鎌状赤血球症治療の世界市場規模：タイプ別、薬剤クラス別、エンドユーザー別、地域範囲別および予測

鎌状赤血球病治療の市場規模と予測

鎌状赤血球症治療市場規模は、2024年には33億米ドルと評価され、2026～2032年にかけて16.85%のCAGRで成長し、2032年には103億米ドルに達すると予測されます。

• 鎌状赤血球症（SCD）は、ヘモグロビンの異常によって赤血球が鎌のような形態になることを特徴とする遺伝性の血液疾患です。これにより血管が閉塞し、疼痛、感染症、臓器障害を引き起こします。
• SCDの治療には、骨髄移植、輸血、症状の管理と合併症の予防を目的とした薬剤療法などがあります。骨髄移植は唯一の治療法ですが、ドナーの適合性の問題から、その可能性は限られており、ほとんどの患者にとって薬剤療法が不可欠となっています。
• 鎌状赤血球症治療の将来は、遺伝子治療や根治的治療法の進歩により、長期的な疾患管理と患者の予後改善への希望がもたらされつつあります。

世界の鎌状赤血球症治療市場力学

世界の鎌状赤血球症治療市場を形成している主要市場力学は以下の通りです。

主要市場促進要因

• 遺伝子治療の進歩：遺伝子治療の進歩：遺伝子治療の開発は、鎌状赤血球症の原因となる遺伝子変異を標的とすることで、鎌状赤血球症の治療に大きな可能性をもたらします。CRISPRやその他の遺伝子編集技術の進歩は、患者に治癒の選択肢を提供し、治療の展望に革命をもたらすと予想されます。
• 鎌状赤血球症の有病率の上昇：特にサハラ以南のアフリカ、中東、インドの一部では鎌状赤血球症の患者数が増加しており、効果的な治療法に対する需要が高まっています。疾病のスクリーニングと早期診断を目的とした公衆衛生上の取り組みが市場の成長を後押ししています。
• 政府と非政府組織（NGO）の取り組み：治療へのアクセスを提供し、認知度を向上させ、新しい治療法の研究に資金を提供するための政府やNGOによる取り組みの増加が、治療市場を支えています。発展途上地域における骨髄登録の増加や薬剤療法へのアクセスを目的としたプログラムも開発に貢献しています。

主要課題

• 治療費の高騰：骨髄移植や遺伝子治療などの治療は高額であるため、低所得地域の患者が治療を受けられる環境は限られています。さらに、SCDの長期管理には定期的な輸血や薬剤療法が必要であり、患者やヘルスケアシステムの経済的負担をさらに増大させています。
• 骨髄移植への限られたアクセス：骨髄移植はSCDを治癒させる可能性を秘めていますが、適合するドナーを見つけることは重要な課題です。患者とドナーの遺伝的一致が必要であるため、特にヘルスケアインフラが限られている地域では、移植の利用が制限されています。
• 開発途上地域における認識とヘルスケアへのアクセスの欠如：低所得地域では、多くの患者が包括的なヘルスケアサービスを受けることができず、診断や治療が困難です。農村部ではSCDの認知度が低いため、診断が遅れ、罹患率や死亡率が高くなります。

主要動向

• 遺伝子治療と治療的アプローチ：遺伝子治療の研究開発により、SCDの根治的治療への道が開かれつつあります。現在、SCDの原因遺伝子を修正するための臨床検査が進行中であり、終生治療に頼っている患者に新たな希望をもたらしています。
• 新しい薬剤療法の導入：SCDに対する薬剤開発は進んでおり、VoxelotorやL-グルタミンなどの新しい治療は、痛みを伴うクリーゼの頻度を減らし、ヘモグロビン濃度を改善することで承認を得ています。これらの治療法はSCD患者の生活の質の向上を目指しています。
• 世界ヘルスへの取り組み：政府、NGO、医療機関は、特に疾病負担の大きいアフリカやインドにおいて、SCD治療へのアクセスを向上させるため、世界ヘルスへの取り組みを強化しています。このようなイニシアチブは、認知度、スクリーニング、治療法の改善を目的としています。

