한글목차

개요

세계 베타 지중해 빈혈 시장은 2023년 4억 8,032만 달러에 달했습니다. 2031년에는 8억 8,904만 달러에 달할 것으로 예상되며, 2024년부터 2031년까지 연평균 8.1%의 성장률을 나타낼 전망입니다.

베타 지중해 빈혈은 헤모글로빈 생성을 감소시키는 혈액 질환입니다. 헤모글로빈은 적혈구의 철분 함유 단백질로 전신 세포에 산소를 운반하는 역할을 합니다.

베타 지중해 빈혈 환자는 헤모글로빈 농도가 낮기 때문에 체내 산소 농도가 낮아집니다. 또한 환자는 적혈구가 부족하여(빈혈) 피부가 창백해지고, 쇠약감, 피로감, 더 심각한 합병증을 유발할 수 있습니다. 베타 지중해 빈혈 환자는 비정상적인 혈전이 발생할 위험이 높습니다.

시장 성장 촉진요인 역학

베타 지중해 빈혈에 대한 연구 개발 및 FDA 승인 증가

세계 베타 지중해 빈혈 시장은 최근 다양한 연구 개발로 인해 크게 성장하고 있습니다. 베타 지중해 빈혈은 일반적으로 병력, 임상 소견 및 영상 소견을 바탕으로 진단됩니다. 또한, 베타 지중해 빈혈 치료에 대한 첨단 연구 개발은 시장 성장을 가속화하는 데 도움이 되고 있습니다.

예를 들어, 2023년 6월 CRISPR Therapeutics는 2023년 유럽혈액학회(EHA) 연례회의에서 수혈 의존성 베타 지중해 빈혈 및 중증 겸상 적혈구증 환자를 대상으로 한 Exa-cell의 주요 임상시험에서 좋은 결과를 발표했습니다, 수혈 의존성 베타 지중해 빈혈(TDT) 환자를 대상으로 평가 중인 자가 유래 체외 CRISPR/Cas9 유전자교정 세포치료제입니다.

또한, 규제 당국의 치료 승인 증가가 시장 성장을 가속하고 있으며, FDA의 승인을 받는 과정에서 일반적으로 질병 모델 동물을 이용한 전임상 실험이 수행됩니다. 동물 모델 실험의 결과가 양호한 경우, FDA는 중재 연구의 구성안을 승인할 수 있습니다.

예를 들어, 2022년 8월 블루버드 바이오(Bluebird Bio, Inc.)는 미국 식품의약국(FDA)이 정기적인 적혈구(RBC) 수혈이 필요한 성인 및 소아 환자의 베타 지중해 빈혈의 근본적인 유전적 원인을 치료하기 위해 맞춤형으로 설계된 1회용 유전자 치료제인 ZYNTEGLO(betibeglogene autotemcel), 일명 beti-cel을 승인했다고 발표했습니다. 이와 같이 위의 요인들이 시장 성장을 가속하고 있습니다.

억제요인

고가의 치료비, 치료에 따른 부작용, 엄격한 규제 지침, 제한된 치료 옵션, 개인 인식 부족 등의 요인이 시장을 저해할 것으로 예상됩니다.

이 보고서에 대한 자세한 내용 - 샘플 요청

목차

제1장 조사 방법과 조사 범위

제2장 정의와 개요

제3장 주요 요약

제4장 시장 역학

• 영향요인
  • 성장 촉진요인
  • 성장 억제요인
  • 영향 분석

제5장 산업 분석

• Porter's Five Forces 분석
• 공급망 분석
• 미충족 요구
• 규제 분석
• 러시아·우크라이나 전쟁의 영향 분석
• DMI의 견해

제6장 유형별

• 베타 지중해빈혈 메이저
• 베타 지중해빈혈 인터 미디어
• 베타 지중해빈혈 마이너

제7장 치료법별

• 철 킬레이트 요법
• 엽산 보충제
• 수혈
• 적혈구 성숙제
• 줄기세포 이식
• 기타

제8장 투여 경로별

• 경구
• 정맥내 투여
• 기타

제9장 최종사용자별

• 병원
• 클리닉
• 기타

제10장 지역별

• 북미
  • 미국
  • 캐나다
  • 멕시코
• 유럽
  • 독일
  • 영국
  • 프랑스
  • 이탈리아
  • 스페인
  • 기타 유럽
• 남미
  • 브라질
  • 아르헨티나
  • 기타 남미
• 아시아태평양
  • 중국
  • 인도
  • 일본
  • 한국
  • 기타 아시아태평양
• 중동 및 아프리카

제11장 경쟁 구도

• 경쟁 시나리오
• 제품 벤치마킹
• 기업 점유율 분석
• 주요 발전과 전략

제12장 기업 개요

• Bristol Myers Squibb
  • 기업 개요
  • 제품 포트폴리오와 설명
  • 재무 개요
  • 주요 발전
• Par Pharmaceuticals
• Qilu Pharmaceuticals
• Taro Pharmaceuticals
• Blue Bird Bio
• Cipla Ltd.
• Novartis A.G
• Teva Pharmaceuticals
• Piramal Pharma Solutions
• LGM Pharma

제13장 부록

영문목차

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