血友病A治療の世界市場-2023年～2030年

概要

血友病A治療の世界市場は2022年に91億米ドルに達し、2023-2030年の予測期間中にCAGR 5％で成長し、2030年には133億米ドルに達すると予測されています。この疾患の原因となる既存の遺伝子変異を修正することを目的とした遺伝子治療の進歩へのシフトなどの動向は、世界の血友病A治療市場を独占すると予想されます。

世界の血友病A治療市場は近年大きく成長しており、今後も上昇傾向が続くと予測されます。半減期延長製剤へのシフト、バイオシミラーやジェネリック医薬品のイントロダクション、非代替療法の出現など、いくつかの重要な動向の影響を受け、市場は変革期を迎えています。

さらに、血友病有病率の上昇、治療法の進歩、臨床試験数の増加が血友病A治療市場規模を押し上げています。血友病A治療のための研究活動の増加、確立されたバイオ医薬品および製薬会社の存在は、北米地域の需要促進要因の一部です。武田薬品工業、SANOFI CORPORATION、Genentech USA, Inc.など、その他の競合企業も積極的に市場に参入しています。

ダイナミクス

血友病Aの有病率の増加が、血友病A治療市場の成長を牽引

世界の血友病Aの有病率の増加は、血友病A治療産業の発展と変革の重要な原動力です。血友病Aは、血液凝固第VIII因子の欠損を特徴とする遺伝性の出血性疾患です。血液凝固第VIII因子の濃度が低いと、出血を防ぐために血液が正しく固まりません。

例えば、National Librabry of Medicine 2023の記事によると、血友病Aは最も一般的な遺伝性止血疾患であり、男性5,000人に1人が罹患し、血友病患者の80％を占めています。血友病Aは世界中で約40万人の男性が罹患しており、その多くが低開発国で診断されていません。そのため、半減期が延長された凝固因子製剤、遺伝子治療、個別化治療などの技術革新を含む研究開発への取り組みが強化され、効果的な治療が必要とされています。その結果、予測期間中の市場を牽引することになります。

遺伝子治療の進歩が血友病A治療市場の成長を牽引する

遺伝子治療の急速な進歩は血友病A治療市場の成長軌道の重要な促進要因です。遺伝子治療は、血友病Aの原因である欠陥遺伝子をベクターで固定し置換する1回限りの点滴を含む新しいアプローチであり、長期的あるいは治癒的な解決策の可能性を示しています。

遺伝子治療を用いた血友病A治療のFDA承認は、市場成長に大きな影響を与えると思われます。例えば、米国食品医薬品局は2023年6月、FDAが承認した検査でアデノ随伴ウイルス血清型5に対する抗体が検出されない重度の血友病Aの成人患者に対する治療薬として、アデノ随伴ウイルスベクターを用いた遺伝子治療薬Roctavianを承認しました。このように、遺伝子治療における新興国市場の発展が、予測期間中に市場を牽引するものと思われます。

血友病A治療に伴う高コストが市場の成長を妨げる

多数の血友病治療の選択肢があることを考慮すると、薬価の高騰は血友病A市場にとって大きな抑制要因です。さらに、血液因子製剤には費用対効果の高いバイオシミラー製剤がないことも、この価格問題を助長しています。

例えば、DDAVP注射液（4mcg/mL）は10ミリリットル入りで約864ドルであり、経済的負担が大きいことがわかります。この経済的障壁は、特に医療予算が限られている地域では、治療へのアクセスを制限する可能性があります。必要な血友病A治療薬への十分なアクセスを確保するためには、このような価格設定の問題に対処することが不可欠です。

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
    • 血友病Aの有病率の上昇
    • 遺伝子治療の進歩
  • 抑制要因
    • 血友病A治療に伴う高コスト
  • 機会
    • 個別化医療への需要の高まり
  • 影響分析

