Summary

The 7 major beta thalassemia markets reached a value of US$ 340.3 Million in 2023. Looking forward, IMARC Group expects the 7MM to reach US$ 822.5 Million by 2034, exhibiting a growth rate (CAGR) of 8.35% during 2024-2034.

The beta thalassemia market has been comprehensively analyzed in IMARC’s new report titled “Beta Thalassemia Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034”. Beta thalassemia is an inherited blood disorder characterized by reduced levels of functional hemoglobin. It is primarily caused by mutations in the hemoglobin beta (HBB) gene. Beta thalassemia has three main forms – minor, intermediate, and major, and the symptoms depend on the severity of the disease. Beta thalassemia minor (beta thalassemia trait) is associated with mild anemia symptoms, such as fatigue, dizziness or weakness, frequent headaches, and pale skin. The most severe symptoms are associated with beta thalassemia major, which includes shortness of breath with exertion, heart palpitations, yellow skin, etc. The diagnosis of beta thalassemia relies on measuring red blood cell indices that reveal microcytic hypochromic anemia. A sample of the bone marrow may be taken to examine the number and shape of red blood cells. The diagnosis also includes hemoglobin analysis that reveals decreased amounts of HbA, increased amounts of hemoglobin F (HbF), as well as the clinical severity of anemia. Additionally, the hemoglobin electrophoresis test can help in identifying the specific type of thalassemia.

The increasing incidences of rare genetic blood disorders coupled with the escalating demand for novel treatment methods for reduced morbidity and improved quality of life are primarily driving the beta thalassemia market. In addition to this, the emerging popularity of bone marrow transplantation among beta thalassemia patients with available donors is further creating a positive outlook for the market. Moreover, the widespread adoption of oral iron chelators, such as deferiprone, which preferentially chelates the iron present in the heart to reduce the risk of beta thalassemia, is also bolstering the market growth. Apart from this, several key players are making extensive investments for an improved understanding of erythropoiesis, which will further lead to the development of new erythroid-stimulating factors effective in thalassemia. This, in turn, is acting as another significant growth-inducing factor. Additionally, numerous government bodies across the globe are introducing programs aimed at controlling the birth rate of thalassemia newborns through screening and diagnosis of populations with a high risk of beta thalassemia, thereby propelling the global market. Furthermore, the introduction of novel medications, such as hydroxyurea and L-glutamine, which help to reduce the symptoms of beta thalassemia by increasing the production of fetal hemoglobin, is expected to drive the global beta thalassemia market in the coming years.

IMARC Group’s new report provides an exhaustive analysis of the beta thalassemia market in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan. This includes treatment practices, in-market and pipeline drugs, the share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. According to the report the United States has the largest patient pool for beta thalassemia and also represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario and unmet medical needs, etc. have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the beta thalassemia market in any manner.

Time Period of the Study

Base Year: 2023
Historical Period: 2018-2023
Market Forecast: 2024-2034

Countries Covered

United States
Germany
France
United Kingdom
Italy
Spain
Japan

Analysis Covered Across Each Country

Historical, current, and future epidemiology scenario
Historical, current, and future performance of the beta thalassemia market
Historical, current, and future performance of various therapeutic categories in the market
Sales of various drugs across the beta thalassemia
Reimbursement scenario in the market
In-market and pipeline drugs
Competitive Landscape:
This report also provides a detailed analysis of the current Beta Thalassemia marketed drugs and late-stage pipeline drugs.

In-Market Drugs

Drug overview
Mechanism of action
Regulatory status
Clinical trial results
Drug uptake and market performance

Late-Stage Pipeline Drugs

Drug overview
Mechanism of action
Regulatory status
Clinical trial results
Drug uptake and market performance

*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.

Key Questions Answered in this Report:
Market Insights

How has the beta thalassemia market performed so far, and how will it perform in the coming years?
What are the markets shares of various therapeutic segments in 2023, and how are they expected to perform till 2034?
What was the country-wise size of the beta thalassemia market across the seven major markets in 2023, and what will it look like in 2034?
What is the growth rate of the beta thalassemia market across the seven major markets, and what will be the expected growth over the next ten years?
What are the key unmet needs in the market?

