遺傳性血管性水腫（HAE）：全球市場展望、流行病學、競爭格局和市場預測報告（32個主要市場），2025-2035年

市場概覽

• 預計西班牙 HAE 市場將從 2025 年的 1.26 億美元成長到 2035 年的 3.15 億美元。
• 以下因素正在推動市場成長：
• 預防性生物製藥和標靶治療的廣泛應用。
• 診斷技術和患者識別方面的改進
• 未來市場擴張取決於便利、長效療法和基因療法的廣泛應用。

遺傳性血管性水腫（HAE）概述

遺傳性血管性水腫（HAE）是一種罕見的體染色體顯性遺傳疾病，其特徵是反覆發作、可自行消退、無搔癢的皮下和黏膜下水腫，主要涉及皮膚、胃腸道和上呼吸道。 I型HAE由SERPING1基因突變引起，導致C1抑制劑（C1-INH）水平降低；而II型HAE則導致C1-INH蛋白功能障礙。兩種情況下，緩激肽的產生均失控，導致血管滲透性增加。也存在正常的C1-INH突變體。發作時表現為局部腫脹和劇烈腹痛，喉頭水腫可導致窒息死亡。診斷依據是C4水平降低以及C1-INH的定量或功能檢測。截至2026年，急性發作的治療方法包括使用血漿來源或重組C1-INH濃縮物，以及緩激肽B2受體拮抗劑icatibant。另一方面，最新的長期預防性治療方案建議使用血漿激肽釋放酶抑制劑beotralstat或單株抗體lanadermab。

主要亮點

• 在韓國，預計到 2025 年，確診的 HAE 患者人數將增加到 2035 年的 230 人。
• HAE 是一種罕見的遺傳性疾病，其特徵是反覆出現可能危及生命的水腫發作。
• 隨著人們意識的提高和基因檢測的普及，全球診斷率正在上升。
• 長期預防性治療改變了疾病管理方式，降低了癲癇發作的頻率。

市場定義：

• 北美洲（美國、加拿大）
• 歐洲（奧地利、比利時、捷克、丹麥、芬蘭、法國、德國、希臘、義大利、荷蘭、挪威、波蘭、葡萄牙、俄羅斯、西班牙、瑞典、瑞士、英國）
• 中東（沙烏地阿拉伯、阿拉伯聯合大公國、科威特、土耳其）
• 亞太地區（澳洲、中國、香港、印度、印尼、日本、馬來西亞、紐西蘭、菲律賓、新加坡、韓國、台灣、泰國、越南）
• 非洲（埃及、奈及利亞、南非、摩洛哥）
• 南美洲/拉丁美洲（阿根廷、巴西、智利、哥倫比亞、墨西哥、秘魯）

格式化和更新訊息

• 詳細報告（PDF）
• 市場預測模型（基於微軟Excel）
• 流行病學數據（MS Excel，互動式工具）
• 高階主管洞察（PPT簡報）
• 其他功能：定期更新、自訂和顧問支援。
• 根據 Thelansis 的政策，我們確保所有最新更新在發布前都反映在報告內容和市場模型中。

主要問題

• 如何最佳化在 32 個市場（北美、歐洲、中東、亞太、非洲和南美/拉丁美洲）的藥物開發和生命週期管理策略？
• 從發病率、盛行率、人群組成以及接受藥物治療的患者人數來看，患者數量分別是多少？
• 未來十年市場收入和病患佔有率的預測是多少？
• 哪些因素對市場趨勢影響最大？
• 受訪專家對目前和新興的治療方法有何看法？
• 哪款在研發線產品最有前景？其上市潛力及未來市場定位如何？
• 主要未被滿足的需求是什麼？ KOL 對目標受眾有何期望？
• 為確保藥物獲得批准並順利進入市場，必須滿足哪些關鍵的監管和支付方要求？

目標國家

• 北美洲（美國、加拿大）
• 歐洲（奧地利、比利時、捷克、丹麥、芬蘭、法國、德國、希臘、義大利、荷蘭、挪威、波蘭、葡萄牙、俄羅斯、西班牙、瑞典、瑞士、英國）
• 中東（沙烏地阿拉伯、阿拉伯聯合大公國、科威特、土耳其）
• 亞太地區（澳洲、中國、香港、印度、印尼、日本、馬來西亞、紐西蘭、菲律賓、新加坡、韓國、台灣、泰國、越南）
• 非洲（埃及、奈及利亞、南非、摩洛哥）
• 南美洲/拉丁美洲（阿根廷、巴西、智利、哥倫比亞、墨西哥、秘魯）

