遗传性血管水肿 (HAE) 治疗市场、机会、成长动力、产业趋势分析与预测，2024-2032

2023 年，全球遗传性血管水肿治疗市场估值为41 亿美元，预计2024 年至2032 年复合年增长率为9.2%。进步，特别是增强遗传性测试。这些进步导致更多的诊断和越来越多的患者寻求有效的治疗。

例如，《罕见疾病顾问报告》强调，在美国，HAE 发作每年导致15,000-30,000 人次到急诊室就诊。 ，其中不到38症状出现后 1-3 年内获得准确诊断的患者百分比。医疗保健专业人员和患者的意识不断增强，加上标靶治疗的突破，预计将推动市场成长。这些突破包括新的补体抑制剂和缓激肽 B2 受体拮抗剂。

标靶治疗领域的持续创新，尤其是新型补体抑制剂和缓激肽 B2 受体拮抗剂，正在推动市场扩张。这些疗法比传统疗法具有卓越的功效和安全性，对患者和医疗保健提供者都有吸引力。例如，2024 年 5 月，Ionis Pharmaceuticals, Inc. 报告了其 3 期 OASIS-HAE 和 OASISplus 研究的可喜成果。他们的药物 donidalorsen 表现出每月 HAE 发作率显着且持续的降低，即使在一年后，无论患者是每月还是每两个月服药一次，发病率都有超过 90% 的改善。药物开发的这些进步正在拓宽 HAE 的治疗前景，进一步促进市场成长。

整个 HAE 治疗市场根据治疗类型、给药途径、年龄层和地区进行划分。

2023年，C1酯酶抑制剂领域引领市场，营收达18亿美元。随着医疗保健专业人员和公众对 HAE 的认识和理解不断增强，早期诊断和治疗变得更加普遍。这种意识的激增增加了对 C1 酯酶抑制剂的需求，C1 酯酶抑制剂是急性 HAE 发作的首选治疗方法。 C1 酯酶抑制剂因其安全性和有效性而受到认可，比更广泛的治疗选择越来越受到青睐。此外，它们越来越多地用于 HAE 患者的长期预防，突显了一个重要的市场趋势，特别是随着该行业从反应性护理转向预防性护理。

预计到 2032 年，成人市场将达到 51 亿美元。诊断数量的激增刺激了对成人需求的治疗的需求。由于对自身病情有更深入的了解，成年患者往往会更主动地寻求治疗，他们受益于患者倡导团体和教育活动的努力，进一步扩大了这一人群对 HAE 治疗的需求。

到 2023 年，北美将占据 46.8% 的重要份额，预计将保持其在全球遗传性血管水肿治疗市场的主导地位。该地区（尤其是美国）强大的研发基础设施促进了新型 HAE 疗法的快速开发和批准。顶级製药公司、受人尊敬的学术机构和先锋研究组织的集中催化了创新，将先进的治疗方法引入市场。此外，针对 HAE 等罕见疾病的专科诊所和治疗中心的激增也增加了患者获得护理的机会。这些中心配备了最新的疗法，在确保患者及时有效的治疗方面发挥关键作用，从而促进了市场成长。

目录

第 1 章：方法与范围

第 2 章：执行摘要

第 3 章：产业洞察

• 产业生态系统分析
• 产业影响力
  • 成长动力
    • HAE标靶治疗的进展
    • HAE 发生率增加
    • 诊断技术的进步
    • 日益向个人化医疗转变
  • 产业陷阱与挑战
    • 新 HAE 疗法的严格监管要求
    • 治疗费用高
• 成长潜力分析
• 监管环境
• 管道分析
• 未来市场趋势
• 波特的分析
• PESTEL分析

