凝血因子缺乏症市场 - 全球及区域分析：按缺乏症类型、按产品类型、按最终用户、按区域 - 分析与预测（2025-2035 年）

凝血因子缺乏症是一种血液凝固障碍，其中正常血液凝固所需的一种或多种蛋白质（凝血因子）缺失或无法正常发挥作用。

结果，血液无法有效形成血块，导致出血过多或受伤或手术后止血困难。最常见的凝血因子缺乏症包括A型血友病 A（凝血因子 VIII 缺乏）、B型血友病（凝血因子 IX 缺乏）和血管性血友病（血管性血友病因子缺乏或功能障碍）。凝血因子缺乏症可以遗传，而A型血友病 A和 B 等疾病是 X 连锁隐性遗传，因此在男性中更为常见。然而，一些因子缺乏是由于影响血液凝结能力的疾病（例如肝病和某些自体免疫疾病）而获得的。这些因子缺乏症通常需要持续使用凝血因子浓缩物或基因疗法治疗，以预防或控制出血发作并有效管理病情。

凝血因子缺乏症市场的主要驱动力之一是治疗方法的进步，尤其是长效重组凝血因子和基因疗法的开发。这些创新比通常需要频繁输注凝血因子的传统治疗方法有了显着的改善。例如，长效重组凝血因子减少了定期输注的需要，使患者治疗方法更加方便，并提高了他们的生活品质。此外，基因疗法旨在透过解决A型血友病 A和B型血友病等疾病背后的基因缺陷来提供潜在的治癒方法，作为突破性解决方案正在获得关注。随着基因疗法在临床试验中不断显示出积极成果，预计它们将具有长期甚至治癒效果，进一步推动对凝血因子缺乏症治疗的需求。这些进步为患者提供了更有效、个人化和负担更轻的治疗选择，从而促进了市场成长的扩大。

儘管凝血因子缺乏症市场正在成长，但仍存在一些挑战阻碍其充分发挥潜力。高昂的治疗费用是一个重大挑战，特别是对于基因治疗和长效重组凝血因子製剂等先进治疗方法而言。儘管这些治疗方法很有前景，但其高昂的前期成本可能成为许多患者，特别是中低收入国家患者获得治疗的障碍。高昂的治疗费用不仅限制了个别患者的经济负担，也给医疗保健系统带来了压力，使政府和保险公司难以提供这些最先进治疗的广泛可及性。因此，患者的治疗负担仍然很高，获得挽救生命的治疗的机会仍然不平等，儘管有创新治疗方法，但这仍然限制了市场的成长潜力。

全球凝血因子缺乏症市场竞争激烈，多家主要企业正在推动创新和市场成长。罗氏公司和诺和诺德公司等主要企业走在前列，开发长效重组凝血因子等先进治疗方法以及针对A型血友病和B型血友病的基因疗法。以广泛产品组合而闻名的辉瑞公司继续扩大其在该领域的产品供应，而基立福公司在治疗凝血障碍的血浆衍生疗法方面取得了长足进步。此外，Alnylam Pharmaceuticals 和 Sangamo Therapeutics 等公司正在探索基因静默和基因编辑技术等新方法，以提供更有效和个人化的治疗。这些产业领导者持续致力于透过创新治疗方法和策略联盟来改善患者预后，从而增强其在凝血因子缺乏症市场的竞争优势。

市场区隔

细分 1：按缺陷类型

• A型血友病A
• B型血友病
• 血管性血友病
• 因子 XI 和因子 XIII 缺乏症
• 其他缺陷

细分2：依产品类型

• 重组凝血因子
• 血浆来源的凝血因子
• 其他的

细分 3：按最终用户

• 医院
• 专科诊所
• 其他的

细分4：按地区

• 北美洲
• 欧洲
• 亚太地区

全球凝血因子缺乏症市场正见证着几个关键的新兴趋势，这些趋势正在重塑诊断和治疗模式。这种方法正日益受到关注，因为它直接针对导致凝血障碍的基因缺陷，减少了终生使用凝血因子浓缩物治疗的需求。此外，市场正见证长效重组凝血因子的开发，透过减少输注频率，提升病患便利性。另一个新兴趋势是个人化医疗的兴起，个人化医疗根据个别基因图谱客製化治疗方案，为患者提供更有效、更有针对性的治疗。这些创新正在推动凝血因子缺乏症市场走向更有效率、以患者为中心的解决方案，有望改善患者的生活品质和出血性疾病的长期管理。

