR of 5.08 vs.

上市授权基于关键的3期基础研究（NCT03938792）的结果，该研究评估了马司他单抗在12岁及以上患有严重血友病A或B且无抑制剂的成人和青少年中的疗效和安全性。在这项研究中，HYMPAVZI显着降低了治疗出血的年出血率（ABR）35%（ABR为5.08 vs。

7.85, p-value 0.0376) during the 12-month active treatment period, demonstrating non-inferiority and superiority compared to routine prophylaxis (RP) with FVIII or FIX administered as part of usual care. The safety profile for HYMPAVZI was consistent with Phase 1/2 results, and the most commonly reported adverse events in the study were injection site reactions, headache, pruritus, and hypertension..

7.85，p值0.0376），与常规护理中使用FVIII或FIX的常规预防（RP）相比，表现出非劣效性和优越性。HYMPAVZI的安全性与1/2期结果一致，研究中最常见的不良事件是注射部位反应，头痛，瘙痒和高血压。。

This marketing authorization is valid in all 27 EU member states, as well as in Iceland, Liechtenstein, and Norway. The EC approval follows the regulatory approval of HYMPAVZI in the United States in October.

该上市授权书在所有27个欧盟成员国以及冰岛、列支敦士登和挪威均有效。欧盟委员会的批准是在10月美国监管机构HYMPAVZI批准之后进行的。

Pfizer’s more than 40-year effort to advance hemophilia treatment began with the introduction of recombinant treatments and has extended to the introduction of newer, advanced treatment modalities. In addition to recent regulatory approvals for HYMPAVZI, Pfizer reported positive results from a Phase 3 program investigating a gene therapy candidate in hemophilia A (giroctocogene fitelparvovec) in July and received regulatory approvals in Europe and the U.S.

。除了最近对HYMPAVZI的监管批准外，辉瑞公司还报告了7月份对血友病a（giroctocogene-fitelparvovec）基因治疗候选药物进行的第三阶段研究的积极结果，并在欧洲和美国获得了监管批准。

for its hemophilia B gene therapy BEQVEZ™ (fidanacogene elaparvovec)..

用于其血友病B基因治疗BEQVEZ™（fidanacogene Elaparvovovec）。。

About HYMPAVZI (marstacimab)

关于海帕夫齐（马司他滨）

Discovered by Pfizer scientists, HYMPAVZI is a rebalancing agent that targets the Kunitz 2 domain of tissue factor pathway inhibitor (TFPI), a natural anticoagulation protein that functions to prevent the formation of blood clots and restore hemostasis.

由辉瑞公司的科学家发现，HYMPAVZI是一种再平衡剂，靶向组织因子途径抑制剂（TFPI）的Kunitz 2结构域，TFPI是一种天然抗凝蛋白，可防止血栓形成并恢复止血。

HYMPAVZI is approved by the EC for the routine prophylaxis of bleeding episodes in patients aged 12 years and older weighing at least 35 kg with severe hemophilia A (congenital factor VIII [FVIII] deficiency, FVIII <1%) without FVIII inhibitors or severe hemophilia B (congenital factor IX [FIX] deficiency, FIX <1%) without FIX inhibitors..

HYMPAVZI被EC批准用于常规预防12岁及以上体重至少35公斤的严重血友病A（先天性VIII因子缺乏症，FVIII<1%）无FVIII抑制剂或严重血友病B（先天性IX因子缺乏症，FIX<1%）无FIX抑制剂的患者的出血发作。。

About the BASIS study

关于基础研究

The pivotal BASIS study is a global Phase 3, open-label, multicenter study to evaluate the efficacy and safety of HYMPAVZI in adolescent and adult participants ages 12 to <75 years with severe hemophilia A (defined as FVIII <1%) or moderately severe to severe hemophilia B (defined as FIX activity ≤2%) with or without inhibitors..

关键基础研究是一项全球3期开放标签多中心研究，旨在评估HYMPAVZI在12至75岁的青少年和成人参与者中的疗效和安全性，这些参与者患有严重血友病a（定义为FVIII<1%）或中度重度至重度血友病B（定义为FIX活性≤2%），有或没有抑制剂。。

The marketing authorization is based on data from 116 people living with severe hemophilia without inhibitors who were treated with marstacimab during a 12-month active treatment period (ATP) versus a RP regimen with FVIII or FIX, administered as part of usual care in a 6-month observational period.

上市授权基于116名无抑制剂的严重血友病患者的数据，这些患者在12个月的积极治疗期（ATP）内接受了marstacimab治疗，而在6个月的观察期内，作为常规护理的一部分，接受了FVIII或FIX的RP方案。

During the ATP, participants received prophylaxis (a 300 mg subcutaneous loading dose of marstacimab, followed by 150 mg subcutaneously once weekly) with potential for dose escalation to 300 mg once weekly in patients weighing ≥ 50 kg when control of bleeding events is judged to be inadequate by the healthcare professional..

在ATP期间，参与者接受了预防（300毫克皮下负荷剂量的马司他单抗，然后每周一次皮下注射150毫克），当医疗保健专业人员判断出血事件控制不充分时，体重≥50公斤的患者可能每周一次剂量增加到300毫克。。

HYMPAVZI reduced the ABR for treated bleeds by 35% after a 12-month ATP compared to RP treatment in patients with hemophilia A or B without inhibitors. In an interim analysis of the long-term extension study, a consistent reduction in mean ABR for treated bleeds of 2.79 (95% CI 1.90-4.09) was observed in up to an additional 16 months of follow-up (n=87).