第 4 章：竞争格局

• 介绍
• 公司市占率分析
• 公司矩阵分析
• 主要市场参与者的竞争分析
• 竞争定位矩阵
• 战略仪表板

第 5 章：市场估计与预测：按治疗类型，2021 - 2032 年

• 主要趋势
• C1-酯酶抑制剂
• 缓激肽 B2 受体拮抗剂
• 激肽释放酶抑制剂
• 其他治疗类型

第 6 章：市场估计与预测：按给药途径，2021 年至 2032 年

• 主要趋势
• 口服
• 注射用

第 7 章：市场估计与预测：按年龄划分，2021 - 2032

• 主要趋势
• 儿科
• 成人
• 老年科

第 8 章：市场估计与预测：按配销通路，2021 - 2032 年

• 主要趋势
• 医院药房
• 药局和零售药局
• 网路药局

第 9 章：市场估计与预测：按地区划分，2021 - 2032 年

• 主要趋势
• 北美洲
  • 我们
  • 加拿大
• 欧洲
  • 德国
  • 英国
  • 法国
  • 西班牙
  • 义大利
  • 荷兰
  • 欧洲其他地区
• 亚太地区
  • 日本
  • 中国
  • 印度
  • 澳洲
  • 韩国
  • 亚太地区其他地区
• 拉丁美洲
  • 巴西
  • 墨西哥
  • 阿根廷
  • 拉丁美洲其他地区
• 中东和非洲
  • 南非
  • 沙乌地阿拉伯
  • 阿联酋
  • 中东和非洲其他地区

第 10 章：公司简介

• Attune Pharmaceuticals
• BioCryst Pharmaceuticals, Inc.
• Biomarin
• CSL Behring LLC
• Intellia Therapeutics
• Ionis Pharmaceuticals
• KalVista Pharmaceuticals, Inc.
• Pharming Healthcare, Inc.
• Pharvaris
• Takeda Pharmaceutical Company Ltd

The Global Hereditary Angioedema Therapeutics Market was valued at USD 4.1 billion in 2023 and is projected to grow at a CAGR of 9.2% from 2024 to 2032. Key drivers include heightened awareness of hereditary angioedema (HAE) and advancements in diagnostic technologies, particularly enhanced genetic testing. These advancements lead to more diagnoses and an increasing patient population seeking effective treatments.

For example, the Rare Disease Advisor Report highlights that HAE episodes account for 15,000-30,000 emergency department visits annually in the U.S. A survey of U.S. physicians indicated that diagnosing HAE can take anywhere from 0-6 months to over a decade, with less than 38% of patients receiving an accurate diagnosis within 1-3 years of symptom onset. Growing awareness among healthcare professionals and patients, combined with breakthroughs in targeted therapies, is expected to propel market growth. These breakthroughs include new complement inhibitors and bradykinin B2 receptor antagonists.

Ongoing innovations in targeted therapies, especially new complement inhibitors and bradykinin B2 receptor antagonists, are fueling market expansion. These therapies, boasting superior efficacy and safety over traditional treatments, appeal to both patients and healthcare providers. For instance, in May 2024, Ionis Pharmaceuticals, Inc. reported promising outcomes from its Phase 3 OASIS-HAE and OASISplus studies. Their drug, donidalorsen, showcased a marked and sustained reduction in monthly HAE attack rates, with over 90% improvement even after a year, regardless of whether patients were dosed monthly or bi-monthly. Such advancements in drug development are broadening the therapeutic landscape for HAE, further bolstering market growth.

The overall HAE therapeutics market is divided based on treatment type, route of administration, age group, and region.

In 2023, the C1 esterase inhibitors segment led the market with revenues reaching USD 1.8 billion. As awareness and understanding of HAE have grown among both healthcare professionals and the public, earlier diagnoses and treatments have become more common. This surge in awareness has heightened the demand for C1 esterase inhibitors, the go-to treatment for acute HAE attacks. Recognized for their safety and effectiveness, C1 esterase inhibitors are increasingly preferred over broader treatment options. Furthermore, their rising use for long-term prevention in HAE patients underscores a significant market trend, especially with the industry's shift from reactive to preventive care.

The adult segment is projected to hit USD 5.1 billion by 2032. Thanks to advancements in diagnostics and heightened awareness, a growing number of adults are being accurately diagnosed with HAE. This surge in diagnoses has spurred demand for therapies tailored to adult needs. Adult patients, often more proactive in their treatment pursuits due to a deeper understanding of their condition, benefit from the efforts of patient advocacy groups and educational campaigns, further amplifying the demand for HAE therapies in this demographic.

North America, holding a significant 46.8% share in 2023, is poised to maintain its dominance in the global hereditary angioedema therapeutics market. The region's robust R&D infrastructure, particularly in the U.S., has facilitated the swift development and approval of novel HAE therapies. The concentration of top-tier pharmaceutical firms, esteemed academic institutions, and pioneering research organizations has catalyzed innovation, ushering advanced treatments into the market. Additionally, the proliferation of specialized clinics and treatment centers for rare diseases like HAE enhances patient access to care. These centers, equipped with the latest therapies, play a pivotal role in ensuring timely and effective treatment for patients, thereby bolstering market growth.

