遺伝性血管性浮腫治療薬の世界市場-2023年～2030年

市場概要

遺伝性血管性浮腫治療薬の世界市場は、2022年に29億米ドルに達し、2030年には最大54億米ドルに達することで有利な成長を示すと予測されています。世界の遺伝性血管性浮腫治療薬市場は、予測期間中（2023～2030年）にCAGR 8.6%を示すと予測されています。予防療法の採用拡大、患者中心のアプローチの重視、新規療法のイントロダクションなどが最近の市場動向です。

世界の遺伝性血管性浮腫治療薬市場は、近年継続的に拡大しています。遺伝性血管性浮腫（HAE）は、体のさまざまな部位で再発する腫脹エピソードを特徴とするまれな遺伝性疾患です。HAEはSERPING1遺伝子の変異によって引き起こされ、SERPING1遺伝子は血液タンパク質C1-インヒビターをコードし、炎症と血液凝固をもたらす一連のプロセスを制御しています。

世界の遺伝性血管性浮腫治療薬の市場範囲は、カリクレイン阻害剤、C1エステラーゼ阻害剤、選択的ブラジキニンb2受容体拮抗剤などの薬物クラスを包含する構成要素で構成され、遺伝性血管性浮腫治療薬の市場シェアで使用量が増加しています。遺伝性血管性浮腫治療薬の世界市場は、遺伝性血管性浮腫の有病率の増加、治療オプションの進歩、ヘルスケア支出の増加などの要因により拡大しています。

市場力学

治療の進歩が遺伝性血管性浮腫治療薬市場の成長を牽引

遺伝性血管性浮腫の患者は、標的治療、遺伝子治療、革新的なドラッグデリバリー方法の開発など、治療オプションの大幅な進歩から多大な恩恵を受けています。例えば、2022年にAmerican College of Allergy, Asthma, and Immunologyが発表した研究によると、CRISPRゲノム編集候補のNTLA-2002は遺伝性血管性浮腫（HAE）のために開発されており、1回限りの全身投与治療です。

NTLA-2002は肝細胞のKLKB1遺伝子を破壊し、カリクレインとして知られる特定のタンパク質の産生を減少させることを目的としています。そのため、治療法の進歩が予測期間中の市場を牽引すると思われます。

意識の高まりが世界の遺伝性血管性浮腫治療薬市場の主な促進要因

ヘルスケア従事者は、遺伝性血管性浮腫、その徴候や症状、潜在的な治療法に関する知識を深めています。診断率の向上、適切な紹介、効率的な病態のケアによる認知度の向上により、市場は成長しています。さらに、各国の政府によるいくつかの措置が、これらの疾患に対する認識を高めています。

例えば、英国議会は2023年5月16日火曜日に遺伝性血管性浮腫（HAE）の日を祝っています。さらに議会は、HAE患者の腫脹エピソードや発作を予防するという野心的な目標を支持しています。また、希少疾病に苦しむ地域社会の健康増進のため、希少疾病フレームワークの公約を熱烈に支持します。

当院は、意思決定者に対し、希少疾患に対する臨床的認識を高め、QOLに配慮した治療を最優先し、意思決定プロセスを改善し、希少疾患を患う人々が心理的サポートを受けやすくすることの必要性を強く求めています。このように、希少疾患に関する認知度の向上は、予測期間を通じて市場を牽引していくと思われます。

治療薬の副作用が市場の成長を妨げる

一部の遺伝性血管性浮腫治療薬の副作用や安全性の問題は、患者のコンプライアンスに悪影響を及ぼし、市場の成長を抑制する可能性があります。例えば、アンドロゲンを予防的に長期間使用することは、肝毒性や男性化などの好ましくない影響をもたらす可能性があります。これらの要因を考慮すると、治療に伴う副作用は遺伝性血管性浮腫治療薬市場の成長を制限する可能性があります。

