B型血友病市場 - 全球和區域分析：按區域 - 分析和預測（2025-2035 年）

Hemophilia B Market - A Global and Regional Analysis: Focus on Country and Region - Analysis and Forecast, 2025-2035

出版日期: 2025年09月22日 | 出版商:

BIS Research | 英文 100 Pages | 商品交期: 1-5個工作天內

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簡介目錄圖表

全球B型血友病市場分析與預測（2025-2035年）

B型血友病是一種罕見的遺傳性出血性疾病，由於凝血因子 IX（一種正常血液凝固所必需的蛋白質）的缺乏或功能障礙，導致血液無法正常凝結。

B型血友病，又稱「聖誕病」，以首位確診患者Stephen Christmas命名。此病是由X染色體上的F9基因突變所引起。因此，B型血友病主要影響男性；女性是常見的帶因者，但由於X染色體惰性，也可能出現較輕微的症狀。

B型血友病市場正經歷強勁成長，主要得益於基因療法和長效療法的進步。傳統的凝血因子IX替代療法正被HemGenix和Beqvez等一次性基因療法補充，在某些情況下甚至被取代。這些治療方法代表了B型血友病臨床管理的重大飛躍，為許多患者提供了功能性治癒的潛力。儘管初始成本較高，但支付方認知到這些療法的長期經濟價值，因為它們減少了患者終生所需的治療次數。

此外，早期檢測和診斷技術的進步使得診斷更加精準、干預更加及時、患者管理更加完善，在加速B型血友病市場成長方面發揮關鍵作用。基因檢測的創新，尤其是次世代定序（NGS）和基於 DNA 的診斷技術，顯著提高了識別凝血因子 IX 基因突變的能力，能夠在新生兒和高危險群中更早發現突變。這推動向個人化治療模式的轉變，即更早地進行預防性治療，最終預防出血事件並改善長期生活品質。

儘管取得了這些進展，B型血友病市場仍面臨許多挑戰。其中一個主要障礙是高昂的治療費用。先進的療法，尤其是那些半衰期延長的凝血因子IX濃縮物和基因治療藥物，雖然具有令人矚目的長期臨床價值，但需要高昂的前期成本，這對廣泛的市場滲透構成了挑戰。這種定價限制了在成本受限的醫療環境中的採用，並增加了償付談判的複雜性。即使是標準的凝血因子替代療法也需要高昂的經常性支出，這給支付方和醫療保健系統帶來了巨大的財務壓力。因此，高昂的經濟負擔仍然是一個主要瓶頸，阻礙了各地區的可擴展性和永續的市場進入。

另一個阻礙因素是基因治療和其他先進生技藥品的複雜性和風險。雖然基因治療是一種突破性的方法，但長期安全性資料仍然有限。人們擔心的問題包括免疫反應、插入誘變和療效的差異性。監管機構要求進行長期的追蹤研究，這可能會延遲商業化時間表並增加開發成本。這種不確定性可能會使醫生和患者不願意選擇新療法，而選擇那些經過充分研究、成熟的替代療法。

全球B型血友病市場競爭激烈，UniQure Biopharma BV、Pfizer公司、Novo Nordisk A/S、HEMA Biologics 和 Alnylam Pharmaceuticals 等主要企業推動創新和市場成長。這些公司在開發可顯著改善患者預後的新治療方法處於領先地位。透過對研發（R&D）的策略性投資、擴大產品系列以及增加全球獲得最先進治療的機會，這些行業領導者塑造B型血友病治療的未來並為市場動態成長做出貢獻。此外，擴展到新興市場和透過區域分銷網路增加影響力是挖掘服務不足患者群體的關鍵策略。這些策略使公司能夠加強其市場地位並加速競爭激烈的B型血友病治療市場中的成長。

B型血友病市場細分

細分1：依地區

北美洲
歐洲
亞太地區

醫療支出的不斷成長和償付體系的不斷完善進一步增強了市場。在美國、歐盟和日本等地區，全面的保險覆蓋保障了病患獲得昂貴治療的途徑。同時，新興經濟體將血友病治療納入國家醫療保健計劃，改善治療可近性和診斷基礎設施。預計這一趨勢將在未來十年顯著推動拉丁美洲、亞洲和中東地區的市場滲透率。

此外，尤其是在新興市場，人們對B型血友病的認知和早期診斷不斷提高，推動市場成長。世界血友病聯盟等組織所進行的教育宣傳活動以及新生兒篩檢計畫的普及，提高檢出率。次世代定序（NGS）和基於 PCR 的方法等基因診斷技術的進步，使得早期準確識別 F9 基因突變成為可能。早期診斷使得在嬰兒期就開始預防性治療成為可能，顯著減少患者一生中的出血發作和關節損傷。

Hemophilia B is a rare, inherited bleeding disorder in which the blood does not clot properly due to a deficiency or malfunction of clotting factor IX (nine), a protein essential for normal blood coagulation. Also known as Christmas disease named after the first diagnosed patient, Stephen Christmas Hemophilia B is caused by mutations in the F9 gene located on the X chromosome. As a result, it primarily affects males, while females are typically carriers, though some can also experience mild symptoms due to X-chromosome inactivation.

