FDA授予HYMPAVZI®（marstacimab）sBLA优先审查，用于治疗两个具有显著医疗需求的血友病A或B患者群体-动脉网

FDA Grants Priority Review for HYMPAVZI® (marstacimab) sBLA for the Treatment of Two Hemophilia A or B Patient Populations with Significant Medical Need

辉瑞等信源发布 2026-02-06 20:34

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可切换为仅中文

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Submission is to expand HYMPAVZI indication to the treatment of hemophilia A or B patients 6 years and older with inhibitors, and pediatric patients (ages 6 to 11) without inhibitors

提交的目的是将HYMPAVZI的适应症扩展到治疗6岁及以上的伴有抑制剂的A型或B型血友病患者，以及不伴有抑制剂的儿科患者（6至11岁）。

If approved, HYMPAVZI would become the first non-factor prophylactic treatment available for children aged 6 to 11 years with hemophilia B

如果获得批准，HYMPAVZI 将成为首个适用于 6 至 11 岁血友病 B 患儿的非因子预防性治疗药物。

NEW YORK--(BUSINESS WIRE)--

纽约--（商业资讯）--

Pfizer Inc. (NYSE: PFE)

辉瑞公司（纽约证券交易所代码：PFE）

today announced that the U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review for the company’s supplemental Biologics License Application (sBLA) for HYMPAVZI

今天宣布，美国食品药品监督管理局（FDA）已接受并授予该公司关于HYMPAVZI的补充生物制品许可申请（sBLA）优先审查资格。

(marstacimab) to expand the approved indication to include the treatment of hemophilia A or B patients 6 years and older with inhibitors, and pediatric patients (ages 6 to 11) with hemophilia A or B without inhibitors. In the U.S., HYMPAVZI is currently approved for the treatment of patients 12 years of age and older with hemophilia A without factor VIII (FVIII) inhibitors, or hemophilia B without factor IX (FIX) inhibitors..

将（marstacimab）的批准适应症扩大到包括治疗6岁及以上伴有抑制物的血友病A或B患者，以及6至11岁不伴有抑制物的血友病A或B儿科患者。在美国，HYMPAVZI目前被批准用于治疗12岁及以上不伴有凝血因子VIII（FVIII）抑制物的血友病A患者，或不伴有凝血因子IX（FIX）抑制物的血友病B患者。

The FDA has set a Prescription Drug User Fee Act (PDUFA) action date in the second quarter of 2026. If approved, HYMPAVZI would offer a combination of bleed protection with a straightforward, once-weekly subcutaneous injection administration, requiring minimal preparation and no routine treatment-related lab monitoring for these difficult-to-treat patient populations..

美国食品药品监督管理局（FDA）已将《处方药使用者费用法案》（PDUFA）的行动日期定在2026年第二季度。如果获得批准，HYMPAVZI将提供一种结合出血保护的治疗方案，通过每周一次的皮下注射给药，操作简单，几乎无需准备，且对于这些难以治疗的患者群体，不需要常规的治疗相关实验室监测。

“There is a significant medical need for younger patients with hemophilia and for those who have developed inhibitors, which neutralize factor replacement therapies and render them ineffective,” said Michael Vincent, M.D., Ph.D., Chief Inflammation & Immunology Officer, Pfizer. “Based on the findings in the BASIS clinical trial program and if approved, we believe HYMPAVZI has the potential to become a transformative option for these patients that have limited or burdensome treatment options today.

“年轻的血友病患者以及那些已产生抑制剂的患者存在显著的医疗需求，这些抑制剂会中和因子替代疗法并使其失效，”辉瑞公司首席炎症与免疫官迈克尔·文森特博士表示。“基于BASIS临床试验项目的结果，如果获得批准，我们相信HYMPAVZI有潜力成为这些目前治疗选择有限或负担沉重的患者的变革性选择。”

We look forward to progressing discussions with regulators to make this medicine available for patients.”.

我们期待与监管机构进一步讨论，以使患者能够获得这种药物。

Hemophilia is diagnosed in early childhood and impacts more than 800,000 people worldwide.