COVID-19影響分析

COVID-19のパンデミックと世界各国での封鎖により、あらゆる業種の企業の財務の健全性が影響を受けています。そのため、COVID-19公衆衛生緊急事態の期間中、米国食品医薬品局（FDA）は、スポンサーと研究者を支援し、試験参加者の安全を確保し、適正臨床実施基準（GCP）を遵守し、試験の完全性に対するリスクを最小限に抑えるための一般的な配慮を含むガイドラインを発表しました。

ロシア・ウクライナ紛争の影響分析

ロシア・ウクライナ紛争は、遺伝性血管性浮腫治療薬の世界市場に影響を与える可能性があります。想定される影響には、サプライチェーンの混乱、影響を受けた地域におけるヘルスケアや治療へのアクセスの制限、医療予算への経済的影響、研究開発努力の遅延の可能性などがあります。市場の具体的な影響を理解するためには、状況を注視することが重要です。

目次

第1章 調査手法と調査範囲

第2章 定義と概要

第3章 エグゼクティブサマリー

第4章 市場力学

• 影響要因
  • 促進要因
    • 治療の進歩
    • 意識の高まり
  • 抑制要因
    • 治療薬の副作用
  • 影響分析

第5章 産業分析

• ポーターの5フォース分析
• サプライチェーン分析
• 価格分析
• 規制分析

第6章 COVID-19分析

第7章 薬剤タイプ別

• カリクレイン阻害剤
• C1エステラーゼ阻害剤
• 選択的ブラジキニンB2受容体拮抗薬
• その他

第8章 治療タイプ別

• オンデマンド
• 予防

第9章 投与経路別

• 静脈内投与
• 皮下
• 経口

第10章 地域別

• 北米
  • 米国
  • カナダ
  • メキシコ
• 欧州
  • ドイツ
  • 英国
  • フランス
  • スペイン
  • イタリア
  • その他欧州
• 南米
  • ブラジル
  • アルゼンチン
  • その他南米
• アジア太平洋
  • 中国
  • インド
  • 日本
  • オーストラリア
  • その他アジア太平洋地域
• 中東・アフリカ

第11章 競合情勢

• 競合シナリオ
• 市況/シェア分析
• M&A分析

第12章 企業プロファイル

• BioCryst Pharmaceuticals, Inc
  • 会社概要
  • 製品ポートフォリオと説明
  • 財務概要
  • 主な発展
• CSL Behring LLC
• Takeda Pharmaceutical Company
• Pharming Healthcare, Inc
• Sanofi
• Attune Pharmaceuticals, Inc
• Ionis Pharmaceuticals
• KalVista Pharmaceuticals, Inc
• Arrowhead Pharmaceuticals
• Advanced Technology & Industrial Co., Ltd

第13章 付録

Market Overview

The Global Hereditary Angioedema Therapeutics Market reached US$ 2.9 billion in 2022 and is projected to witness lucrative growth by reaching up to US$ 5.4 billion by 2030. The global hereditary angioedema therapeutics market is expected to exhibit a CAGR 8.6% during the forecast period (2023-2030). The growing adoption of prophylactic therapies, focus on patient-centric approaches, and introduction of novel therapies are some recent market trends.

The global hereditary angioedema therapeutics market has been expanding continuously in recent years. Hereditary angioedema (HAE) is a rare genetic disorder characterized by recurrent swelling episodes in different parts of the body. HAE is caused by mutations in the SERPING1 gene, which encodes for the blood protein C1-inhibitor and controls a series of processes that result in inflammation and blood clotting.

The global hereditary angioedema therapeutics market scope comprises components encompassing drug class such as kallikrein inhibitor, c1 esterase inhibitor, and selective bradykinin b2 receptor antagonist which has increased usage of hereditary angioedema therapeutics market share. The global market for hereditary angioedema therapeutics is expanding as a result of factors including the increasing prevalence of hereditary angioedema, advancements in treatment options, and increasing healthcare expenditure.