本报告概述了全球凝血因子缺乏症市场，包括缺乏症类型、产品类型、最终用户和地区的趋势，以及参与市场的公司概况。

目录

执行摘要

第一章全球凝血因子缺乏症市场：产业展望

• 介绍
• 市场趋势
• 法律规范
• 流行病学分析
• 临床试验分析
• 市场动态
  • 影响分析
  • 市场驱动因素
  • 市场挑战
  • 市场机会

2. 全球凝血因子缺乏症市场（依缺乏症类型），2023-2035

• A型血友病A
• B型血友病
• 血管性血友病
• 因子XI缺乏
• 因子 XIII 缺乏症
• 其他的

3. 全球凝血因子缺乏症市场（依产品类型），2023-2035

• 血浆来源的凝血因子
• 重组凝血因子
• 其他的

4. 2023 年至 2035 年全球凝血因子缺乏症市场（依最终使用者划分）

• 医院
• 专科诊所
• 其他的

5. 2023-2035 年全球凝血因子缺乏症市场（按地区）

• 北美洲
• 欧洲
• 亚太地区

6. 全球凝血因子缺乏症市场：竞争格局与公司概况

• 关键策略和发展
  • 合併与收购
  • 协同效应活动
  • 业务扩展和资金筹措
  • 产品发布和核准
  • 其他活动
• 公司简介
  • F. Hoffmann-La Roche Ltd
  • Sanofi SA
  • Grifols, SA
  • Novo Holdings A/S
  • Pfizer Inc.
  • Alnylam Pharmaceuticals
  • Sangamo Therapeutics
  • Centessa Pharmaceuticals
  • Staidson Biopharma Inc.
  • Ultragenyx Pharmaceutical
  • OPKO Health, Inc.
  • Novo Nordisk
  • Takeda Pharmaceutical Company Limited
  • Biogen Inc
  • Octapharma AG

第七章调查方法

Global Coagulation Factor Deficiency Market, Analysis and Forecast: 2025-2035

Coagulation factor deficiency is a blood clotting disorder where one or more of the proteins (clotting factors) necessary for normal blood coagulation are either missing or not functioning properly. This results in the blood's inability to form clots effectively, leading to excessive bleeding or difficulty stopping bleeding after injury or surgery. The most common coagulation factor deficiencies include Hemophilia A (deficiency of Factor VIII), Hemophilia B (deficiency of Factor IX), and Von Willebrand Disease (deficiency or dysfunction of Von Willebrand factor). Coagulation factor deficiencies can be inherited, with conditions such as Hemophilia A and B being X-linked recessive, meaning they are more commonly seen in males. However, some factor deficiencies can also be acquired due to conditions that affect the blood's clotting ability, such as liver disease or certain autoimmune disorders. These deficiencies often require ongoing treatment with clotting factor concentrates or gene therapy to prevent or control bleeding episodes and manage the condition effectively.

One of the key drivers of the coagulation factor deficiency market is the advancement in treatment options, particularly the development of long-acting recombinant clotting factors and gene therapies. These innovations offer significant improvements over traditional treatments, which typically require frequent infusions of clotting factors. Long-acting recombinant clotting factors, for example, reduce the need for regular infusions, making treatment more convenient for patients and improving their quality of life. Additionally, gene therapy, which aims to provide a potential cure by addressing the genetic defects underlying conditions such as Hemophilia A and Hemophilia B, is gaining traction as a groundbreaking solution. As gene therapy continues to show positive results in clinical trials, it promises long-term or even curative benefits, further driving demand for coagulation factor deficiency treatments. These advancements are contributing to increased market growth as they offer patients more effective, personalized, and less burdensome treatment options.