The Hemophilia B market is experiencing robust growth, driven primarily by advancements in gene therapy and long-acting treatments. Traditional factor IX replacement therapies are being supplemented-and in some cases replaced by one-time gene therapies such as Hemgenix and Beqvez, which have demonstrated long-term efficacy and reduced annualized bleeding rates. These therapies represent a significant leap forward in the clinical management of Hemophilia B, offering a potential functional cure for many patients. Despite their high upfront costs, payers are increasingly recognizing their long-term economic value due to reduced lifetime treatment needs.

Additionally, technological advancements in early detection and diagnostics are playing a pivotal role in accelerating the growth of the hemophilia B market by enabling more accurate diagnosis, timely intervention, and improved patient management. Innovations in genetic testing particularly next-generation sequencing (NGS) and DNA-based diagnostics have significantly enhanced the ability to identify mutations in the factor IX gene, allowing for early detection in newborns and at-risk populations. This facilitates a shift toward personalized care models, where prophylactic treatment can be initiated early, ultimately preventing bleeding episodes and improving long-term quality of life.

Despite these advances, the hemophilia B market faces several challenges. One significant hurdle is the high cost of treatment. Advanced treatments particularly extended half-life factor IX products and gene therapies offer compelling long-term clinical value but come with significant upfront pricing, which poses a challenge for broad market penetration. This pricing dynamic limits adoption in cost-constrained healthcare environments and introduces complexities in reimbursement negotiations. Even standard factor replacement therapies represent a high recurring expenditure, placing considerable financial pressure on payers and healthcare systems. As a result, affordability remains a critical bottleneck that impedes scalability and sustainable market access across diverse geographies.

Another restraining factor is the complexity and risk associated with gene therapies and other advanced biologics. Although gene therapies offer a transformative approach, long-term safety data is still limited. There are concerns about immune responses, insertional mutagenesis, and variability in treatment durability. Regulatory agencies require long-term follow-up studies, which can delay commercialization timelines and increase development costs. These uncertainties may lead to hesitancy among physicians and patients when choosing novel therapies over established, well-studied alternatives.

The global hemophilia B market is highly competitive, with several leading companies driving innovation and market growth, such as UniQure Biopharma B.V., Pfizer Inc., Novo Nordisk A/S., HEMA Biologics., and Alnylam Pharmaceuticals. These companies are at the forefront of developing novel therapies that are significantly improving patient outcomes. Through strategic investments in research and development (R&D), expanding product portfolios, and increasing global access to cutting-edge treatments, these industry leaders are shaping the future of hemophilia B care and contributing to the dynamic growth of the market. Additionally, expanding into emerging markets and increasing their presence through regional distribution networks are key strategies to tap into underserved patient populations. These strategies are enabling companies to strengthen their market position and accelerate growth in the competitive hemophilia B treatment landscape.

Hemophilia B Market Segmentation:

Segmentation 1: by Region

North America
Europe
Asia-Pacific

The market is further bolstered by growing healthcare spending and improved reimbursement frameworks. In regions such as the U.S., EU, and Japan, comprehensive insurance coverage supports access to costly therapies. Meanwhile, emerging economies are expanding their national health programs to include hemophilia care, thereby increasing treatment accessibility and diagnostic infrastructure. This trend is expected to drive a significant rise in market penetration across Latin America, Asia, and the Middle East in the coming decade.

Moreover, the increasing awareness and early diagnosis of Hemophilia B, especially in emerging markets is fuelling the market growth. Educational campaigns by organizations such as the World Federation of Hemophilia and broader access to newborn screening programs have improved detection rates. Advances in genetic diagnostics, such as next-generation sequencing (NGS) and PCR-based methods, allow for early and precise identification of F9 gene mutations. Early diagnosis enables the initiation of prophylactic treatment in infancy, which can significantly reduce bleeding episodes and joint damage over the patient's lifetime.