血友病在儿童早期被诊断出来，影响着全球超过80万人。

The inability of the blood to clot properly can increase the risk of painful bleeding, including inside the joints, which can cause joint scarring and damage.

血液无法正常凝结会增加痛苦出血的风险，包括关节内部出血，这可能导致关节瘢痕和损伤。

2,3

Children’s joints have growing cartilage and bone, which makes them particularly susceptible to damage caused by repeated bleeding episodes.

儿童的关节有生长中的软骨和骨骼，这使得他们特别容易受到反复出血事件造成的损害。

Inhibitors, or antibodies, develop in approximately 20% of people with hemophilia A and 3% of people with hemophilia B. People living with inhibitors to FVIII and FIX are unable to continue taking factor replacement therapies as they no longer prevent or stop bleeding episodes, particularly in individuals who are refractory to immune tolerance induction therapy..

在大约 20% 的 A 型血友病患者和 3% 的 B 型血友病患者中会产生抑制剂或抗体。对于 FVIII 和 FIX 有抑制剂的患者，他们无法继续接受凝血因子替代疗法，因为这些疗法不能再预防或阻止出血事件，尤其是对免疫耐受诱导治疗无反应的个体。

1,5,6

1，5，6

“For children living with hemophilia A or B between ages 6 and 11, treatment approaches that prevent bleeds are particularly important to protect growing joints,” said Guy Young, M.D., Director, Hemostasis and Thrombosis Center at Children's Hospital, Los Angeles. “HYMPAVZI would address a critical unmet medical need for these patients and those with inhibitors if approved, particularly patients ages 6 to 11 with hemophilia B who do not have non-factor treatment options available today.”.

“对于6至11岁之间患有A型或B型血友病的儿童，预防出血的治疗方案对保护正在生长的关节尤为重要，”洛杉矶儿童医院止血与血栓中心主任Guy Young博士表示。“如果获得批准，HYMPAVZI将满足这些患者以及伴有抑制物患者的迫切未满足医疗需求，特别是目前没有非因子治疗选择的6至11岁B型血友病患者。”

The FDA grants Priority Review to medicines that may offer significant advances in treatment or may provide a treatment where no adequate therapy exists. Priority Review designation by the FDA shortens the standard sBLA review period by four months.

FDA授予优先审查资格给那些可能在治疗方面提供重大进展或在没有适当疗法的情况下提供治疗的药物。FDA的优先审查资格将标准的sBLA审查期缩短了四个月。

The FDA also granted HYMPAVZI Breakthrough Therapy Designation for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in younger pediatric (≥6 to <12 years of age) patients with hemophilia B with and without inhibitors. The FDA’s Breakthrough Therapy Designation is intended to expedite the development and review of medicines with the potential to treat a serious or life-threatening disease, when preliminary clinical evidence indicates the medicine may demonstrate substantial improvement over existing therapies..

美国食品药品监督管理局（FDA）还授予HYMPAVZI突破性疗法认定，用于预防或减少患有B型血友病（无论是否伴有抑制物）的较年轻儿科患者（≥6至<12岁）出血事件的常规预防治疗。FDA的突破性疗法认定旨在加速开发和审查那些在初步临床证据显示可能比现有疗法有显著改进、用于治疗严重或危及生命疾病的药物。

The submission for HYMPAVZI in adults and adolescents is based on efficacy and safety data from the inhibitor cohort of the Phase 3 BASIS trial (

HYMPAVZI 在成人和青少年中的提交基于第 3 阶段 BASIS 试验的抑制剂队列的有效性和安全性数据 (

NCT03938792

). The submission for HYMPAVZI in children aged 6 to 11 years with or without inhibitors is supported by efficacy and safety data from the Phase 3 BASIS KIDS trial (

). 在6至11岁有或没有抑制剂的儿童中，HYMPAVZI的提交得到了3期BASIS KIDS试验的有效性和安全性数据的支持 (

NCT05611801

）。

The use of HYMPAVZI for the treatment of patients 12 years and older living with hemophilia A or B with inhibitors is also under review by the European Medicines Agency.