Despite the growth of the coagulation factor deficiency market, several challenges continue to impede its full potential. One major challenge is the high cost of treatment, particularly for advanced therapies such as gene therapy and long-acting recombinant clotting factors. These therapies, while promising, come with significant upfront costs, which can be a barrier to access for many patients, especially in low- and middle-income countries. The high cost not only limits affordability for individual patients but also places a strain on healthcare systems, making it difficult for governments and insurers to provide widespread access to these cutting-edge treatments. As a result, the treatment burden on patients remains substantial, and access to life-saving therapies remains unequal, limiting the market's growth potential despite the availability of innovative treatments.

The global coagulation factor deficiency market is highly competitive, with several key players driving innovation and market growth. Leading companies such as Hoffmann-La Roche Ltd and Novo Nordisk A/S are at the forefront, developing advanced therapies such as long-acting recombinant clotting factors and gene therapies for Hemophilia A and Hemophilia B. Pfizer Inc., known for its broad portfolio, continues to expand its offerings in this space, while Grifols, S.A. is making strides with its plasma-derived therapies for coagulation disorders. Additionally, companies such as Alnylam Pharmaceuticals and Sangamo Therapeutics are exploring novel approaches, including gene silencing and gene editing techniques, to provide more effective and personalized treatments. These industry leaders are continually working to improve patient outcomes through innovative therapies and strategic collaborations, enhancing their competitive edge in the coagulation factor deficiency market.

Market Segmentation:

Segmentation 1: by Deficiency Type

• Hemophilia A
• Hemophilia B
• Von Willebrand Disease
• Factor XI & Factor XIII Deficiencies
• Other Deficiencies

Segmentation 2: by Product Type

• Recombinant Coagulation Factors
• Plasma-derived Coagulation Factors
• Others

Segmentation 3: by End-User

• Hospitals
• Specialty Clinics
• Others

Segmentation 4: by Region

• North America
• Europe
• Asia-Pacific

The global coagulation factor deficiency market is experiencing several key emerging trends that are reshaping the landscape of diagnosis and treatment. One prominent trend is the increasing use of gene therapy, which holds the potential to provide long-term or curative solutions for conditions such as Hemophilia A and Hemophilia B. This approach is gaining traction as it directly addresses the genetic defects responsible for these coagulation disorders, reducing the need for lifelong treatments with clotting factor concentrates. Additionally, the market is seeing a rise in the development of long-acting recombinant clotting factors, which improve patient convenience by reducing the frequency of infusions. Another emerging trend is the advancement of personalized medicine, where treatments are tailored to individual genetic profiles, offering more effective and precise care for patients. With these innovations, the coagulation factor deficiency market is moving toward more efficient, patient-centric solutions that promise to improve both the quality of life and the long-term management of bleeding disorders.

Table of Contents

Executive Summary

Scope and Definition

Market/Product Definition

Inclusion and Exclusion

Key Questions Answered

Analysis and Forecast Note

1. Global Coagulation Factor Deficiency Market: Industry Outlook

• 1.1 Introduction
• 1.2 Market Trends
• 1.3 Regulatory Framework
• 1.4 Epidemiology Analysis
• 1.5 Clinical Trial Analysis
• 1.6 Market Dynamics
  • 1.6.1 Impact Analysis
  • 1.6.2 Market Drivers
  • 1.6.3 Market Challenges
  • 1.6.4 Market Opportunities

2. Global Coagulation Factor Deficiency Market (Deficiency Type), ($Billion), 2023-2035

• 2.1 Hemophilia A
• 2.2 Hemophilia B
• 2.3 Von Willebrand Disease
• 2.4 Factor XI Deficiency
• 2.5 Factor XIII Deficiency
• 2.6 Others