Epidemiology Insights

What is the number of prevalent cases (2018-2034) of beta thalassemia across the seven major markets?
What is the number of prevalent cases (2018-2034) of beta thalassemia by age across the seven major markets?
What is the number of prevalent cases (2018-2034) of beta thalassemia by gender across the seven major markets?
What is the number of prevalent cases (2018-2034) of beta thalassemia by type across the seven major markets?
How many patients are diagnosed (2018-2034) with beta thalassemia across the seven major markets?
What is the size of the beta thalassemia patient pool (2018-2023) across the seven major markets?
What would be the forecasted patient pool (2024-2034) across the seven major markets?
What are the key factors driving the epidemiological trend of beta thalassemia?
What will be the growth rate of patients across the seven major markets?

Beta thalassemia: Current Treatment Scenario, Marketed Drugs and Emerging Therapies

What are the current marketed drugs and what are their market performance?
What are the key pipeline drugs and how are they expected to perform in the coming years?
How safe are the current marketed drugs and what are their efficacies?
How safe are the late-stage pipeline drugs and what are their efficacies?
What are the current treatment guidelines for beta thalassemia drugs across the seven major markets?
Who are the key companies in the market and what are their market shares?
What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the beta thalassemia market?
What are the key regulatory events related to the beta thalassemia market?
What is the structure of clinical trial landscape by status related to the beta thalassemia market?
What is the structure of clinical trial landscape by phase related to the beta thalassemia market?
What is the structure of clinical trial landscape by route of administration related to the beta thalassemia market?

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Table of Contents

1 Preface

2 Scope and Methodology
2.1 Objectives of the Study
2.2 Stakeholders
2.3 Data Sources
2.3.1 Primary Sources
2.3.2 Secondary Sources
2.4 Market Estimation
2.4.1 Bottom-Up Approach
2.4.2 Top-Down Approach
2.5 Forecasting Methodology

3 Executive Summary

4 Beta thalassemia - Introduction
4.1 Overview
4.2 Regulatory Process
4.3 Epidemiology (2018-2023) and Forecast (2024-2034)
4.4 Market Overview (2018-2023) and Forecast (2024-2034)
4.5 Competitive Intelligence

5 Beta thalassemia - Disease Overview
5.1 Introduction
5.2 Symptoms and Diagnosis
5.3 Pathophysiology
5.4 Causes and Risk Factors
5.5 Treatment

