ベータサラセミアの世界市場：2024-2031年

概要

世界のベータサラセミア市場は、2023年に4億8,032万米ドルに達し、2031年には8億8,904万米ドルに達すると予測され、予測期間2024年～2031年のCAGRは8.1%で成長する見込みです。

ベータサラセミアは、ヘモグロビンの生成を減少させる血液疾患です。ヘモグロビンは、全身の細胞に酸素を運ぶ赤血球中の鉄含有タンパク質です。

ベータサラセミア患者では、ヘモグロビンの濃度が低いため、体内の酸素濃度が低下します。また、罹患者は赤血球が不足し（貧血）、青白い肌、脱力感、疲労感、さらに深刻な合併症を引き起こすことがあります。ベータサラセミア患者は、異常血栓を発症するリスクが高いです。

市場促進要因ダイナミクス

ベータサラセミアに対する研究開発とFDA承認の増加

世界のベータサラセミア市場は、最近の様々な研究開発により大きな成長を遂げています。ベータサラセミアは通常、病歴や臨床所見、画像所見に基づいて診断されます。また、ベータサラセミアの治療における先進的な研究開発は、市場の成長を加速させるのに役立っています。

例えば、2023年6月、CRISPR Therapeutics社は、2023年欧州血液学会（EHA）年次総会で発表された輸血依存性ベータサラセミアおよび重症鎌状赤血球症を対象としたExa-celの主要臨床試験の良好な結果を発表しました。Exa-celは、輸血依存性ベータサラセミア（TDT）患者を対象に評価されている、治験中の自己由来生体外CRISPR/Cas9遺伝子編集細胞療法です。

さらに、規制当局による治療認可の増加が市場の成長を後押ししています。FDAから承認を得る過程では、一般的に疾患モデル動物を用いた前臨床実験が行われます。動物モデル実験の結果が良好であれば、FDAは介入研究の構成案を承認することができます。

例えば、2022年8月、Bluebird Bio, Inc.は、米国食品医薬品局（FDA）が、定期的な赤血球（RBC）輸血を必要とする成人および小児患者におけるベータサラセミアの根本的な遺伝的原因を治療するためにカスタムデザインされた1回限りの遺伝子治療薬であるZYNTEGLO（betibeglogene autotemcel）、別名beti-celを承認したと発表しました。このように、上記の要因が市場の成長を後押ししています。

抑制要因

高額な治療費、治療に伴う副作用、厳しい規制ガイドライン、限られた治療オプション、個人の認識不足などの要因が市場の妨げになると予想されます。

このレポートの詳細について- サンプル請求

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
    • ベータサラセミアの研究開発とFDA承認の増加
    • サラセミアの有病率の上昇
  • 抑制要因
    • 治療費の高騰
  • 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
• アンメットニーズ
• 規制分析
• ロシア・ウクライナ戦争影響分析
• DMIの見解

第6章 タイプ別

• ベータサラセミアメジャー
• ベータサラセミアインターメディア
• ベータサラセミアマイナー

第7章 治療法別

• 鉄キレート療法
• 葉酸サプリメント
• 輸血
• 赤血球成熟剤
• 幹細胞移植
• その他

第8章 投与経路別

• 経口
• 静脈内投与
• その他

第9章 エンドユーザー別

• 病院
• クリニック
• その他

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
  • イタリア
  • スペイン
  • 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
  • 韓国
  • 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
• 製品ベンチマーク
• 企業シェア分析
• 主な発展と戦略

第12章 企業プロファイル

• Bristol Myers Squibb
  • 会社概要
  • 製品ポートフォリオと説明
  • 財務概要
  • 主な発展
• Par Pharmaceuticals
• Qilu Pharmaceuticals
• Taro Pharmaceuticals
• Blue Bird Bio
• Cipla Ltd.
• Novartis A.G
• Teva Pharmaceuticals
• Piramal Pharma Solutions
• LGM Pharma

第13章 付録

Overview

The global beta thalassemia market reached US$ 480.32 million in 2023 and is expected to reach US$ 889.04 million by 2031 growing with a CAGR of 8.1% during the forecast period 2024-2031.

Beta thalassemia is a blood disorder that reduces the production of hemoglobin. Hemoglobin is the iron-containing protein in red blood cells that carries oxygen to cells throughout the body.

In people with beta thalassemia, low levels of hemoglobin reduce oxygen levels in the body. Affected individuals also have a shortage of red blood cells (anemia), which can cause pale skin, weakness, fatigue, and more serious complications. People with beta thalassemia are at an increased risk of developing abnormal blood clots.

Market Drivers: Dynamics

Increasing Research & Developments and Rising FDA Approvals for Beta Thalassemia

The global beta thalassemia market has witnessed significant growth due to various recent research and developments. Beta Thalassemia is usually diagnosed based on medical history and clinical and imaging findings. Also, advanced research and developments in treating beta-thalassemia help to accelerate market growth.