目次

第1章 世界の鎌状赤血球症治療市場の導入

• 市場概要
• 調査範囲
• 前提条件

第2章 エグゼクティブサマリー

第3章 VERIFIED MARKET RESEARCHの調査手法

• データマイニング
• バリデーション
• 一次資料
• データソース一覧

第4章 世界の鎌状赤血球症治療市場展望

• 概要
• 市場力学
  • 促進要因
  • 抑制要因
  • 機会
• ポーターのファイブフォースモデル
• バリューチェーン分析

第5章 鎌状赤血球症治療の世界市場：タイプ別

• 概要
• 骨髄移植
• 輸血

第6章 鎌状赤血球症治療の世界市場：薬剤クラス別

• 概要
• ヒドロキシ尿素
• L-グルタミン
• ボクセロトール

第7章 鎌状赤血球症治療の世界市場：エンドユーザー別

• 概要
• 病院
• 専門クリニック

第8章 鎌状赤血球症治療の世界市場：地域別

• 概要
• 北米
  • 米国
  • カナダ
  • メキシコ
• 欧州
  • ドイツ
  • フランス
  • スペイン
  • イタリア
  • 英国
  • その他の欧州
• アジア太平洋
  • 中国
  • 日本
  • インド
  • 韓国
  • オーストラリア
  • その他のアジア太平洋
• その他
  • ラテンアメリカ
  • 中東・アフリカ

第9章 世界の鎌状赤血球症治療市場の競合情勢

• 概要
• 各社の市場ランキング
• 主要開発戦略

第10章 企業プロファイル

• Global Blood Therapeutics, Inc.
• Novartis AG
• Pfizer Inc.
• Emmaus Life Sciences, Inc.
• Bluebird Bio, Inc.
• Sangamo Therapeutics, Inc.
• CRISPR Therapeutics AG.

第11章 主要開発

• 製品上市/開発
• 合併と買収
• 事業拡大
• パートナーシップと提携

第12章 付録

• 関連調査

Sickle Cell Disease Treatment Market Size And Forecast

Sickle Cell Disease Treatment Market size was valued at USD 3.3 Billion in 2024 and is projected to reach USD 10.3 Billion by 2032, growing at a CAGR of 16.85% from 2026 to 2032.

• Sickle Cell Disease (SCD) is an inherited blood disorder characterized by abnormal hemoglobin, resulting in red blood cells adopting a sickle-like shape. This leads to blockages in blood vessels causing pain, infections, and organ damage.
• Treatments for SCD include bone marrow transplants, blood transfusions, and drug therapies aimed at managing the symptoms and preventing complications. While bone marrow transplants are the only potential cure, their availability is limited due to donor compatibility issues making drug treatments essential for most patients.
• The future of SCD treatment is evolving with advancements in gene therapies and curative treatments offering hope for long-term disease management and improved patient outcomes.

Global Sickle Cell Disease Treatment Market Dynamics

The key market dynamics that are shaping the global sickle cell disease treatment market include:

Key Market Drivers:

• Advancements in Gene Therapy: The development of gene therapies offers significant potential for treating SCD by targeting the genetic mutations that cause the disease. Breakthroughs in CRISPR and other gene-editing technologies are anticipated to revolutionize the treatment landscape providing curative options for patients.
• Rising Prevalence of Sickle Cell Disease: The growing number of SCD cases, particularly in sub-Saharan Africa, the Middle East, and parts of India is increasing the demand for effective treatment solutions. Public health initiatives aimed at screening and diagnosing the disease early are boosting market growth.
• Government and Non-Governmental Organization (NGO) Initiatives: Increased efforts by governments and NGOs to provide access to care, improve awareness, and fund research for new therapies are supporting the treatment market. Programs aimed at increasing bone marrow registries and access to drug therapies in developing regions are also contributing to market expansion.

Key Challenges:

• High Costs of Treatment: Treatments such as bone marrow transplants and gene therapies are expensive limiting their accessibility to patients in lower-income regions. Additionally, long-term management of SCD requires regular blood transfusions and drug therapies, further increasing the economic burden on patients and healthcare systems.
• Limited Access to Bone Marrow Transplants: While bone marrow transplants offer the potential for curing SCD, finding compatible donors remains a significant challenge. The need for genetic matches between patients and donors restricts the availability of transplants, particularly in regions with limited healthcare infrastructure.
• Lack of Awareness and Healthcare Access in Developing Regions: In low-income regions, many patients lack access to comprehensive healthcare services, making diagnosis and treatment difficult. Limited awareness of SCD in rural areas results in delayed diagnosis leading to higher morbidity and mortality rates.

Key Trends:

• Gene Therapy and Curative Approaches: Research and development in gene therapies are paving the way for potentially curative treatments for SCD. Clinical trials are ongoing to determine the efficacy of these therapies in modifying or correcting the faulty gene responsible for SCD offering new hope for patients who currently rely on lifelong treatments.
• Introduction of New Drug Therapies: Drug development for SCD is progressing, with new therapies such as voxelotor and L-glutamine gaining approval for reducing the frequency of painful crises and improving hemoglobin levels. These therapies are aimed at improving the quality of life for SCD patients.
• Global Health Initiatives: Governments, NGOs, and healthcare organizations are increasing their focus on global health initiatives to ensure better access to SCD treatments, particularly in Africa and India, where the disease burden is high. These initiatives aim to improve awareness, screening, and treatment availability.