欧洲药品管理局也在审查使用HYMPAVZI治疗12岁及以上患有A型或B型血友病伴抑制剂的患者。

About HYMPAVZI

关于HYMPAVZI

Discovered by Pfizer scientists, HYMPAVZI has a unique mechanism of action that is differentiated from FVIII and FIX replacement treatments. Instead of replacing missing or insufficient clotting factors, HYMPAVZI is intentionally designed to target tissue factor pathway inhibitor (TFPI), one of the body’s natural mechanisms that inhibits the initiation of blood clotting.

辉瑞公司的科学家们发现，HYMPAVZI 具有一种独特的作用机制，与FVIII和FIX替代疗法有所不同。HYMPAVZI 并非替代缺失或不足的凝血因子，而是有意设计用于靶向组织因子途径抑制剂 (TFPI)，这是人体内抑制凝血启动的一种天然机制。

By targeting the Kunitz 2 domain of TFPI, HYMPAVZI may help re-establish balance between bleeding and blood clot formation with the goal of offering a combination of bleed protection and straightforward administration..

通过靶向TFPI的Kunitz 2域，HYMPAVZI可能有助于重新建立出血和血栓形成之间的平衡，目标是提供出血保护和简单管理的组合。

HYMPAVZI is a hemophilia treatment that has received regulatory approvals in more than 40 countries for eligible patients living with hemophilia A without factor VIII inhibitors, or hemophilia B without factor IX inhibitors. HYMPAVZI was the first anti-TFPI approved in the U.S. and EU for the treatment of hemophilia A or B and the first hemophilia medicine approved in the U.S.

HYMPAVZI 是一种血友病治疗药物，已在 40 多个国家获得监管批准，适用于不伴有 VIII 因子抑制物的 A 型血友病患者或不伴有 IX 因子抑制物的 B 型血友病患者。HYMPAVZI 是美国和欧盟批准的首个抗 TFPI 药物，用于治疗 A 型或 B 型血友病，同时也是美国批准的首个血友病药物。

and EU to be administered via a pre-filled, auto-injector pen. For eligible people living with hemophilia B, it is the first once-weekly subcutaneous prophylactic treatment. HYMPAVZI is a subcutaneous treatment option with a once-weekly dosing schedule and minimal preparation required for each individual administration..

并通过预填充的自动注射笔进行管理。对于符合资格的B型血友病患者，这是首个每周一次的皮下预防治疗方案。HYMPAVZI是一种每周一次的皮下治疗选择，每次使用所需的准备工作最少。

About the BASIS Clinical Trial

关于BASIS临床试验

The pivotal BASIS study is a global, Phase 3, open-label, multicenter study to evaluate the efficacy data and safety profile 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.

BASIS 关键研究是一项全球性、III 期、开放标签、多中心研究，旨在评估 HYMPAVZI 在 12 至 <75 岁的重度 A 型血友病（定义为 FVIII <1%）或中重度至重度 B 型血友病（定义为 FIX 活性 ≤2%）青少年和成人患者中的疗效数据和安全性，无论是否伴有抑制物。

The with inhibitor cohort included 48 people living with hemophilia with inhibitors who were treated with HYMPAVZI during a 12-month active treatment period (ATP) versus an on-demand intravenous regimen with bypassing agents, administered as part of usual care in a six-month observational period. During the ATP, participants received prophylaxis (a 300 mg subcutaneous loading dose of HYMPAVZI, followed by 150 mg subcutaneously once weekly) with potential for dose escalation to 300 mg once weekly.