For instance, in June 2023, CRISPR Therapeutics announced the positive results from pivotal trials of Exa-cel for transfusion-dependent beta-thalassemia and severe sickle cell disease presented at the 2023 Annual European Hematology Association (EHA) Congress. Exa-cel is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited cell therapy that is being evaluated for patients with transfusion-dependent beta-thalassemia (TDT).

Moreover, the increase in treatment approvals by regulatory bodies helps to drive market growth. The process of getting approval from the FDA generally involves preclinical experiments in an animal model of the disease. If the results of the animal model experiments are favorable then the FDA can approve the proposed structure of an interventional study.

For instance, in August 2022, Bluebird Bio, Inc. announced the U.S. Food and Drug Administration (FDA) has approved ZYNTEGLO (betibeglogene autotemcel) also known as beti-cel, a one-time gene therapy custom-designed to treat the underlying genetic cause of beta-thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions. Thus, the above factors help to boost the market growth.

Restraints

Factors such as the high cost of treatment, side effects associated with the treatment, stringent regulatory guidelines, limited treatment options, and lack of awareness among individuals are expected to hamper the market.

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Segment Analysis

The global beta thalassemia market is segmented based on type, treatment, route of administration, end users, and region.

The stem cell transplant segment accounted for approximately 38.5% of the global beta thalassemia market share

The stem cell transplant segment is expected to hold the largest market share over the forecast period. A stem cell transplant can cure beta-thalassemia. Bone marrow transplantation (BMT) is a medical procedure during which stem cells are transferred from a healthy individual into the blood-producing tissue in the bone marrow of the individual who has a blood disease such as B-thalassemia.

Moreover, government initiatives such as awareness programs, product launches, and approvals would drive this market growth. For instance, in May 2023, the Coal India CSR-funded Hematopoietic Stem Cell Transplant (HSCT) program is a unique initiative aimed at providing a one-time cure opportunity to underprivileged Thalassemia patients who have a matched sibling donor but do not have the financial resources to cover the cost of the procedure.

The program has completed 356 bone marrow transplants for Thalassemia patients across 10 impaneled hospitals in India during the two phases.

Similarly in January 2024, Vertex Pharmaceuticals Incorporated announced that the U.S. Food and Drug Administration (FDA) has approved CASGEVY (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited cell therapy, for the treatment of transfusion-dependent beta-thalassemia (TDT) in patients 12 years and older.

Geographical Analysis

North America accounted for approximately 41.3% of the global beta thalassemia market share

North America region is expected to hold the largest market share over the forecast period owing to the rising number of thalassemia patients in the UK driving this market growth in this region.

For instance, in December 2023, the United Kingdom's National Health Service (NHS) will begin funding allogeneic hematopoietic stem cell transplants for adult patients with beta-thalassemia following new guidance from NHS England's Clinical Priorities Advisory Group.

The NHS estimates that around 600 adults across the U.K. have transfusion-dependent thalassemia - the most severe form of the disease - while around 2,281 people with thalassaemia are registered on the U.K.'s National Haemoglobinopathy. This includes 1,332 patients over the age of 18.

In this region, key player's presence and new product approvals would drive this market growth. For instance, in August 2022, bluebird bio, Inc. announced the FDA has approved ZYNTEGLO (betibeglogene autotemcel), also known as beti-cel, a one-time gene therapy custom-designed to treat the underlying genetic cause of beta-thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions.

Market Segmentation

By Type

• Beta-Thalassemia Major
• Beta-Thalassemia Intermedia
• Beta-Thalassemia Minor

By Treatment

• Iron Chelation Therapy
• Folic Acid Supplements
• Blood Transfusions
• Erythroid Maturation Agents
• Stem Cell Transplant
• Others

By Route of Administration

• Oral
• Intravenous
• Others

By End Users

• Hospital
• Clinics
• Others

By Region

• North America
  • U.S.
  • Canada
  • Mexico
• Europe
  • Germany
  • U.K.
  • France
  • Italy
  • Spain
  • Rest of Europe
• South America
  • Brazil
  • Argentina
  • Rest of South America
• Asia-Pacific
  • China
  • India
  • Japan
  • South Korea
  • Rest of Asia-Pacific
• Middle East and Africa

Competitive Landscape

The major global players in the beta thalassemia market include Bristol Myers Squibb, Par Pharmaceuticals, Qilu Pharmaceuticals, Taro Pharmaceuticals, Blue Bird Bio, Cipla Ltd., Novartis A.G, Teva Pharmaceuticals, Piramal Pharma Solutions, and LGM Pharma among others.