Global Sickle Cell Disease Treatment Market Regional Analysis

Here is a more detailed regional analysis of the global sickle cell disease treatment market

North America:

• North America dominates the global sickle cell disease treatment market, with the United States holding the largest market share. This is driven by a well-established healthcare infrastructure, advancements in gene therapies, and increased funding for research and development. The U.S. government, through the National Institutes of Health (NIH), has initiated various programs to support SCD treatment research and improve patient care. Additionally, the availability of advanced treatments such as gene therapy and new drug approvals is contributing to market growth.
• Organizations such as the National Institutes of Health (NIH) and the Centers for Disease Control and Prevention (CDC) are actively involved in research and awareness campaigns to address the issues presented by SCD. There is also extensive collaboration between pharmaceutical corporations and research institutions to produce more effective medicines such as voxelotor and crizanlizumab which have just received FDA approval. With higher awareness, improved diagnoses, and increased treatment options, the North American market is positioned to expand further meeting the patient population's unmet requirements and enhancing the overall quality of life for those living with sickle cell disease.

Asia Pacific:

• The Asia-Pacific region is witnessing significant growth in the SCD treatment market driven by increasing awareness of the disease and improving healthcare infrastructure. Countries like India and Pakistan have high incidences of SCD, with public health programs being implemented to improve screening and diagnosis. Government initiatives aimed at increasing access to bone marrow transplants and affordable drug therapies are expected to fuel market growth. Additionally, rising investments in healthcare and partnerships with global health organizations are improving the availability of advanced treatment options in the region.
• Advances in treatment choices and technologies are changing the SCD treatment landscape in Asia Pacific. Traditional treatments, such as hydroxyurea, blood transfusions, and pain management medicines are being supplemented with new therapies, such as gene therapy and developing biologics with the potential to greatly enhance quality of life. Clinical trials for novel medicines are gaining traction, as pharmaceutical companies engage in R&D to bring viable cures to market. Furthermore, public-private partnerships and collaborations encourage the delivery of vital pharmaceuticals and access to comprehensive care.

Global Sickle Cell Disease Treatment Market: Segmentation Analysis

The Global Sickle Cell Disease Treatment Market is segmented based on Type, Drug Class, End-User, and Geography.

Sickle Cell Disease Treatment Market, By Type

• Bone Marrow Transplant
• Blood Transfusion

Based on Type, the Global Sickle Cell Disease Treatment Market is bifurcated into Bone Marrow Transplant and Blood Transfusion. The bone marrow transplant segment is dominant due to potential curative options for sickle cell disease (SCD), though it is limited by the availability of compatible donors. Blood Transfusion is a widely used treatment method, particularly for managing acute symptoms and preventing complications such as stroke and organ damage in SCD patients.

Sickle Cell Disease Treatment Market, By Drug Class

• Hydroxyurea
• L-glutamine
• Voxelotor

Based on Drug Class, the Global Sickle Cell Disease Treatment Market is segmented into Hydroxyurea, L-glutamine, and Voxelotor. Hydroxyurea is the most commonly used medication for SCD, significantly reducing the frequency of painful episodes and the need for blood transfusions. Voxelotor and L-glutamine are newer drug therapies aimed at improving hemoglobin levels and reducing the severity of disease symptoms providing additional treatment options for patients.

Sickle Cell Disease Treatment Market, By End-User

• Hospitals
• Specialty Clinics

Based on the End-User, the Global Sickle Cell Disease Treatment Market is bifurcated into Hospitals and Specialty Clinics. Hospitals dominate the market as they provide comprehensive treatment options including blood transfusions, bone marrow transplants, and access to specialized medications. Specialty Clinics focus on long-term care management for SCD patients offering personalized treatment plans and outpatient care for ongoing monitoring.

Key Players

• The Global Sickle Cell Disease Treatment Market study report provides valuable insights with an emphasis on the global market. The major players in the market include Global Blood Therapeutics, Inc., Novartis AG, Pfizer Inc., Emmaus Life Sciences, Inc., Bluebird Bio, Inc., Sangamo Therapeutics, Inc., and CRISPR Therapeutics AG.

Our market analysis also includes a section solely dedicated to these major players, offering insights into their financial statements, product benchmarking, and SWOT analysis. The competitive landscape section covers key development strategies, market share, and ranking analysis of the above-mentioned players globally.