Executive Summary

Scope and Definition

Market/Product Definition

Inclusion and Exclusion

Key Questions Answered

Analysis and Forecast Note

1. Global Hemophilia B Market: Industry Analysis

1.1 Market Overview and Ecosystem
1.2 Epidemiological Analysis
1.3 Key Market Trends
- 1.3.1 Impact Analysis
1.4 Regulatory Landscape
1.5 Pipeline Analysis
1.6 Market Dynamics
- 1.6.1 Overview
- 1.6.2 Market Drivers
- 1.6.3 Market Restraints
- 1.6.4 Market Opportunities

2. Global Hemophilia B Market (Region), Value ($Million), 2023-2035

2.1 North America
- 2.1.1 Market Dynamics
- 2.1.2 Market Sizing and Forecast
- 2.1.3 North America Hemophilia B Market, by Country ($Million), 2023-2035
  - 2.1.3.1 U.S.
2.2 Europe
- 2.2.1 Market Dynamics
- 2.2.2 Market Sizing and Forecast
- 2.2.3 Europe Hemophilia B Market, by Country ($Million), 2023-2035
  - 2.2.3.1 U.K.
  - 2.2.3.2 France
  - 2.2.3.3 Germany
  - 2.2.3.4 Italy
  - 2.2.3.5 Spain
2.3 Asia-Pacific
- 2.3.1 Market Dynamics
- 2.3.2 Market Sizing and Forecast
- 2.3.3 Asia-Pacific Hemophilia B Market, by Country ($Million), 2023-2035
  - 2.3.3.1 Japan

3. Competitive Landscape and Company Profiles

3.1 Competitive Landscape
- 3.1.1 Mergers and Acquisitions
- 3.1.2 Partnership, Alliances and Business Expansion
- 3.1.3 New Offerings
- 3.1.4 Regulatory Activities
- 3.1.5 Funding Activities
3.2 Company Profiles
- 3.2.1 UniQure Biopharma B.V.
  - 3.2.1.1 Overview
  - 3.2.1.2 Top Products / Product Portfolio
  - 3.2.1.3 Top Competitors
  - 3.2.1.4 Target Customers/End-Users
  - 3.2.1.5 Key Personnel
  - 3.2.1.6 Analyst View
- 3.2.2 Pfizer Inc.
  - 3.2.2.1 Overview
  - 3.2.2.2 Top Products / Product Portfolio
  - 3.2.2.3 Top Competitors
  - 3.2.2.4 Target Customers/End-Users
  - 3.2.2.5 Key Personnel
  - 3.2.2.6 Analyst View
- 3.2.3 CSL Behring.
  - 3.2.3.1 Overview
  - 3.2.3.2 Top Products / Product Portfolio
  - 3.2.3.3 Top Competitors
  - 3.2.3.4 Target Customers/End-Users
  - 3.2.3.5 Key Personnel
  - 3.2.3.6 Analyst View
- 3.2.4 Sanofi
  - 3.2.4.1 Overview
  - 3.2.4.2 Top Products / Product Portfolio
  - 3.2.4.3 Top Competitors
  - 3.2.4.4 Target Customers/End-Users
  - 3.2.4.5 Key Personnel
  - 3.2.4.6 Analyst View
- 3.2.5 ApcinteX Ltd.
  - 3.2.5.1 Overview
  - 3.2.5.2 Top Products / Product Portfolio
  - 3.2.5.3 Top Competitors
  - 3.2.5.4 Target Customers/End-Users
  - 3.2.5.5 Key Personnel
  - 3.2.5.6 Analyst View
- 3.2.6 Sangamo Therapeutics.
  - 3.2.6.1 Overview
  - 3.2.6.2 Top Products / Product Portfolio
  - 3.2.6.3 Top Competitors
  - 3.2.6.4 Target Customers/End-Users
  - 3.2.6.5 Key Personnel
  - 3.2.6.6 Analyst View
- 3.2.7 Novo Nordisk A/S.
  - 3.2.7.1 Overview
  - 3.2.7.2 Top Products / Product Portfolio
  - 3.2.7.3 Top Competitors
  - 3.2.7.4 Target Customers/End-Users
  - 3.2.7.5 Key Personnel
  - 3.2.7.6 Analyst View
- 3.2.8 HEMA Biologics.
  - 3.2.8.1 Overview
  - 3.2.8.2 Top Products / Product Portfolio
  - 3.2.8.3 Top Competitors
  - 3.2.8.4 Target Customers/End-Users
  - 3.2.8.5 Key Personnel
  - 3.2.8.6 Analyst View
- 3.2.9 Alnylam Pharmaceuticals.
  - 3.2.9.1 Overview
  - 3.2.9.2 Top Products / Product Portfolio
  - 3.2.9.3 Top Competitors
  - 3.2.9.4 Target Customers/End-Users
  - 3.2.9.5 Key Personnel
  - 3.2.9.6 Analyst View
- 3.2.10 Centessa Pharmaceuticals
  - 3.2.10.1 Overview
  - 3.2.10.2 Top Products / Product Portfolio
  - 3.2.10.3 Top Competitors
  - 3.2.10.4 Target Customers/End-Users
  - 3.2.10.5 Key Personnel
  - 3.2.10.6 Analyst View
- 3.2.11 Others