3. Global Coagulation Factor Deficiency Market (Product Type), ($Billion), 2023-2035

• 3.1 Plasma-derived Coagulation Factors
• 3.2 Recombinant Coagulation Factors
• 3.3 Other

4. Global Coagulation Factor Deficiency Market (End-User), ($Billion), 2023-2035

• 4.1 Hospitals
• 4.2 Specialty Clinics
• 4.3 Others

5. Global Coagulation Factor Deficiency Market (Region), ($Billion), 2023-2035

• 5.1 North America
  • 5.1.1 Key Findings
  • 5.1.2 Market Dynamics
  • 5.1.3 Market Sizing and Forecast
    • 5.1.3.1 North America Coagulation Factor Deficiency Market, by Country
      • 5.1.3.1.1 U.S.
• 5.2 Europe
  • 5.2.1 Key Findings
  • 5.2.2 Market Dynamics
  • 5.2.3 Market Sizing and Forecast
    • 5.2.3.1 Europe Coagulation Factor Deficiency Market, by Country
      • 5.2.3.1.1 Germany
      • 5.2.3.1.2 U.K.
      • 5.2.3.1.3 France
      • 5.2.3.1.4 Italy
• 5.3 Asia Pacific
  • 5.3.1 Key Findings
  • 5.3.2 Market Dynamics
  • 5.3.3 Market Sizing and Forecast
    • 5.3.3.1 Asia Pacific Coagulation Factor Deficiency Market, by Country
      • 5.3.3.1.1 China
      • 5.3.3.1.2 Japan