第5章 産業分析

• ポーターのファイブフォース分析
• 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 治療タイプ別

• 因子補充療法
  • 遺伝子組換え第VIII因子製剤
  • 血漿由来第VIII因子製剤
• 非因子補充療法
  • エミシズマブ
  • デスモプレシン
  • アミノカプロン酸
• 遺伝子治療

第8章 投与経路別

• 静脈内
• 経口
• 経鼻

第9章 重症度別

• 軽度血友病A
• 中度血友病A
• 重度血友病A

第10章 流通チャネル別

• 病院薬局
• 小売薬局
• オンライン薬局

第11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
• 中東・アフリカ

第12章 競合情勢

• 競合シナリオ
• 市況/シェア分析
• M&A分析

第13章 企業プロファイル

• Takeda Pharmaceutical Company Limited
  • 企業概要
  • 製品ポートフォリオと説明
  • 財務概要
  • 主な動向
• SANOFI CORPORATION
• Genentech USA, Inc.
• Novo Nordisk
• Pfizer Inc.
• CSL Behring LLC.
• Grifols
• Sagent Pharmaceuticals
• Octapharma USA Inc.
• UBI Pharma Inc.

第14章 付録

Overview

Global Hemophilia A Treatment Market reached US$ 9.1 billion in 2022 and is expected to reach US$ 13.3 billion by 2030 growing with a CAGR of 5% during the forecast period 2023-2030. The trend such as the shift towards gene therapy advancements that aims to correct existing gene mutations responsible for this condition is expected to dominate the global hemophilia A treatment market.

The global hemophilia A treatment market has grown significantly in recent years and is projected to continue on its upward trend. The market is undergoing a transformational period, influenced by several significant trends such shift towards extended half-life products, introduction of biosimilar and generics, and emergence of non-replacement therapies.

Furthermore, the rising prevalence of hemophilia, increasing advancements in treatments, increasing number of clinical trials are driving up the hemophilia A treatment market size. The growing research activities for treating hemophilia A and presence of establish biopharmaceutical and pharmaceutical companies are some of the factors in demand from North American regions. With significant competitors like Takeda Pharmaceutical Company Limited, SANOFI CORPORATION, Genentech USA, Inc., and others actively operating in the market.

Dynamics

The Growing Prevalence of Hemophilia A to Drive the Growth of the Hemophilia A Treatment Market

The growing global prevalence of hemophilia A is a crucial driving force in the development and transformation of the hemophilia A treatment industry. Hemophilia A is a hereditary bleeding condition characterized by a deficiency in the clotting factor VIII. Blood doesn't clot correctly to prevent bleeding when factor VIII levels are low.

For instance, according to National Librabry of Medicine 2023 article, Hemophilia A, the most common hereditary hemostasis condition, affects one in every 5,000 males and accounts for 80% of hemophilia cases. Hemophilia A affects around 400000 males worldwide, with many going undiagnosed in underdeveloped countries. Thus, many people going undiagnosed there is need of effective treatment with increased R&D efforts, including innovations such as extended half-life clotting factor products, gene therapies, and personalized treatments. Thereby, driving the market over the forecast period.

The Advancements in Gene Therapies will Drive the Hemophilia A Treatment Market Growth

Rapid advances in gene therapies are a critical driver of the hemophilia A therapy market's growth trajectory. Gene treatments, a novel approach that involves one-time infusion that use a vector to fix and replace the defective gene responsible for hemophilia A, showing a possibility of long-term or even curative solutions.

The FDA approvals for hemophilia A treatment using gene therapy will create a major impact on the market growth. For instance, in June 2023, U.S. Food and Drug Administration approved Roctavian, an adeno-associated virus vector-based gene therapy for the treatment of adults with severe hemophilia A without pre-existing antibodies to adeno-associated virus serotype 5 detected by an FDA-approved test. Thus, owing to the developing advancement undertaking in gene therapy to drive the market over the forecast period.

The High Cost Associated with the Hemophilia A Treatment Will Hamper the Growth of the Market

The high cost of medication is a significant restraint for the hemophilia A market, Considering the availability of a number of hemophilia treatment choices, each medicine maintains its individual branding, resulting in elevated price ranges. Furthermore, the lack of cost-effective biosimilar options for blood factor medications contributes to this pricing issue.

