凝血因子缺乏症市場 - 全球及區域分析：按缺乏症類型、按產品類型、按最終用戶、按區域 - 分析與預測（2025-2035 年）

凝血因子缺乏症是一種血液凝固障礙，其中正常血液凝固所需的一種或多種蛋白質（凝血因子）缺失或無法正常發揮作用。

結果，血液無法有效形成血塊，導致出血過多或受傷或手術後止血困難。最常見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礙）。凝血因子缺乏症可以遺傳，而A型血友病 A和 B 等疾病是 X 連鎖隱性遺傳，因此在男性中更為常見。然而，一些因子缺乏是由於影響血液凝結能力的疾病（例如肝病和某些自體免疫疾病）而獲得的。這些因子缺乏症通常需要持續使用凝血因子濃縮物或基因療法治療，以預防或控制出血發作並有效管理病情。

凝血因子缺乏症市場的主要驅動力之一是治療方法的進步，尤其是長效重組凝血因子和基因療法的開發。這些創新比通常需要頻繁輸注凝血因子的傳統治療方法有了顯著的改善。例如，長效重組凝血因子減少了定期輸注的需要，使患者治療方法更加方便，並提高了他們的生活品質。此外，基因療法旨在透過解決A型血友病 A和B型血友病等疾病背後的基因缺陷來提供潛在的治癒方法，作為突破性解決方案正在獲得關注。隨著基因療法在臨床試驗中不斷顯示出積極成果，預計它們將具有長期甚至治癒效果，進一步推動對凝血因子缺乏症治療的需求。這些進步為患者提供了更有效、個人化和負擔更輕的治療選擇，從而促進了市場成長的擴大。

儘管凝血因子缺乏症市場正在成長，但仍存在一些挑戰阻礙其充分發揮潛力。高昂的治療費用是一個重大挑戰，特別是對於基因治療和長效重組凝血因子製劑等先進治療方法而言。儘管這些治療方法很有前景，但其高昂的前期成本可能成為許多患者，特別是中低收入國家患者獲得治療的障礙。高昂的治療費用不僅限制了個別患者的經濟負擔，也給醫療保健系統帶來了壓力，使政府和保險公司難以提供這些最先進治療的廣泛可及性。因此，患者的治療負擔仍然很高，獲得挽救生命的治療的機會仍然不平等，儘管有創新治療方法，但這仍然限制了市場的成長潛力。

全球凝血因子缺乏症市場競爭激烈，多家主要企業正在推動創新和市場成長。羅氏公司和諾和諾德公司等主要企業走在前列，開發長效重組凝血因子等先進治療方法以及針對A型血友病和B型血友病的基因療法。以廣泛產品組合而聞名的輝瑞公司繼續擴大其在該領域的產品供應，而基立福公司在治療凝血障礙的血漿衍生療法方面取得了長足進步。此外，Alnylam Pharmaceuticals 和 Sangamo Therapeutics 等公司正在探索基因靜默和基因編輯技術等新方法，以提供更有效和個人化的治療。這些產業領導者持續致力於透過創新治療方法和策略聯盟來改善患者預後，從而增強其在凝血因子缺乏症市場的競爭優勢。

市場區隔

細分 1：按缺陷類型

• 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

細分2：依產品類型

• 重組凝血因子
• 血漿來源的凝血因子
• 其他

細分 3：按最終用戶

• 醫院
• 專科診所
• 其他

細分4：按地區

• 北美洲
• 歐洲
• 亞太地區

全球凝血因子缺乏症市場正見證著幾個關鍵的新興趨勢，這些趨勢正在重塑診斷和治療模式。這種方法正日益受到關注，因為它直接針對導致凝血障礙的基因缺陷，減少了終生使用凝血因子濃縮物治療的需求。此外，市場正見證長效重組凝血因子的開發，透過減少輸注頻率，提升病患便利性。另一個新興趨勢是個人化醫療的興起，個人化醫療根據個別基因圖譜客製化治療方案，為患者提供更有效、更有針對性的治療。這些創新正在推動凝血因子缺乏症市場走向更有效率、以患者為中心的解決方案，有望改善患者的生活品質和出血性疾病的長期管理。

本報告概述了全球凝血因子缺乏症市場，包括缺乏症類型、產品類型、最終用戶和地區的趨勢，以及參與市場的公司概況。

目錄

執行摘要

第1章全球凝血因子缺乏症市場：產業展望

• 介紹
• 市場趨勢
• 法律規範
• 流行病學分析
• 臨床試驗分析
• 市場動態
  • 影響分析
  • 市場促進因素
  • 市場挑戰
  • 市場機會

2. 全球凝血因子缺乏症市場（依缺乏症類型），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

3. 全球凝血因子缺乏症市場（依產品類型），2023-2035

• 血漿來源的凝血因子
• 重組凝血因子
• 其他

4. 2023 年至 2035 年全球凝血因子缺乏症市場（依最終使用者分類）

• 醫院
• 專科診所
• 其他

5. 2023-2035 年全球凝血因子缺乏症市場（按地區）

• 北美洲
• 歐洲
• 亞太地區

6. 全球凝血因子缺乏症市場：競爭格局與公司概況

• 關鍵策略和發展
  • 合併與收購
  • 協同效應活動
  • 業務擴展和資金籌措
  • 產品發布和核准
  • 其他活動
• 公司簡介
  • 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7章調查方法

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