大公司

• Intellia Therapeutics
• Astria Therapeutics, Inc.
• Ionis Pharmaceuticals, Inc.
• CSL Behring
• ADARx Pharmaceuticals, Inc.
• KalVista Pharmaceuticals, Ltd.
• BioCryst Pharmaceuticals
• Shanghai Argo Biopharmaceutical Co., Ltd.
• Pharvaris Netherlands BV
• Nang Kuang Pharmaceutical Co., Ltd.

目錄

第1章：主要調查結果及分析師說明

• 主要趨勢：市場概況、SWOT分析、商業利益與風險等。

第2章：疾病背景

• 疾病定義、分類、病因和病理生理學、藥物標靶等。

第3章：流行病學

• 重點
• 發病率/盛行率
• 已確診並正在接受藥物治療的患者族群。
• 合併症
• 其他相關患者群

第4章 市場規模及預測

• 重點
• 市場促進因素與限制因素
• 按藥物類別分類的趨勢
• 各國具體趨勢

第5章 競爭情勢

• 目前的治療方法
• 重點
• 診斷和治療過程/演算法
• 目前主要療法－概述及關鍵意見領袖見解
• 新興療法
• 重點
• 值得關注的後期新興療法—概述、市場上市預期和關鍵意見領袖見解
• 值得關注的早期管道

第6章：未滿足的需求與目標產品分析

• 尚未滿足的主要需求及其透過新興療法實現的潛力。
• TPP分析與KOL展望

第7章 監理與報銷環境

第8章附錄

Global Hereditary Angioedema (HAE) Market Outlook

Thelansis's "Global Hereditary Angioedema (HAE) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report (Top 32 Markets) - 2025 To 2035" covers disease overview, epidemiology, kidney biopsy cases, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Hereditary Angioedema treatment modalities options for the 32 markets (North America, Europe, Middle East, Asia Pacific, Africa, South / Latin America).

Hereditary Angioedema (HAE) Overview

Hereditary angioedema (HAE) is a rare autosomal dominant disorder characterized by recurrent, self-limiting episodes of non-pruritic subcutaneous and submucosal edema primarily affecting the skin, gastrointestinal tract, and upper airway. Driven by mutations in the SERPING1 gene, type I HAE causes deficient C1-inhibitor (C1-INH) levels while type II results in dysfunctional C1-INH protein, both leading to unregulated bradykinin production and vascular permeability. Normal C1-INH variants also exist. Attacks present as localized swelling or debilitating abdominal pain, with laryngeal edema causing fatal asphyxiation risk. Diagnosis relies on low C4 levels and specific C1-INH quantitative or functional assays. Treatment in 2026 utilizes plasma-derived or recombinant C1-INH concentrates and the bradykinin B2 receptor antagonist icatibant for acute attacks, while modern long-term prophylaxis favors the plasma kallikrein inhibitor berotralstat or the monoclonal antibody lanadelumab.

Key Highlights

• In South Korea, diagnosed prevalent HAE cases are projected to increase from 186 in 2025 to 230 by 2035.
• HAE is a rare genetic disorder characterized by recurrent, potentially life-threatening swelling attacks.
• Improved awareness and genetic testing are increasing diagnosis rates globally.
• Long-term prophylactic therapies are transforming disease management and reducing attack frequency.

Market Overview

• The Spain HAE market is projected to grow from $126M in 2025 to $315M by 2035.
• Market growth is driven by:
• Strong uptake of prophylactic biologics and targeted therapies
• Improved diagnosis and patient identification
• Future expansion will depend on convenient long-acting and gene-based treatment approaches.