伴有抑制剂的队列包括48名在12个月的积极治疗期（ATP）内接受HYMPAVZI治疗的血友病伴抑制剂患者，对照组为按需静脉注射旁路制剂，作为常规护理的一部分，在六个月的观察期内进行。在ATP期间，参与者接受了预防性治疗（300毫克皮下负荷剂量的HYMPAVZI，随后每周一次150毫克皮下注射），并有可能将剂量增加至每周一次300毫克。

An additional three patients in the inhibitor cohort were on routine prophylactic treatment prior to the study and not included in the primary efficacy analysis. The primary endpoint measures the treated ABR (annualized bleeding rate) during the 12-month ATP with HYMPAVZI compared to treated ABR on prior on-demand bypass therapy.

在抑制剂队列中，另有三名患者在研究开始前已接受常规预防治疗，因此未纳入主要疗效分析。主要终点是比较HYMPAVZI在12个月治疗期间的年化出血率（ABR）与之前按需旁路治疗的年化出血率。

For further information, visit .

欲了解更多信息，请访问。

clinicaltrials.gov

临床试验.gov

。

About the BASIS KIDS Clinical Trial

关于BASIS KIDS临床试验

The BASIS KIDS study is a global, Phase 3, open-label study investigating the safety and efficacy of HYMPAVZI in children 1 to <18 years of age with severe hemophilia A or moderately severe to severe hemophilia B with or without inhibitors. There were 68 patients aged 6 to 11 years treated with HYMPAVZI during a 12-month ATP versus routine prophylaxis with factor replacement therapy (without inhibitor), or routine prophylaxis or on-demand treatment with bypassing agents (with inhibitor), administered as part of usual care in a 12-month period prior to enrollment.

BASIS KIDS研究是一项全球性、III期、开放标签研究，旨在调查HYMPAVZI在1至未满18岁患有重度A型血友病或中重度至重度B型血友病（无论是否伴有抑制物）的儿童中的安全性和有效性。研究中有68名6至11岁的患者在12个月的ATP治疗期内接受了HYMPAVZI治疗，与之对比的是常规因子替代疗法预防治疗（无抑制物），或使用旁路制剂进行常规预防或按需治疗（有抑制物），这些对照治疗是在入组前12个月内作为常规护理的一部分进行的。

During the ATP in this age group, participants received prophylaxis (a 150 mg subcutaneous loading dose of HYMPAVZI, followed by 75 mg subcutaneous once weekly) with the potential for dose escalation to 150 mg once weekly. The primary endpoint measures treated ABR during the 12-month ATP with HYMPAVZI compared to ABR on prior routine prophylaxis with factor replacement therapy, or routine prophylaxis or on-demand treatment with bypassing agents.

在此年龄段的ATP期间，参与者接受了预防治疗（150毫克皮下负荷剂量的HYMPAVZI，随后每周一次75毫克皮下注射），有可能剂量递增至每周一次150毫克。主要终点指标是比较使用HYMPAVZI在12个月ATP期间的治疗后ABR与之前常规因子替代疗法预防治疗、常规预防治疗或按需使用旁路制剂的ABR。

For further information, visit .

欲了解更多信息，请访问。

clinicaltrials.gov

临床试验.gov

。

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.

血友病是一组罕见的遗传性血液疾病，由凝血因子缺乏（A型血友病中的FVIII，B型血友病中的FIX）引起，导致无法正常凝血。血友病在儿童早期即可诊断，全球有超过80万人受到影响。

The inability of the blood to clot properly can increase the risk of painful bleeding, including inside the joints, which can cause joint scarring and damage. People living with hemophilia can suffer permanent joint damage following repeated bleeding episodes.

血液无法正常凝固会增加痛苦出血的风险，包括关节内出血，这可能导致关节瘢痕和损伤。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

Children’s joints have growing cartilage and bone, which makes them particularly susceptible to damage caused by repeated bleeding episodes.

儿童的关节有生长中的软骨和骨骼，这使得他们特别容易受到反复出血事件造成的损害。

For decades, the most common treatment approach for hemophilia A and B has been factor replacement therapy, which replaces the missing clotting factors.

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，该疗法补充了缺失的凝血因子。

2,7

Factor replacement therapies increase the amount of clotting factor in the body to levels that improve clotting, resulting in less bleeding.