Global Sickle Cell Disease Treatment Market Key Developments

• In March 2024, Global Blood Therapeutics received FDA approval for its drug voxelotor, which is designed to improve hemoglobin levels and reduce complications in SCD patients.
• In August 2024, Novartis announced positive results from its clinical trials of gene therapy for SCD, showing promising results in reducing the need for blood transfusions.
• In October 2024, Bluebird Bio announced advancements in its gene-editing therapy using CRISPR technology, with early trials showing potential for curative treatment in SCD patients.

TABLE OF CONTENTS

1 INTRODUCTION OF GLOBAL SICKLE CELL DISEASE TREATMENT MARKET

• 1.1 Overview of the Market
• 1.2 Scope of Report
• 1.3 Assumptions

2 EXECUTIVE SUMMARY

3 RESEARCH METHODOLOGY OF VERIFIED MARKET RESEARCH

• 3.1 Data Mining
• 3.2 Validation
• 3.3 Primary Interviews
• 3.4 List of Data Sources

4 GLOBAL SICKLE CELL DISEASE TREATMENT MARKET OUTLOOK

• 4.1 Overview
• 4.2 Market Dynamics
  • 4.2.1 Drivers
  • 4.2.2 Restraints
  • 4.2.3 Opportunities
• 4.3 Porters Five Force Model
• 4.4 Value Chain Analysis

5 GLOBAL SICKLE CELL DISEASE TREATMENT MARKET, BY TYPE

• 5.1 Overview
• 5.2 Bone Marrow Transplant
• 5.3 Blood Transfusion

6 GLOBAL SICKLE CELL DISEASE TREATMENT MARKET, BY DRUG CLASS

• 6.1 Overview
• 6.2 Hydroxyurea
• 6.3 L-glutamine
• 6.4 Voxelotor

7 GLOBAL SICKLE CELL DISEASE TREATMENT MARKET, BY END-USER

• 7.1 Overview
• 7.2 Hospitals
• 7.3 Specialty Clinics

8 GLOBAL SICKLE CELL DISEASE TREATMENT MARKET, BY GEOGRAPHY

• 8.1 Overview
• 8.2 North America
  • 8.2.1 U.S.
  • 8.2.2 Canada
  • 8.2.3 Mexico
• 8.3 Europe
  • 8.3.1 Germany
  • 8.3.2 France
  • 8.3.3 Spain
  • 8.3.4 Italy
  • 8.3.5 U.K.
  • 8.3.6 Rest of Europe
• 8.4 Asia Pacific
  • 8.4.1 China
  • 8.4.2 Japan
  • 8.4.3 India
  • 8.4.4 South Korea
  • 8.4.5 Australia
  • 8.4.6 Rest of Asia Pacific
• 8.5 Rest of the World
  • 8.5.1 Latin America
  • 8.5.2 Middle East and Africa

9 GLOBAL SICKLE CELL DISEASE TREATMENT MARKET COMPETITIVE LANDSCAPE

• 9.1 Overview
• 9.2 Company Market Ranking
• 9.3 Key Development Strategies

10 COMPANY PROFILES

• 10.1 Global Blood Therapeutics, Inc.
  • 10.1.1 Overview
  • 10.1.2 Financial Performance
  • 10.1.3 Product Outlook
  • 10.1.4 Key Developments
• 10.2 Novartis AG
  • 10.2.1 Overview
  • 10.2.2 Financial Performance
  • 10.2.3 Product Outlook
  • 10.2.4 Key Developments
• 10.3 Pfizer Inc.
  • 10.3.1 Overview
  • 10.3.2 Financial Performance
  • 10.3.3 Product Outlook
  • 10.3.4 Key Developments
• 10.4 Emmaus Life Sciences, Inc.
  • 10.4.1 Overview
  • 10.4.2 Financial Performance
  • 10.4.3 Product Outlook
  • 10.4.4 Key Developments
• 10.5 Bluebird Bio, Inc.
  • 10.5.1 Overview
  • 10.5.2 Financial Performance
  • 10.5.3 Product Outlook
  • 10.5.4 Key Developments
• 10.6 Sangamo Therapeutics, Inc.
  • 10.6.1 Overview
  • 10.6.2 Financial Performance
  • 10.6.3 Product Outlook
  • 10.6.4 Key Developments
• 10.7 CRISPR Therapeutics AG.
  • 10.7.1 Overview
  • 10.7.2 Financial Performance
  • 10.7.3 Product Outlook
  • 10.7.4 Key Developments

11 KEY DEVELOPMENTS

• 11.1 Product Launches/Developments
• 11.2 Mergers and Acquisitions
• 11.3 Business Expansions
• 11.4 Partnerships and Collaborations

12 Appendix

• 12.1 Related Research