Market Definition:

• North America (United States, Canada)
• Europe (Austria, Belgium, Czech Republic, Denmark, Finland, France, Germany, Greece, Italy, Netherlands, Norway, Poland, Portugal, Russia, Spain, Sweden, Switzerland, United Kingdom)
• Middle East (Saudi Arabia, UAE, Kuwait, Turkey)
• Asia Pacific (Australia, China, Hong Kong, India, Indonesia, Japan, Malaysia, New Zealand, Philippines, Singapore, South Korea, Taiwan, Thailand, Vietnam)
• Africa (Egypt, Nigeria, South Africa, Morocco)
• South / Latin America (Argentina, Brazil, Chile, Colombia, Mexico, Peru)

Insights driven by robust research, including:

• In-depth interviews with leading KOLs and payers
• Physician surveys
• RWE analysis for claims and EHR datasets
• Secondary research (e.g., peer-reviewed journal articles, third-party research databases)

Deliverables format and updates*:

• Detailed Report (PDF)
• Market Forecast Model (MS Excel-based automated dashboard)
• Epidemiology (MS Excel; interactive tool)
• Executive Insights (PowerPoint presentation)
• Others: regular updates, customizations, consultant support
• As per Thelansis's policy, we ensure that we include all the recent updates before releasing the report content and market model.

Salient features of Market Forecast model:

• 10-year market forecast (2025-2035)
• Bottom-up patient-based market forecasts validated through the top-down sales methodology
• Covers clinically and commercially-relevant patient populations/ line of therapies
• Annualized drug-level sales and patient share projections
• Utilizes our proprietary Epilansis and Analog tool (e.g., drug uptake and erosion) datasets and conjoint analysis approach
• Detailed methodology/sources & assumptions
• Graphical and tabular outputs
• Users can customize the model based on requirements

Key business questions answered:

• How can drug development and lifecycle management strategies be optimized across the 32 markets (North America, Europe, Middle East, Asia Pacific, Africa, South / Latin America)?
• How large is the patient population in terms of incidence, prevalence, segments, and those receiving drug treatments?
• What is the 10-year market outlook for sales and patient share?
• Which events will have the greatest impact on the market's trajectory?
• What insights do interviewed experts provide on current and emerging treatments?
• Which pipeline products show the most promise, and what is their potential for launch and future positioning?
• What are the key unmet needs and KOL expectations for target profiles?
• What key regulatory and payer requirements must be met to secure drug approval and favorable market access?

Countries Covered

• North America (United States, Canada)
• Europe (Austria, Belgium, Czech Republic, Denmark, Finland, France, Germany, Greece, Italy, Netherlands, Norway, Poland, Portugal, Russia, Spain, Sweden, Switzerland, United Kingdom)
• Middle East (Saudi Arabia, UAE, Kuwait, Turkey)
• Asia Pacific (Australia, China, Hong Kong, India, Indonesia, Japan, Malaysia, New Zealand, Philippines, Singapore, South Korea, Taiwan, Thailand, Vietnam)
• Africa (Egypt, Nigeria, South Africa, Morocco)
• South / Latin America (Argentina, Brazil, Chile, Colombia, Mexico, Peru)

Companies Mentioned

• Intellia Therapeutics
• Astria Therapeutics, Inc.
• Ionis Pharmaceuticals, Inc.
• CSL Behring
• ADARx Pharmaceuticals, Inc.
• KalVista Pharmaceuticals, Ltd.
• BioCryst Pharmaceuticals
• Shanghai Argo Biopharmaceutical Co., Ltd.
• Pharvaris Netherlands B.V.
• Nang Kuang Pharmaceutical Co., Ltd.

Table of Contents

1. Key Findings and Analyst Commentary

• Key trends: market snapshots, SWOT analysis, commercial benefits and risks, etc.

2. Disease Context

• Disease definition, classification, etiology and pathophysiology, drug targets, etc.

3. Epidemiology

• Key takeaways
• Incidence / Prevalence
• Diagnosed and Drug-Treated populations
• Comorbidities
• Other relevant patient segments

4. Market Size and Forecast

• Key takeaways
• Market drivers and constraints
• Drug-class specific trends
• Country-specific trends

5. Competitive Landscape

• Current therapies
• Key takeaways
• Dx and Tx journey/algorithm
• Key current therapies - profiles and KOL insights
• Emerging therapies
• Key takeaways
• Notable late-phase emerging therapies - profiles, launch expectations, KOL insights
• Notable early-phase pipeline

6. Unmet Need and TPP Analysis

• Top unmet needs and future attainment by emerging therapies
• TPP analysis and KOL expectations

7. Regulatory and Reimbursement Environments (by country and payer insights)

8. Appendix (e.g., bibliography, methodology)