因子替代疗法增加体内凝血因子的含量，使其达到改善凝血的水平，从而减少出血。

2,3

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.

静脉输液的负担被认为是部分血友病患者治疗依从性的障碍，这部分是由于不便、时间限制和静脉通路不佳所致。

8,9

Approximately 20% of people with hemophilia A and 3% of people with hemophilia B are unable to continue taking factor replacement therapies because they develop inhibitors to FVIII and FIX, respectively.

大约20%的A型血友病患者和3%的B型血友病患者因为分别对FVIII和FIX产生抑制剂而无法继续接受因子替代疗法。

1,6,7

These patients often have higher treatment burden, including potential complications from bleeding such as hospitalization and death, as well as higher treatment-related costs.

这些患者通常面临更高的治疗负担，包括因出血可能导致的并发症（如住院和死亡）以及更高的治疗相关费用。

10,11,12

10，11，12

HYMPAVZI (marstacimab-hncq) U.S. Important Safety Information

HYMPAVZI（马斯塔西单抗-hncq）美国重要安全信息

Important: Before you start using HYMPAVZI, it is very important to talk to your healthcare provider about using factor VIII and factor IX products

重要提示：在您开始使用HYMPAVZI之前，非常有必要与您的医疗保健提供者讨论使用凝血因子VIII和凝血因子IX产品的事宜。

(products that help blood clot but work in a different way than HYMPAVZI). You may need to use factor VIII or factor IX medicines to treat episodes of breakthrough bleeding during treatment with HYMPAVZI. Carefully follow your healthcare provider’s instructions regarding when to use factor VIII or factor IX medicines and the prescribed dose during your treatment with HYMPAVZI..

（帮助血液凝固但作用方式与HYMPAVZI不同的产品）。在使用HYMPAVZI治疗期间，您可能需要使用因子VIII或因子IX药物来治疗突破性出血。请仔细遵循您的医疗保健提供者关于何时使用因子VIII或因子IX药物以及在使用HYMPAVZI治疗期间的处方剂量的指示。

Before using HYMPAVZI, tell your healthcare provider about all of your medical conditions, including if you:

在使用HYMPAVZI之前，请告诉您的医疗保健提供者您的所有健康状况，包括您是否：

have a planned surgery. Your healthcare provider may stop treatment with HYMPAVZI before your surgery. Talk to your healthcare provider about when to stop using HYMPAVZI and when to start it again if you have a planned surgery.

计划进行手术。您的医疗保健提供者可能会在手术前停止使用HYMPAVZI的治疗。如果您计划进行手术，请与您的医疗保健提供者讨论何时停止使用HYMPAVZI以及何时重新开始使用。

have a severe short-term (acute) illness such as an infection or injury.

患有严重的短期（急性）疾病，如感染或受伤。

have been told that you have a risk for blood clots.

被告知有血栓风险。

are pregnant or plan to become pregnant. HYMPAVZI may harm your unborn baby.

怀孕或计划怀孕。HYMPAVZI可能会伤害您未出生的宝宝。

Females who are able to become pregnant:

能够怀孕的女性：

Your healthcare provider will do a pregnancy test before you start your treatment with HYMPAVZI.

在您开始使用HYMPAVZI治疗之前，您的医疗保健提供者将为您进行妊娠测试。

You should use effective birth control (contraception) during treatment with HYMPAVZI and for at least 2 months after the last dose of HYMPAVZI.

您应在使用 HYMPAVZI 治疗期间以及最后一次服用 HYMPAVZI 后至少两个月内使用有效的避孕措施。

Tell your healthcare provider right away if you become pregnant or think that you may be pregnant during treatment with HYMPAVZI.

如果您在使用HYMPAVZI治疗期间怀孕或认为自己可能怀孕，请立即告知您的医疗保健提供者。

are breastfeeding or plan to breastfeed. It is not known if HYMPAVZI passes into your breast milk.

正在哺乳或计划哺乳。尚不清楚HYMPAVZI是否会进入您的乳汁中。

Tell your healthcare provider about all the medicines you take,

告诉您的医疗保健提供者您所服用的所有药物，

including prescription medicines, over-the-counter medicines, vitamins, and herbal supplements.