Table of Contents

Chapter 1 Methodology and Scope

• 1.1 Market scope and definitions
• 1.2 Research design
  • 1.2.1 Research approach
  • 1.2.2 Data collection methods
• 1.3 Base estimates and calculations
  • 1.3.1 Base year calculation
  • 1.3.2 Key trends for market estimation
• 1.4 Forecast model
• 1.5 Primary research and validation
  • 1.5.1 Primary sources
  • 1.5.2 Data mining sources

Chapter 2 Executive Summary

• 2.1 Industry 360° synopsis

Chapter 3 Industry Insights

• 3.1 Industry ecosystem analysis
• 3.2 Industry impact forces
  • 3.2.1 Growth drivers
    • 3.2.1.1 Advancement in targeted therapies for HAE
    • 3.2.1.2 Increasing incidence of HAE
    • 3.2.1.3 Advancement in diagnostic technologies
    • 3.2.1.4 Increasing shift towards personalized medicine
  • 3.2.2 Industry pitfalls and challenges
    • 3.2.2.1 Stringent regulatory requirements for new HAE therapies
    • 3.2.2.2 High cost of treatment
• 3.3 Growth potential analysis
• 3.4 Regulatory landscape
• 3.5 Pipeline analysis
• 3.6 Future market trends
• 3.7 Porter's analysis
• 3.8 PESTEL analysis

Chapter 4 Competitive Landscape, 2023

• 4.1 Introduction
• 4.2 Company market share analysis
• 4.3 Company matrix analysis
• 4.4 Competitive analysis of major market players
• 4.5 Competitive positioning matrix
• 4.6 Strategic dashboard

Chapter 5 Market Estimates and Forecast, By Treatment Type, 2021 - 2032 ($ Mn)

• 5.1 Key trends
• 5.2 C1-esterase inhibitor
• 5.3 Bradykinin B2 receptor antagonist
• 5.4 Kallikrein inhibitor
• 5.5 Other treatment types

Chapter 6 Market Estimates and Forecast, By Route of administration, 2021 - 2032 ($ Mn)

• 6.1 Key trends
• 6.2 Oral
• 6.3 Parenteral

Chapter 7 Market Estimates and Forecast, By Age Group, 2021 - 2032 ($ Mn)

• 7.1 Key trends
• 7.2 Pediatric
• 7.3 Adult
• 7.4 Geriatric

Chapter 8 Market Estimates and Forecast, By Distribution Channel, 2021 - 2032 ($ Mn)

• 8.1 Key trends
• 8.2 Hospital pharmacies
• 8.3 Drug store and retail pharmacies
• 8.4 Online pharmacies

Chapter 9 Market Estimates and Forecast, By Region, 2021 - 2032 ($ Million)

• 9.1 Key trends
• 9.2 North America
  • 9.2.1 U.S.
  • 9.2.2 Canada
• 9.3 Europe
  • 9.3.1 Germany
  • 9.3.2 UK
  • 9.3.3 France
  • 9.3.4 Spain
  • 9.3.5 Italy
  • 9.3.6 Netherlands
  • 9.3.7 Rest of Europe
• 9.4 Asia Pacific
  • 9.4.1 Japan
  • 9.4.2 China
  • 9.4.3 India
  • 9.4.4 Australia
  • 9.4.5 South Korea
  • 9.4.6 Rest of Asia Pacific
• 9.5 Latin America
  • 9.5.1 Brazil
  • 9.5.2 Mexico
  • 9.5.3 Argentina
  • 9.5.4 Rest of Latin America
• 9.6 Middle East and Africa
  • 9.6.1 South Africa
  • 9.6.2 Saudi Arabia
  • 9.6.3 UAE
  • 9.6.4 Rest of Middle East and Africa

Chapter 10 Company Profiles

• 10.1 Attune Pharmaceuticals
• 10.2 BioCryst Pharmaceuticals, Inc.
• 10.3 Biomarin
• 10.4 CSL Behring LLC
• 10.5 Intellia Therapeutics
• 10.6 Ionis Pharmaceuticals
• 10.7 KalVista Pharmaceuticals, Inc.
• 10.8 Pharming Healthcare, Inc.
• 10.9 Pharvaris
• 10.10 Takeda Pharmaceutical Company Ltd