6 Patient Journey

7 Beta Thalassemia - Epidemiology and Patient Population
7.1 Epidemiology - Key Insights
7.2 Epidemiology Scenario - Top 7 Markets
7.2.1 Epidemiology Scenario (2018-2023)
7.2.2 Epidemiology Forecast (2024-2034)
7.2.3 Epidemiology by Age (​2018-2034​)
7.2.4 Epidemiology by Gender (​2018-2034​)
7.2.5 Epidemiology by Type (​2018-2034​)
7.2.6 Diagnosed Cases (​2018-2034​)
7.2.7 Patient Pool/Treated Cases (​2018-2034​)
7.3 Epidemiology Scenario - United States
7.3.1 Epidemiology Scenario (2018-2023)
7.3.2 Epidemiology Forecast (2024-2034)
7.3.3 Epidemiology by Age (​2018-2034​)
7.3.4 Epidemiology by Gender (​2018-2034​)
7.3.5 Epidemiology by Type (​2018-2034​)
7.3.6 Diagnosed Cases (​2018-2034​)
7.3.7 Patient Pool/Treated Cases (​2018-2034​)
7.4 Epidemiology Scenario - Germany
7.4.1 Epidemiology Scenario (2018-2023)
7.4.2 Epidemiology Forecast (2024-2034)
7.4.3 Epidemiology by Age (​2018-2034​)
7.4.4 Epidemiology by Gender (​2018-2034​)
7.4.5 Epidemiology by Type (​2018-2034​)
7.4.6 Diagnosed Cases (​2018-2034​)
7.4.7 Patient Pool/Treated Cases (​2018-2034​)
7.5 Epidemiology Scenario - France
7.5.1 Epidemiology Scenario (2018-2023)
7.5.2 Epidemiology Forecast (2024-2034)
7.5.3 Epidemiology by Age (​2018-2034​)
7.5.4 Epidemiology by Gender (​2018-2034​)
7.5.5 Epidemiology by Type (​2018-2034​)
7.5.6 Diagnosed Cases (​2018-2034​)
7.5.7 Patient Pool/Treated Cases (​2018-2034​)
7.6 Epidemiology Scenario - United Kingdom
7.6.1 Epidemiology Scenario (2018-2023)
7.6.2 Epidemiology Forecast (2024-2034)
7.6.3 Epidemiology by Age (​2018-2034​)
7.6.4 Epidemiology by Gender (​2018-2034​)
7.6.5 Epidemiology by Type (​2018-2034​)
7.6.6 Diagnosed Cases (​2018-2034​)
7.6.7 Patient Pool/Treated Cases (​2018-2034​)
7.7 Epidemiology Scenario - Italy
7.7.1 Epidemiology Scenario (2018-2023)
7.7.2 Epidemiology Forecast (2024-2034)
7.7.3 Epidemiology by Age (​2018-2034​)
7.7.4 Epidemiology by Gender (​2018-2034​)
7.7.5 Epidemiology by Type (​2018-2034​)
7.7.6 Diagnosed Cases (​2018-2034​)
7.7.7 Patient Pool/Treated Cases (​2018-2034​)
7.8 Epidemiology Scenario - Spain
7.8.1 Epidemiology Scenario (2018-2023)
7.8.2 Epidemiology Forecast (2024-2034)
7.8.3 Epidemiology by Age (​2018-2034​)
7.8.4 Epidemiology by Gender (​2018-2034​)
7.8.5 Epidemiology by Type (​2018-2034​)
7.8.6 Diagnosed Cases (​2018-2034​)
7.8.7 Patient Pool/Treated Cases (​2018-2034​)
7.9 Epidemiology Scenario - Japan
7.9.1 Epidemiology Scenario (2018-2023)
7.9.2 Epidemiology Forecast (2024-2034)
7.9.3 Epidemiology by Age (​2018-2034​)
7.9.4 Epidemiology by Gender (​2018-2034​)
7.9.5 Epidemiology by Type (​2018-2034​)
7.9.6 Diagnosed Cases (​2018-2034​)
7.9.7 Patient Pool/Treated Cases (​2018-2034​)

8 Beta Thalassemia - Treatment Algorithm, Guidelines, and Medical Practices
8.1 Guidelines, Management and Treatment
8.2 Treatment Algorithm

9 Beta Thalassemia- Unmet Needs

10 Beta Thalassemia- Key Endpoints of Treatment

11 Beta Thalassemia - Marketed Products
11.1 List of Beta Thalassemia Marketed Drugs Across the Top 7 Markets
11.1.1 Reblozyl (luspatercept-aamt) - Celgene
11.1.1.1 Drug Overview
11.1.1.2 Mechanism of Action
11.1.1.3 Regulatory Status
11.1.1.4 Clinical Trial Results
11.1.1.5 Sales Across Major Markets
11.1.2 Zynteglo - Bluebird bio
11.1.2.1 Drug Overview
11.1.2.2 Mechanism of Action
11.1.2.3 Regulatory Status
11.1.2.4 Clinical Trial Results
11.1.2.5 Sales Across Major Markets
11.1.3 Ferriprox - Chiesi Group
11.1.3.1 Drug Overview
11.1.3.2 Mechanism of Action
11.1.3.3 Regulatory Status
11.1.3.4 Clinical Trial Results
11.1.3.5 Sales Across Major Markets

Kindly note that the above only represents a partial list of marketed drugs, and the complete list has been provided in the report.

12 Beta Thalassemia - Pipeline Drugs
12.1 List of Beta Thalassemia Pipeline Drugs Across the Top 7 Markets
12.1.1 VIT-2763 - Vifor Pharma
12.1.1.1 Drug Overview
12.1.1.2 Mechanism of Action
12.1.1.3 Clinical Trial Results
12.1.1.4 Safety and Efficacy
12.1.1.5 Regulatory Status
12.1.2 CTX001 - CRISPR Therapeutics
12.1.2.1 Drug Overview
12.1.2.2 Mechanism of Action
12.1.2.3 Clinical Trial Results
12.1.2.4 Safety and Efficacy
12.1.2.5 Regulatory Status

Kindly note that the above only represents a partial list of pipeline drugs, and the complete list has been provided in the report.