包括处方药、非处方药、维生素和草本补充剂。

What are the possible side effects of HYMPAVZI?

HYMPAVZI可能有哪些副作用？

HYMPAVZI may cause serious side effects, including:

HYMPAVZI 可能导致严重的副作用，包括：

blood clots (thromboembolic events).

血栓（血栓栓塞事件）。

HYMPAVZI may increase the risk for your blood to clot in blood vessels in your arm, leg, lung, or head which can be life-threatening. Blood clots have happened in people using HYMPAVZI. You may have an increased risk of blood clots if you have certain risk factors. Stop using HYMPAVZI and get medical help right away if you develop any of these signs or symptoms of blood clots:.

HYMPAVZI可能会增加您的血液在手臂、腿部、肺部或头部的血管中凝结的风险，这可能危及生命。使用HYMPAVZI的人群中已出现过血栓。如果您有某些风险因素，您可能面临更高的血栓风险。如果您出现以下任何血栓的迹象或症状，请立即停止使用HYMPAVZI并寻求医疗帮助：。

swelling or pain in arms or legs

手臂或腿部肿胀或疼痛

redness or discoloration in your arms or legs

手臂或腿部的发红或变色

shortness of breath

呼吸急促

pain in chest or upper back

胸部或上背部疼痛

fast heart rate

心率快

cough up blood

咳血

feel faint

感觉头晕

headache

头痛

numbness in your face

脸部麻木

eye pain or swelling

眼睛疼痛或肿胀

trouble seeing

视力有问题

allergic reactions.

过敏反应。

HYMPAVZI may cause allergic reactions, including rash and itching. Stop using HYMPAVZI and get medical help right away if you develop any of the following symptoms of a severe allergic reaction:

HYMPAVZI 可能会引起过敏反应，包括皮疹和瘙痒。如果出现以下任何严重过敏反应的症状，请立即停止使用 HYMPAVZI 并寻求医疗帮助：

swelling of your face, lips, mouth, or tongue

脸部、嘴唇、口腔或舌头肿胀

trouble breathing

呼吸困难

wheezing

喘息

dizziness or fainting

头晕或昏厥

fast heartbeat or pounding in your chest

心跳加快或胸口怦怦跳动

sweating

出汗

The most common side effects of HYMPAVZI include:

HYMPAVZI最常见的副作用包括：

swelling, hardening, redness, bruising, and pain at injection site

注射部位肿胀、硬化、发红、淤青和疼痛

headache

头痛

itching

瘙痒

These are not all the possible side effects of HYMPAVZI. Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.

这些并非 HYMPAVZI 的所有可能副作用。如需了解有关副作用的医疗建议，请咨询您的医生。您可以拨打 1-800-FDA-1088 向 FDA 报告副作用。

The full Prescribing Information can be found

完整的处方信息可以找到

here

这里

。

About Pfizer: Breakthroughs That Change Patients’ Lives

关于辉瑞：改变患者生活的突破性进展

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，为人们带来能够延长生命并显著改善生活的疗法。我们努力在医疗保健产品的发现、开发和生产过程中，为质量、安全性和价值树立标杆，包括创新药物和疫苗。

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.

每天，辉瑞的同事们都在发达市场和新兴市场开展工作，以促进健康、预防、治疗和攻克当今最令人畏惧的疾病。作为全球首屈一指的创新型生物制药公司之一，我们与医疗保健提供者、政府和当地社区合作，支持并扩大全球范围内可靠且负担得起的医疗保健服务的可及性。

For 175 years, we have worked to make a difference for all who rely on us. .