6. Global Coagulation Factor Deficiency Market: Competitive Landscape and Company Profiles

• 6.1 Key Strategies and Development
  • 6.1.1 Mergers and Acquisitions
  • 6.1.2 Synergistic Activities
  • 6.1.3 Business Expansions and Funding
  • 6.1.4 Product Launches and Approvals
  • 6.1.5 Other Activities
• 6.2 Company Profiles
  • 6.2.1 F. Hoffmann-La Roche Ltd
    • 6.2.1.1 Overview
    • 6.2.1.2 Top Products / Product Portfolio
    • 6.2.1.3 Top Competitors
    • 6.2.1.4 Target Customers/End-Users
    • 6.2.1.5 Key Personnel
    • 6.2.1.6 Analyst View
  • 6.2.2 Sanofi S.A.
    • 6.2.2.1 Overview
    • 6.2.2.2 Top Products / Product Portfolio
    • 6.2.2.3 Top Competitors
    • 6.2.2.4 Target Customers/End-Users
    • 6.2.2.5 Key Personnel
    • 6.2.2.6 Analyst View
  • 6.2.3 Grifols, S.A.
    • 6.2.3.1 Overview
    • 6.2.3.2 Top Products / Product Portfolio
    • 6.2.3.3 Top Competitors
    • 6.2.3.4 Target Customers/End-Users
    • 6.2.3.5 Key Personnel
    • 6.2.3.6 Analyst View
  • 6.2.4 Novo Holdings A/S
    • 6.2.4.1 Overview
    • 6.2.4.2 Top Products / Product Portfolio
    • 6.2.4.3 Top Competitors
    • 6.2.4.4 Target Customers/End-Users
    • 6.2.4.5 Key Personnel
    • 6.2.4.6 Analyst View
  • 6.2.5 Pfizer Inc.
    • 6.2.5.1 Overview
    • 6.2.5.2 Top Products / Product Portfolio
    • 6.2.5.3 Top Competitors
    • 6.2.5.4 Target Customers/End-Users
    • 6.2.5.5 Key Personnel
    • 6.2.5.6 Analyst View
  • 6.2.6 Alnylam Pharmaceuticals
    • 6.2.6.1 Overview
    • 6.2.6.2 Top Products / Product Portfolio
    • 6.2.6.3 Top Competitors
    • 6.2.6.4 Target Customers/End-Users
    • 6.2.6.5 Key Personnel
    • 6.2.6.6 Analyst View
  • 6.2.7 Sangamo Therapeutics
    • 6.2.7.1 Overview
    • 6.2.7.2 Top Products / Product Portfolio
    • 6.2.7.3 Top Competitors
    • 6.2.7.4 Target Customers/End-Users
    • 6.2.7.5 Key Personnel
    • 6.2.7.6 Analyst View
  • 6.2.8 Centessa Pharmaceuticals
    • 6.2.8.1 Overview
    • 6.2.8.2 Top Products / Product Portfolio
    • 6.2.8.3 Top Competitors
    • 6.2.8.4 Target Customers/End-Users
    • 6.2.8.5 Key Personnel
    • 6.2.8.6 Analyst View
  • 6.2.9 Staidson Biopharma Inc.
    • 6.2.9.1 Overview
    • 6.2.9.2 Top Products / Product Portfolio
    • 6.2.9.3 Top Competitors
    • 6.2.9.4 Target Customers/End-Users
    • 6.2.9.5 Key Personnel
    • 6.2.9.6 Analyst View
  • 6.2.10 Ultragenyx Pharmaceutical
    • 6.2.10.1 Overview
    • 6.2.10.2 Top Products / Product Portfolio
    • 6.2.10.3 Top Competitors
    • 6.2.10.4 Target Customers/End-Users
    • 6.2.10.5 Key Personnel
    • 6.2.10.6 Analyst View
  • 6.2.11 OPKO Health, Inc.
    • 6.2.11.1 Overview
    • 6.2.11.2 Top Products / Product Portfolio
    • 6.2.11.3 Top Competitors
    • 6.2.11.4 Target Customers/End-Users
    • 6.2.11.5 Key Personnel
    • 6.2.11.6 Analyst View
  • 6.2.12 Novo Nordisk
    • 6.2.12.1 Overview
    • 6.2.12.2 Top Products / Product Portfolio
    • 6.2.12.3 Top Competitors
    • 6.2.12.4 Target Customers/End-Users
    • 6.2.12.5 Key Personnel
    • 6.2.12.6 Analyst View
  • 6.2.13 Takeda Pharmaceutical Company Limited
    • 6.2.13.1 Overview
    • 6.2.13.2 Top Products / Product Portfolio
    • 6.2.13.3 Top Competitors
    • 6.2.13.4 Target Customers/End-Users
    • 6.2.13.5 Key Personnel
    • 6.2.13.6 Analyst View
  • 6.2.14 Biogen Inc
    • 6.2.14.1 Overview
    • 6.2.14.2 Top Products / Product Portfolio
    • 6.2.14.3 Top Competitors
    • 6.2.14.4 Target Customers/End-Users
    • 6.2.14.5 Key Personnel
    • 6.2.14.6 Analyst View
  • 6.2.15 Octapharma AG
    • 6.2.15.1 Overview
    • 6.2.15.2 Top Products / Product Portfolio
    • 6.2.15.3 Top Competitors
    • 6.2.15.4 Target Customers/End-Users
    • 6.2.15.5 Key Personnel
    • 6.2.15.6 Analyst View

7. Research Methodology

List of Figures

• Figure: Global Coagulation Factor Deficiency Market (by Region), $Billion, 2024 and 2035
• Figure: Global Coagulation Factor Deficiency Market Key Trends, Analysis

List of Tables

• Table: Global Coagulation Factor Deficiency Market Dynamics, Impact Analysis
• Table: Global Coagulation Factor Deficiency Market (by Region), $Billion, 2024-2035
• Table: Global Coagulation Factor Deficiency Market (by Deficiency Type), $Billion, 2024-2035
• Table: Global Coagulation Factor Deficiency Market (by Product Type), $Billion, 2024-2035
• Table: Global Coagulation Factor Deficiency Market (by End-User), $Billion, 2024-2035