Market Dynamics

Advancements in Treatment to Drive the Growth of the Hereditary Angioedema Therapeutics Market

Patients with hereditary angioedema have benefitted immensely from significant advances in therapy options, including the development of targeted therapies, gene therapies, and innovative drug delivery methods. For instance, a study published by American College of Allergy, Asthma, and Immunology in 2022 states that, the CRISPR genome editing candidate NTLA-2002 is being developed for hereditary angioedema (HAE) and is a one-time, systemically delivered treatment.

It is intended to disrupt the KLKB1 gene in liver cells, so decreasing the production of a particular protein known as kallikrein, whose unchecked activity is to blame for the recurrent, crippling, and perhaps deadly swelling events that happen in people with HAE. Thereby, the advancements in treatment will drive the market over the forecast period.

Growing Awareness is the Major Driver in the Global Hereditary Angioedema Therapeutics Market

Healthcare practitioners are becoming more knowledgeable about hereditary angioedema, its signs and symptoms, and the potential treatments. The market is growing because of increased awareness due to increased diagnosis rates, proper referrals, and efficient condition care. Furthermore, several steps taken by governments of different countries is creating awareness about these disorder.

For instance, UK Parliament celebrated hereditary angioedema (HAE) day on Tuesday 16 May 2023. Moreover, the Parliament House also supports the ambitious target of preventing swelling episodes or attacks in HAE patients. It enthusiastically supports the pledges made in the Rare Disease Framework to enhance the health of communities afflicted by rare diseases.

The House strongly urges decision-makers to acknowledge the necessity of raising clinical awareness of rare diseases, giving quality-of-life considerations a top priority in treatment recommendations, improving shared decision-making processes, and ensuring that people with affected conditions have better access to psychological support. Thus, increasing awareness about the disorder will drive the market over the forecast period.

Side Effects of Therapies will Hamper the Growth of the Market.

The side effects and safety issues of some hereditary angioedema treatments may have an adverse influence on patient compliance and restrain the market growth. For instance, using androgens prophylactically for a prolonged period of time can have unfavorable effects such as liver toxicity and virilization. Considering these factors, the side effects associated with treatment can limit the hereditary angioedema therapeutics market growth.

COVID-19 Impact Analysis

The financial health of companies across all industries has been impacted by the COVID-19 pandemic and lockdown in numerous nations throughout the world. Therefore, for the period of the COVID-19 public health emergency, the U.S. Food and Drug Administration (FDA) issued guidelines that include general considerations to aid sponsors and researchers, ensuring the safety of trial participants, adhering to good clinical practice (GCP), and minimizing risks to trial integrity.

Russia-Ukraine War Impact Analysis

The Russia-Ukraine conflict has the potential to have an influence on the global market for hereditary angioedema treatments. Possible consequences include supply chain disruptions, limited access to healthcare and treatment in affected areas, economic ramifications on healthcare budgets, and a potential delay in research and development efforts. It is critical to keep a close watch on the situation in order to comprehend the market's specific consequences.

Segment Analysis

The global hereditary angioedema therapeutics market is segmented based on drug class, treatment type, route of administration, and region.

The On-Demand Segment Accounts for 53.2% of Market Share Owing to Ease of Access.

The on-demand segment is projected to dominate the global hereditary angioedema therapies market. On-demand treatments are preferred for their quick alleviation and focused intervention because they include giving drugs during acute bouts to reduce symptoms. They offer flexible treatment options for patients with rare or less severe forms of hereditary angioedema. The dominance of this market has also been aided by developments in on-demand therapies, such as the development of safer and more potent drugs.

Recombinant human C1-INH (rhC1-INH), Icatibant, a bradykinin B2-receptor antagonist, and Ecallantide, a kallikrein inhibitor (only available in the US; see FDA-approved drugs for on-demand therapy of HAE attacks), are all available intravenously.