For example, the DDAVP injectable solution (4 mcg/mL) costs roughly around $864 for a 10 milliliter supply, showing the financial strain. This financial barrier may limit treatment accessibility, particularly in regions with limited healthcare budgets. Addressing these pricing problems is essential for ensuring adequate access to necessary hemophilia A medications.

Segment Analysis

The global hemophilia A treatment market is segmented based on treatment type, route of administration, severity type, distribution channel, and region.

Owing to the Proven Efficacy, the Factor Replacement Therapy Segment Accounted for Approximately 52.2% of the Hemophilia A Treatment Market Share

In treatment type, the factor replacement therapy segment is expected to dominate the market, due to proven efficacy contributing to its significance. The factor replacement therapy consist of treatment such as recombinant factor VIII and plasma-derived Factor VIII. This therapy entails the administration of synthetic clotting factor VIII to replace inadequate clotting factor in hemophilia patients leadingto increased in amount of factor in the body to levels that lead to better clotting, and therefore less bleeding.

This treatment remains a cornerstone in managing hemophilia A owing to increasing FDA approvals. For instance, in February 2023, the Food and Drug Administration approved Sanofi SA's therapy to treat hemophilia A, and is expects to launch it in the U.S in April. Moreover, this Sanofi's replacement medicine, Altuviiio, is entering a market dominated by competitors such as Takeda Pharmaceutical, Bayer AG, and Novo Nordisk, which produce factor replacement therapies that have been the conventional treatment for decades. Thus, due to better efficacy and increasing FDA approval the segment is expected to dominate over the forecast period.

Geographical Penetration

North America Accounted for Approximately 39.2% of the Market Share in 2022, Owing to the Increasing Number of Clinical Trials

North America, particularly the U.S., dominates the global hemophilia A treatment market due to growing number of clinical trials, and increasing research activities by major organization in the region is expected to create a major impact on this market. For instance, in May 2023, Pfizer Inc. stated that the pivotal Phase 3 BASIS clinical trial testing marstacimab (NCT03938792) accomplished its primary objectives, demonstrating statically significant and clinically relevant results.

Furthermore, Marstacimab, a novel, investigational anti-tissue factor pathway inhibitor (anti-TFPI) being studied for the treatment of hemophilia A or B in people who do not have inhibitors to Factor VIII (FVIII) or Factor IX (FIX), was administered weekly in the trial as a subcutaneous 300 mg loading dose followed by 150 mg once weekly. Thus, owing to above factors the region is expected to grow over the forecast period.

Competitive Landscape

The major global players in the hemophilia A treatment market include: Takeda Pharmaceutical Company Limited, SANOFI CORPORATION, Genentech USA, Inc., Novo Nordisk, Pfizer Inc., CSL Behring LLC., Grifols, Sagent Pharmaceuticals, Octapharma USA Inc., UBI Pharma Inc., and among others.

COVID-19 Impact Analysis

The COVID-19 pandemic has had a significant impact on the hemophilia A treatment market, revealing the connection between the virus and the disorder through increased factor VIII activity in critically ill COVID-19 patients. This connection has resulted in an increase in hospitalizations and bleeding rates in people with hemophilia A and COVID-19, demanding careful attention to bleeding episode therapy. Additionally, COVID-19 management methods such as thromboprophylaxis might come into use in hemophilia A treatment. COVID-19-induced inflammatory response complicates factor VIII levels in mild hemophilia A cases, altering replacement therapy considerations.

Adaptable approaches must be developed to address the pandemic's broader healthcare implications on hemophilia A care. Particularly, rare occurrences of acquired Hemophilia A following COVID-19 infection and immunization indicate the importance of continued monitoring for hemophilia A patients. In the complex interplay between COVID-19 and hemophilia A, a comprehensive approach is required to handle both problems and potential opportunities.