13. Beta Thalassemia - Attribute Analysis of Key Marketed and Pipeline Drugs

14. Beta Thalassemia – Clinical Trial Landscape
14.1 Drugs by Status
14.2 Drugs by Phase
14.3 Drugs by Route of Administration
14.4 Key Regulatory Events

15 Beta Thalassemia - Market Scenario
15.1 Market Scenario - Key Insights
15.2 Market Scenario - Top 7 Markets
15.2.1 Beta Thalassemia - Market Size
15.2.1.1 Market Size (2018-2023)
15.2.1.2 Market Forecast (2024-2034)
15.2.2 Beta Thalassemia - Market Size by Therapies
15.2.2.1 Market Size by Therapies (2018-2023)
15.2.2.2 Market Forecast by Therapies (2024-2034)
15.3 Market Scenario - United States
15.3.1 Beta Thalassemia - Market Size
15.3.1.1 Market Size (2018-2023)
15.3.1.2 Market Forecast (2024-2034)
15.3.2 Beta Thalassemia - Market Size by Therapies
15.3.2.1 Market Size by Therapies (2018-2023)
15.3.2.2 Market Forecast by Therapies (2024-2034)
15.3.3 Beta Thalassemia - Access and Reimbursement Overview
15.4 Market Scenario - Germany
15.4.1 Beta Thalassemia - Market Size
15.4.1.1 Market Size (2018-2023)
15.4.1.2 Market Forecast (2024-2034)
15.4.2 Beta Thalassemia - Market Size by Therapies
15.4.2.1 Market Size by Therapies (2018-2023)
15.4.2.2 Market Forecast by Therapies (2024-2034)
15.4.3 Beta Thalassemia - Access and Reimbursement Overview
15.5 Market Scenario - France
15.5.1 Beta Thalassemia - Market Size
15.5.1.1 Market Size (2018-2023)
15.5.1.2 Market Forecast (2024-2034)
15.5.2 Beta Thalassemia - Market Size by Therapies
15.5.2.1 Market Size by Therapies (2018-2023)
15.5.2.2 Market Forecast by Therapies (2024-2034)
15.5.3 Beta Thalassemia- Access and Reimbursement Overview
15.6 Market Scenario - United Kingdom
15.6.1 Beta Thalassemia- Market Size
15.6.1.1 Market Size (2018-2023)
15.6.1.2 Market Forecast (2024-2034)
15.6.2 Beta Thalassemia- Market Size by Therapies
15.6.2.1 Market Size by Therapies (2018-2023)
15.6.2.2 Market Forecast by Therapies (2024-2034)
15.6.3 Beta Thalassemia- Access and Reimbursement Overview
15.7 Market Scenario - Italy
15.7.1 Beta Thalassemia- Market Size
15.7.1.1 Market Size (2018-2023)
15.7.1.2 Market Forecast (2024-2034)
15.7.2 Beta Thalassemia- Market Size by Therapies
15.7.2.1 Market Size by Therapies (2018-2023)
15.7.2.2 Market Forecast by Therapies (2024-2034)
15.7.3 Beta Thalassemia- Access and Reimbursement Overview
15.8 Market Scenario - Spain
15.8.1 Beta Thalassemia- Market Size
15.8.1.1 Market Size (2018-2023)
15.8.1.2 Market Forecast (2024-2034)
15.8.2 Beta Thalassemia- Market Size by Therapies
15.8.2.1 Market Size by Therapies (2018-2023)
15.8.2.2 Market Forecast by Therapies (2024-2034)
15.8.3 Beta Thalassemia- Access and Reimbursement Overview
15.9 Market Scenario - Japan
15.9.1 Beta Thalassemia - Market Size
15.9.1.1 Market Size (2018-2023)
15.9.1.2 Market Forecast (2024-2034)
15.9.2 Beta Thalassemia- Market Size by Therapies
15.9.2.1 Market Size by Therapies (2018-2023)
15.9.2.2 Market Forecast by Therapies (2024-2034)
15.9.3 Beta Thalassemia- Access and Reimbursement Overview

16 Beta Thalassemia- Recent Events and Inputs From Key Opinion Leaders

17 Beta Thalassemia Market - SWOT Analysis
17.1 Strengths
17.2 Weaknesses
17.3 Opportunities
17.4 Threats

18 Beta Thalassemia Market – Strategic Recommendations

19 Appendix