Key Developments

• In April 2023, a non-profit organization named ECHO India teamed up with the Post Graduate Institute of Child Health (PGICH) to launch a nationwide program focused on the Prevention and Control of Beta Thalassemia and Other Hemoglobinopathies.
• In April 2022, Vertex Pharmaceuticals Incorporated in collaboration with CRISPR Therapeutics announced the completion of the rolling Biologics License Applications (BLAs) to the U.S. Food and Drug Administration (FDA) for the investigational treatment exagamglogene autotemcel (exa-cel) for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia.
• In March 2022, Bristol Myers Squibb announced that the U.S. Food and Drug Administration (FDA) has extended the review of the supplemental biologics license application (sBLA) for Reblozyl) for the treatment of anemia in adults with non-transfusion-dependent (NTD) beta-thalassemia.

Why Purchase the Report?

• To visualize the global beta thalassemia market segmentation based on type, treatment, route of administration, end users, 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 global beta thalassemia market-level with all segments.
• PDF report consists of a comprehensive analysis after exhaustive qualitative interviews and an in-depth study.
• Product mapping available as excel consisting of key products of all the major players.

The global beta thalassemia market report would provide approximately 69 tables, 70 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 Type
• 3.2. Snippet by Treatment
• 3.3. Snippet by Route of Administration
• 3.4. Snippet by End Users
• 3.5. Snippet by Region

4. Dynamics

• 4.1. Impacting Factors
  • 4.1.1. Drivers
    • 4.1.1.1. Increasing Research & Developments and Rising FDA Approvals for Beta Thalassemia
    • 4.1.1.2. Rising Prevalence of Thalassemia
  • 4.1.2. Restraints
    • 4.1.2.1. High Cost of the Treatment
  • 4.1.3. Impact Analysis

5. Industry Analysis

• 5.1. Porter's 5 Forces Analysis
• 5.2. Supply Chain Analysis
• 5.3. Unmet Needs
• 5.4. Regulatory Analysis
• 5.5. Russia-Ukraine War Impact Analysis
• 5.6. DMI Opinion

6. By Type

• 6.1. Introduction
  • 6.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
  • 6.1.2. Market Attractiveness Index, By Type
• 6.2. Beta-Thalassemia Major
  • 6.2.1. Introduction
  • 6.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 6.3. Beta-Thalassemia Intermedia
• 6.4. Beta-thalassemia Minor

7. By Treatment

• 7.1. Introduction
  • 7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment
  • 7.1.2. Market Attractiveness Index, By Treatment
• 7.2. Iron Chelation Therapy
  • 7.2.1. Introduction
  • 7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 7.3. Folic Acid Supplements
• 7.4. Blood Transfusions
• 7.5. Erythroid Maturation Agents
• 7.6. Stem Cell Transplant
• 7.7. Others

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. Oral
  • 8.2.1. Introduction
  • 8.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 8.3. Intravenous
• 8.4. Others

9. By End Users

• 9.1. Introduction
  • 9.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By End Users
  • 9.1.2. Market Attractiveness Index, By End Users
• 9.2. Hospitals
  • 9.2.1. Introduction
  • 9.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 9.3. Clinics
• 9.4. Others

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 Type
  • 10.2.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment
  • 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 End Users
  • 10.2.7. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
    • 10.2.7.1. U.S.
    • 10.2.7.2. Canada
    • 10.2.7.3. 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 Type
  • 10.3.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment
  • 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 End Users
  • 10.3.7. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
    • 10.3.7.1. Germany
    • 10.3.7.2. U.K.
    • 10.3.7.3. France
    • 10.3.7.4. Italy
    • 10.3.7.5. Spain
    • 10.3.7.6. 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 Type
  • 10.4.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment
  • 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 End Users
  • 10.4.7. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
    • 10.4.7.1. Brazil
    • 10.4.7.2. Argentina
    • 10.4.7.3. 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 Type
  • 10.5.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment
  • 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 End Users
  • 10.5.7. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
    • 10.5.7.1. China
    • 10.5.7.2. India
    • 10.5.7.3. Japan
    • 10.5.7.4. South Korea
    • 10.5.7.5. 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 Type
  • 10.6.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment
  • 10.6.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Route of Administration
  • 10.6.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End Users

11. Competitive Landscape

• 11.1. Competitive Scenario
• 11.2. Product Benchmarking
• 11.3. Company Share Analysis
• 11.4. Key Developments and Strategies

12. Company Profiles

• 12.1. Bristol Myers Squibb*
  • 12.1.1. Company Overview
  • 12.1.2. Product Portfolio and Description
  • 12.1.3. Financial Overview
  • 12.1.4. Key Developments
• 12.2. Par Pharmaceuticals
• 12.3. Qilu Pharmaceuticals
• 12.4. Taro Pharmaceuticals
• 12.5. Blue Bird Bio
• 12.6. Cipla Ltd.
• 12.7. Novartis A.G
• 12.8. Teva Pharmaceuticals
• 12.9. Piramal Pharma Solutions
• 12.10. LGM Pharma

LIST NOT EXHAUSTIVE

13. Appendix

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