By Treatment Type

• Factor Replacement Therapy
  • Recombinant Factor VIII
  • Plasma-derived Factor VIII

• Non-Factor Replacement Therapy
  • Emicizumab
  • Desmopressin
  • Aminocaproic Acid

• Gene Therapy

By Route of Administration

• Intravenous
• Oral
• Nasal

By Severity Type

• Mild Hemophilia A
• Moderate Hemophilia A
• Severe Hemophilia A

By Distribution Channel

• Hospital Pharmacies
• Retail Pharmacies
• Online Pharmacies

By Region

• North America
  • U.S.
  • Canada
  • Mexico

• Europe
  • Germany
  • U.K.
  • France
  • Spain
  • Italy
  • Rest of Europe

• South America
  • Brazil
  • Argentina
  • Rest of South America

• Asia-Pacific
  • China
  • India
  • Japan
  • Australia
  • Rest of Asia-Pacific

• Middle East and Africa

Key Developments

• On July 4, 2023, Chugai Pharmaceutical Co., Ltd. launched Hemlibra, a medication developed by Chugai for routine prophylaxis of bleeding episodes in individuals with hemophilia A who do not use factor VIII inhibitors.
• On June 30, 2023, The U.S. FDA approved BioMarin Pharmaceutical's (BMRN.O) gene therapy for severe hemophilia A, This will patients with inherited bleeding disorder an option for regular injections of missing blood proteins.
• On March 1, 2023, 2seventy bio, Inc., a leading immuno-oncology cell therapy company, announced a $15 million preclinical milestone as part of a joint research and development collaboration focusing on an in vivo gene editing treatment for hemophilia A.

DMI Opinion

The global hemophilia A treatment market is being driven by trends such as the growing importance of gene therapy advancements that focus on fixing the genetic mutations that cause this condition. This market is undergoing a transformation, with longer half-life drugs, biosimilars, generics, and non-replacement therapies all becoming accessible.

The increasing global prevalence of hemophilia A is a major problem, with approximately 1 in 5,000 males affected by this genetic bleeding illness, emphasizing the need for improved therapies, especially considering the high number of untreated cases. Gene treatments, such as Roctavian, that have received FDA approval, provide therapeutic potential. Despite this growth, the market faces the difficulty of high treatment costs as a result of branding and a lack of cost-effective options.

The treatment landscape is driven by the dominance of factor replacement therapy, particularly recombinant factor VIII and plasma-derived Factor VIII, with North America, particularly the United States, commanding significant shares due to clinical trials and research initiatives. The link between the virus and hemophilia A becomes evident in the course of the COVID-19 pandemic, demanding precise management techniques and emphasizing the need for extensive and adaptable ways to address both challenges and potential.

Why Purchase the Report?

• To visualize the global hemophilia A treatment market segmentation based on treatment type, route of administration, severity type, distribution channel 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 hemophilia A treatment 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 in excel consisting of key products of all the major players.

The global hemophilia A treatment 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 Treatment Type
• 3.2. Snippet by Route of Administration
• 3.3. Snippet by Severity Type
• 3.4. Snippet by Distribution Channel
• 3.5. Snippet by Region

4. Dynamics

• 4.1. Impacting Factors
  • 4.1.1. Drivers
    • 4.1.1.1. The Rising Prevalence of Hemophilia A
    • 4.1.1.2. The Advancements in Gene Therapies
  • 4.1.2. Restraints
    • 4.1.2.1. The High Cost Associated with the Hemophilia A Treatment A
  • 4.1.3. Opportunity
    • 4.1.3.1. Increasing Demand for Personalized Medicine
  • 4.1.4. 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 Treatment Type

• 7.1. Introduction
  • 7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
  • 7.1.2. Market Attractiveness Index, By Treatment Type
• 7.2. Factor Replacement Therapy*
  • 7.2.1. Introduction
  • 7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
    • 7.2.2.1. Recombinant Factor VIII
    • 7.2.2.2. Plasma-derived Factor VIII
• 7.3. Non-Factor Replacement Therapies
  • 7.3.1. Emicizumab
  • 7.3.2. Desmopressin
  • 7.3.3. Aminocaproic Acid
• 7.4. Gene Therapy