与没有抑制剂的血友病a或B患者的RP治疗相比，HYMPAVZI在12个月的ATP治疗后，治疗出血的ABR降低了35%。在长期延长研究的中期分析中，在另外16个月的随访中（n=87），观察到治疗出血的平均ABR持续降低2.79（95%CI 1.90-4.09）。

HYMPAVZI demonstrated non-inferiority across all bleeding-related secondary endpoints: spontaneous bleeds, joint bleeds, target joint bleeds, and total bleeds..

HYMPAVZI在所有与出血相关的次要终点均表现出非劣效性：自发性出血，关节出血，目标关节出血和总出血。。

The safety profile for HYMPAVZI was consistent with Phase 1/2 results and treatment was generally well-tolerated. The most commonly reported adverse events were injection site reactions, headache, pruritus, and hypertension.

HYMPAVZI的安全性与1/2期结果一致，治疗通常耐受性良好。最常见的不良事件是注射部位反应，头痛，瘙痒和高血压。

The inhibitor cohort of the BASIS study is ongoing, with results expected in the third quarter of 2025. Pfizer is also conducting BASIS KIDS, an open-label study investigating the safety and efficacy of marstacimab in children 1 to <18 years of age with severe hemophilia A or moderately severe to severe hemophilia B with or without inhibitors..

BASIS研究的抑制剂队列正在进行中，预计2025年第三季度会有结果。辉瑞公司还正在进行BASIS KIDS，这是一项开放标签研究，旨在研究马司他单抗在1至18岁患有严重A型血友病或中度重度至重度B型血友病的儿童中的安全性和有效性，无论是否使用抑制剂。。

About Hemophilia

关于血友病

Hemophilia is a family of rare genetic blood diseases caused by a clotting factor deficiency (FVIII in hemophilia A, FIX in hemophilia B), which prevents normal blood clotting. Hemophilia is diagnosed in early childhood and impacts more than 800,000 people worldwide.1 The inability of the blood to clot properly can increase the risk of painful bleeding inside the joints, which can cause joint scarring and damage.

血友病是由凝血因子缺乏症（血友病a中的FVIII，血友病B中的FIX）引起的罕见遗传性血液疾病家族，其阻止正常血液凝固。血友病在儿童早期就被诊断出来，影响着全世界80多万人。1血液无法正确凝结会增加关节内痛苦出血的风险，从而导致关节瘢痕形成和损伤。

People living with hemophilia can suffer permanent joint damage following repeated bleeding episodes.2,3.

血友病患者在反复出血后可能会遭受永久性关节损伤。2,3。

For decades, the most common treatment approach for hemophilia A and B has been factor replacement therapy, which replaces the missing clotting factors. Factor replacement therapies increase the amount of clotting factor in the body to levels that improve clotting, resulting in less bleeding.5,6

几十年来，血友病A和B最常见的治疗方法是因子替代疗法，它替代了缺失的凝血因子。因子替代疗法可将体内凝血因子的含量增加到改善凝血的水平，从而减少出血。5,6

The burden of intravenous infusions is believed to be a barrier to treatment adherence for some people living with hemophilia due in part to inconvenience, time constraints, and poor venous access.7,8,9,10 In a patient/physician/specialist nurse survey across six European countries, lack of time for treatment and convenience were among the leading reasons for not using the prescribed amount of clotting factor or skipping treatment administration.7.

静脉输注的负担被认为是一些血友病患者治疗依从性的障碍，部分原因是不便，时间限制和静脉通路不良[7,8,9,10]。在六个欧洲国家的患者/医生/专科护士调查中，缺乏治疗时间和便利性是不使用规定量的凝血因子或跳过治疗管理的主要原因。

About Pfizer: Breakthroughs That Change Patients’ Lives

关于辉瑞：改变患者生活的突破

At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines.

在辉瑞，我们运用科学和全球资源为人们带来治疗方法，延长并显着改善他们的生活。我们努力为包括创新药物和疫苗在内的保健产品的发现、开发和制造制定质量、安全和价值标准。

Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world.

辉瑞公司的同事们每天都在发达市场和新兴市场开展工作，促进健康、预防、治疗和治愈，挑战我们这个时代最可怕的疾病。作为世界首屈一指的创新生物制药公司之一，我们与医疗保健提供者、政府和当地社区合作，支持和扩大全球可靠、负担得起的医疗保健服务。

For 175 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at www.Pfizer.com. In addition, to learn more, please visit us on www.Pfizer.com and follow us on X at @Pfizer and @Pfizer_News, LinkedIn, YouTube and like us on Facebook at www.facebook.com/Pfizer/..

175年来，我们一直致力于为所有依赖我们的人带来改变。我们经常在我们的网站www.Pfizer.com上发布对投资者可能很重要的信息。此外，要了解更多信息，请访问我们的网站www.Pfizer.com，并在X上关注我们@Pfizer和@Pfizer\u News，LinkedIn，YouTube，并在Facebook上关注我们，网址为www.Facebook.com/Pfizer/。。