175 年来，我们一直努力为所有依赖我们的人带来改变。

We routinely post information that may be important to investors on our website at

我们经常在我们的网站上发布可能对投资者重要的信息，网址为

www.Pfizer.com

. In addition, to learn more, please visit us on

此外，欲了解更多信息，请访问我们

www.Pfizer.com

and follow us on X at

关注我们在X平台上的账号

@Pfizer

@辉瑞

and

和

@Pfizer_News

@辉瑞新闻

，

领英

，

YouTube

and like us on Facebook at

并在Facebook上关注我们

www.facebook.com/Pfizer/

。

Disclosure notice

披露通知

The information contained in this release is as of February 6, 2026. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.

本新闻稿中的信息截至2026年2月6日。辉瑞不对因新信息或未来事件或发展而更新本新闻稿中包含的前瞻性声明承担任何义务。

This release contains forward-looking information about HYMPAVZI

此版本包含有关HYMPAVZI的前瞻性信息

(marstacimab), an anti-tissue factor pathway inhibitor, including its potential benefits and submission to regulatory authorities of the Phase 3 BASIS data for HYMPAVZI for the treatment of adults and adolescents living with hemophilia A or B with inhibitors and the Phase 3 BASIS KIDS data for HYMPAVZI for the treatment of children aged 6 to 11 years with or without inhibitors, that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements.

(marstacimab)，一种抗组织因子途径抑制剂，包括其潜在益处以及向监管机构提交的三期BASIS数据，用于治疗伴有抑制物的血友病A或B成人和青少年患者，以及HYMPAVZI的三期BASIS KIDS数据，用于治疗6至11岁有或没有抑制物的儿童患者，这些数据涉及重大风险和不确定性，可能导致实际结果与这些声明所表达或暗示的结果有实质性差异。

Risks and uncertainties include, among other things, uncertainties regarding the commercial success of HYMPAVZI; the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for our clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; whether and when applications may be filed with regulatory authorities in particular jurisdictions for HYMPAVZI for any potential indication; whether and when any such applications that may be pending or filed for HYMPAVZI (including applications submitted to the FDA and EMA for adults and adolescents living with hemophilia A or B with inhibitors and to the FDA for children aged 6 to 11 years with or without inhibitors) may be approved by regulatory authorities, which will depend on myriad factors, including making a determination as to whether the produc.

风险和不确定性包括（除其他事项外）：关于HYMPAVZI商业成功的不确定性；研发过程中的固有不确定性，包括是否能够达到预期的临床终点、临床试验的启动和/或完成日期、监管提交日期、监管批准日期和/或上市日期，以及可能出现不利的新临床数据并对现有临床数据进行进一步分析的可能性；临床试验数据可能因监管机构而有不同的解释和评估的风险；监管机构是否会对我们的临床研究设计和结果感到满意；是否以及何时会在特定司法管辖区为HYMPAVZI的任何潜在适应症向监管机构提交申请；是否以及何时会批准任何待审或已提交的HYMPAVZI申请（包括针对患有血友病A或B且具有抑制剂的成年和青少年患者提交给FDA和EMA的申请，以及针对6至11岁有或没有抑制剂的儿童提交给FDA的申请），这将取决于众多因素，包括确定产品是否...

A further description of risks and uncertainties can be found in Pfizer’s Annual Report on Form 10-K for the fiscal year ended December 31, 2024 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned “Risk Factors” and “Forward-Looking Information and Factors That May Affect Future Results”, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S.

辉瑞公司在截至2024年12月31日的财政年度的Form 10-K年报及其后续的Form 10-Q报告中提供了风险和不确定性的进一步描述，特别是其中标题为“风险因素”和“前瞻性信息及可能影响未来结果的因素”的部分，以及其后续提交的Form 8-K报告中也有相关说明，所有这些文件均已提交给美国证券交易委员会。

Securities and Exchange Commission and available at .

证券交易委员会，可访问。

www.sec.gov

and

和

www.pfizer.com

。

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参考文献

__________________________

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https://www1.wfh.org/publications/files/pdf-2588.pdf

。

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。

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努金特 D，卡尔尼恩斯 W，奎罗尔 F，等。血友病经历、结果与机遇 (HERO) 研究：与治疗相关的人群特征。

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. 2001;7(2):198-206. doi:

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