8. By Route of Administration

• 8.1. Introduction
  • 8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
  • 8.1.2. Market Attractiveness Index, By Route of Administration
• 8.2. Intravenous *
  • 8.2.1. Introduction
  • 8.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 8.3. Oral
• 8.4. Nasal

9. By Severity Type

• 9.1. Introduction
  • 9.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Severity Type
  • 9.1.2. Market Attractiveness Index, By Severity Type
• 9.2. Mild Hemophilia A*
  • 9.2.1. Introduction
  • 9.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 9.3. Moderate Hemophilia A
• 9.4. Severe Hemophilia A

10. By Distribution Channel

• 10.1. Introduction
  • 10.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
  • 10.1.2. Market Attractiveness Index, By Distribution Channel
• 10.2. Hospitals Pharmacies*
  • 10.2.1. Introduction
  • 10.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 10.3. Retail Pharmacies
• 10.4. Online Pharmacies

11. By Region

• 11.1. Introduction
  • 11.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Region
  • 11.1.2. Market Attractiveness Index, By Region
• 11.2. North America
  • 11.2.1. Introduction
  • 11.2.2. Key Region-Specific Dynamics
  • 11.2.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
  • 11.2.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
  • 11.2.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Severity Type
  • 11.2.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
  • 11.2.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 11.2.7.1. U.S.
    • 11.2.7.2. Canada
    • 11.2.7.3. Mexico
• 11.3. Europe
  • 11.3.1. Introduction
  • 11.3.2. Key Region-Specific Dynamics
  • 11.3.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
  • 11.3.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
  • 11.3.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Severity Type
  • 11.3.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
  • 11.3.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 11.3.7.1. Germany
    • 11.3.7.2. U.K.
    • 11.3.7.3. France
    • 11.3.7.4. Spain
    • 11.3.7.5. Italy
    • 11.3.7.6. Rest of Europe
• 11.4. South America
  • 11.4.1. Introduction
  • 11.4.2. Key Region-Specific Dynamics
  • 11.4.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
  • 11.4.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
  • 11.4.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Severity Type
  • 11.4.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
  • 11.4.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 11.4.7.1. Brazil
    • 11.4.7.2. Argentina
    • 11.4.7.3. Rest of South America
• 11.5. Asia-Pacific
  • 11.5.1. Introduction
  • 11.5.2. Key Region-Specific Dynamics
  • 11.5.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
  • 11.5.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
  • 11.5.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Severity Type
  • 11.5.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
  • 11.5.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 11.5.7.1. China
    • 11.5.7.2. India
    • 11.5.7.3. Japan
    • 11.5.7.4. Australia
    • 11.5.7.5. Rest of Asia-Pacific
• 11.6. Middle East and Africa
  • 11.6.1. Introduction
  • 11.6.2. Key Region-Specific Dynamics
  • 11.6.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
  • 11.6.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
  • 11.6.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Severity Type
  • 11.6.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel

12. Competitive Landscape

• 12.1. Competitive Scenario
• 12.2. Market Positioning/Share Analysis
• 12.3. Mergers and Acquisitions Analysis

13. Company Profiles

• 13.1. Takeda Pharmaceutical Company Limited*
  • 13.1.1. Company Overview
  • 13.1.2. Product Portfolio and Description
  • 13.1.3. Financial Overview
  • 13.1.4. Key Developments
• 13.2. SANOFI CORPORATION
• 13.3. Genentech USA, Inc.
• 13.4. Novo Nordisk
• 13.5. Pfizer Inc.
• 13.6. CSL Behring LLC.
• 13.7. Grifols
• 13.8. Sagent Pharmaceuticals
• 13.9. Octapharma USA Inc.
• 13.10. UBI Pharma Inc.

LIST NOT EXHAUSTIVE

14. Appendix

• 14.1. About Us and Services
• 14.2. Contact Us