According to research presented at the American Academy of Allergy, Asthma & Immunology's annual meeting, using sebetralstat therapy on demand has been proven to significantly reduce the symptoms and intensity of hereditary angioedema attacks. When compared to a placebo, sebetralstat showed a notable decrease in the total number of hereditary angioedema attacks and the intensity of symptoms. Notably, during acute attacks, the alleviation offered by sebetralstat was noticed within the first 30 to 60 minutes of treatment.

Geographical Analysis

North America Accounted for Approximately 39.7% of the Market Share Owing to the Increasing Prevalence of Hereditary Angioedema

North America dominates the global hereditary angioedema therapeutics market because of factors such as a higher prevalence of hereditary angioedema, greater awareness, early diagnosis, and well-established healthcare infrastructure. The region has a huge patient pool and favorable reimbursement regulations, which encourage the market's growth.

According to BioNews, Inc 2022 article, a survey of American doctors revealed that between May 2019 and April 2020, there were between 1,230 and 1,331 cases of hereditary angioedema with normal C1 inhibitors (HAE-nl-C1INH) in the country. Moreover according to Lippincott journal an article published in 2022, an estimated prevalence of HAE is approximately 1 in 50,000 people, which translates to roughly 6,000 patients with HAE in the US. Therefore, the growing prevalence of hereditary angioedema in North America to dominate the market over the forecast period.

Competitive Landscape

The major global players in the hereditary angioedema therapeutics market include: BioCryst Pharmaceuticals, Inc, CSL Behring LLC, Takeda Pharmaceutical Company, Pharming Healthcare, Inc, Sanofi, Attune Pharmaceuticals, Inc, Ionis Pharmaceuticals, KalVista Pharmaceuticals, Inc, Arrowhead Pharmaceuticals, and Advanced Technology & Industrial Co., Ltd, among others.

Why Purchase the Report?

• To visualize the global hereditary angioedema therapeutics market segmentation based on drug class, treatment type, route of administration, and region, as well as understand key commercial assets and players.
• Identify commercial opportunities by analyzing trends and co-development.
• Excel data sheet with numerous data points of hereditary angioedema therapeutics market-level with all segments.
• PDF report consists of a comprehensive analysis after exhaustive qualitative interviews and an in-depth study.
• Product mapping is available as Excel consisting of key products of all the major players.

The global hereditary angioedema therapeutics market report would provide approximately 53 tables, 54 figures and 195 Pages.

Target Audience 2023

• Manufacturers/ Buyers
• Industry Investors/Investment Bankers
• Research Professionals
• Emerging Companies

Table of Contents

1. Methodology and Scope

• 1.1. Research Methodology
• 1.2. Research Objective and Scope of the Report

2. Definition and Overview

3. Executive Summary

• 3.1. Snippet by Drug Class
• 3.2. Snippet by Treatment Type
• 3.3. Snippet by Route of Administration
• 3.4. Snippet by Region

4. Dynamics

• 4.1. Impacting Factors
  • 4.1.1. Drivers
    • 4.1.1.1. Advancements in Treatment
    • 4.1.1.2. Growing Awareness is the Major Driver in the Global Hereditary Angioedema Therapeutics Market.
  • 4.1.2. Restraints
    • 4.1.2.1. The Side Effects of Therapies
  • 4.1.3. Impact Analysis

5. Industry Analysis

• 5.1. Porter's 5 Forces Analysis
• 5.2. Supply Chain Analysis
• 5.3. Pricing Analysis
• 5.4. Regulatory Analysis

6. COVID-19 Analysis

• 6.1. Analysis of COVID-19
  • 6.1.1. Scenario Before COVID-19
  • 6.1.2. Scenario During COVID-19
  • 6.1.3. Scenario Post COVID-19
• 6.2. Pricing Dynamics Amid COVID-19
• 6.3. Demand-Supply Spectrum
• 6.4. Government Initiatives Related to the Market During Pandemic
• 6.5. Manufacturers Strategic Initiatives
• 6.6. Conclusion

7. By Drug Type

• 7.1. Introduction
  • 7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Class
  • 7.1.2. Market Attractiveness Index, By Drug Class
• 7.2. Kallikrein Inhibitor*
  • 7.2.1. Introduction
  • 7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 7.3. C1 Esterase Inhibitor
• 7.4. Selective Bradykinin B2 Receptor Antagonist
• 7.5. Others

8. By Treatment Type

• 8.1. Introduction
  • 8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
  • 8.1.2. Market Attractiveness Index, By Treatment Type
• 8.2. On-demand*
  • 8.2.1. Introduction
  • 8.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 8.3. Prophylaxis

9. By Route of Administration

• 9.1. Introduction
  • 9.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
  • 9.1.2. Market Attractiveness Index, By Route of Administration
• 9.2. Intravenous*
  • 9.2.1. Introduction
  • 9.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 9.3. Subcutaneous
• 9.4. Oral

10. By Region

• 10.1. Introduction
  • 10.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Region
  • 10.1.2. Market Attractiveness Index, By Region
• 10.2. North America
  • 10.2.1. Introduction
  • 10.2.2. Key Region-Specific Dynamics
  • 10.2.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Class
  • 10.2.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
  • 10.2.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
  • 10.2.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 10.2.6.1. The U.S.
    • 10.2.6.2. Canada
    • 10.2.6.3. Mexico
• 10.3. Europe
  • 10.3.1. Introduction
  • 10.3.2. Key Region-Specific Dynamics
  • 10.3.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Class
  • 10.3.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
  • 10.3.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
  • 10.3.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 10.3.6.1. Germany
    • 10.3.6.2. The U.K.
    • 10.3.6.3. France
    • 10.3.6.4. Spain
    • 10.3.6.5. Italy
    • 10.3.6.6. Rest of Europe
• 10.4. South America
  • 10.4.1. Introduction
  • 10.4.2. Key Region-Specific Dynamics
  • 10.4.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Class
  • 10.4.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
  • 10.4.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
  • 10.4.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 10.4.6.1. Brazil
    • 10.4.6.2. Argentina
    • 10.4.6.3. Rest of South America
• 10.5. Asia-Pacific
  • 10.5.1. Introduction
  • 10.5.2. Key Region-Specific Dynamics
  • 10.5.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Class
  • 10.5.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
  • 10.5.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
  • 10.5.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 10.5.6.1. China
    • 10.5.6.2. India
    • 10.5.6.3. Japa
    • 10.5.6.4. Australia
    • 10.5.6.5. Rest of Asia-Pacific
• 10.6. Middle East and Africa
  • 10.6.1. Introduction
  • 10.6.2. Key Region-Specific Dynamics
  • 10.6.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Class
  • 10.6.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
  • 10.6.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration

11. Competitive Landscape

• 11.1. Competitive Scenario
• 11.2. Market Positioning/Share Analysis
• 11.3. Mergers and Acquisitions Analysis

12. Company Profiles

• 12.1. BioCryst Pharmaceuticals, Inc*
  • 12.1.1. Company Overview
  • 12.1.2. Product Portfolio and Description
  • 12.1.3. Financial Overview
  • 12.1.4. Key Developments
• 12.2. CSL Behring LLC
• 12.3. Takeda Pharmaceutical Company
• 12.4. Pharming Healthcare, Inc
• 12.5. Sanofi
• 12.6. Attune Pharmaceuticals, Inc
• 12.7. Ionis Pharmaceuticals
• 12.8. KalVista Pharmaceuticals, Inc
• 12.9. Arrowhead Pharmaceuticals
• 12.10. Advanced Technology & Industrial Co., Ltd

LIST NOT EXHAUSTIVE

13. Appendix

• 13.1. About Us and Services
• 13.2. Contact Us