Thalassemia Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034
The 7 major thalassemia markets reached a value of US$ 2.0 Billion in 2023. Looking forward, IMARC Group expects the 7MM to reach US$ 3.0 Billion by 2034, exhibiting a growth rate (CAGR) of 3.84% during 2024-2034.
The thalassemia market has been comprehensively analyzed in IMARC's new report titled "Thalassemia Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Thalassemia refers to a genetic blood disorder that primarily affects the production of hemoglobin, a protein found in red blood cells responsible for carrying oxygen throughout the body. There are two main types of the ailment: alpha thalassemia and beta thalassemia. The symptoms of the disease can vary depending on the type and severity of the condition. Individuals with thalassemia may experience fatigue, weakness, shortness of breath, pale or yellowish skin, delayed growth and development, an enlarged spleen, etc. In more severe cases, the disorder can cause bone deformities, anemia, and damage to various organs, such as the heart and liver. Thalassemia is diagnosed through a combination of medical history, physical examination, and laboratory tests. A complete blood count (CBC) can reveal low levels of red blood cells and hemoglobin, which are typical indicators of the ailment. Additionally, a hemoglobin electrophoresis test can determine the specific type of thalassemia present. Genetic testing can also be done to confirm the diagnosis and identify carriers of the disease.
The increasing cases of mutations in genes that control the production of hemoglobin in the body are primarily driving the thalassemia market. In addition to this, the rising usage of certain medications, such as erythropoietin and androgens, to stimulate the formation of red blood cells in the bone marrow and alleviate symptoms of anemia in thalassemia patients, is creating a positive outlook for the market. Moreover, the escalating utilization of stem cell transplantation for restoring normal hemoglobin production in the body, thereby eliminating the need for blood transfusions, is also augmenting the market growth. Besides this, the widespread adoption of hemoglobin electrophoresis for diagnosis, since it can determine the quantity of each hemoglobin variant present, which can help to further classify the type and severity of thalassemia, is acting as another significant growth-inducing factor. Additionally, the emerging popularity of various oral iron chelators, including deferoxamine, deferitrin, deferiprone, etc., that preferentially chelate the iron in the heart to lower the risk of thalassemia, is also bolstering the market growth. Furthermore, the inflating utilization of several novel medications, such as hydroxyurea and L-glutamine, which aid in lowering the symptoms of the ailment by increasing the production of fetal hemoglobin, is expected to drive the thalassemia market in the coming years.
IMARC Group's new report provides an exhaustive analysis of the 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, 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 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 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 thalassemia market
Historical, current, and future performance of various therapeutic categories in the market
Sales of various drugs across the thalassemia market
Reimbursement scenario in the market
In-market and pipeline drugs
Competitive Landscape:
This report also provides a detailed analysis of the current 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 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 thalassemia market across the seven major markets in 2023 and what will it look like in 2034?
What is the growth rate of the 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 thalassemia across the seven major markets?
What is the number of prevalent cases (?2018-2034?) of thalassemia by age across the seven major markets?
What is the number of prevalent cases (?2018-2034?) of thalassemia by gender across the seven major markets?
What is the number of prevalent cases (?2018-2034?) of thalassemia by type across the seven major markets?
How many patients are diagnosed (?2018-2034?) with thalassemia across the seven major markets?
What is the size of the 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 thalassemia?
What will be the growth rate of patients across the seven major markets?
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 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 thalassemia market?
What are the key regulatory events related to the thalassemia market?
What is the structure of clinical trial landscape by status related to the thalassemia market?
What is the structure of clinical trial landscape by phase related to the thalassemia market?
What is the structure of clinical trial landscape by route of administration related to the thalassemia market?
The thalassemia market has been comprehensively analyzed in IMARC's new report titled "Thalassemia Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Thalassemia refers to a genetic blood disorder that primarily affects the production of hemoglobin, a protein found in red blood cells responsible for carrying oxygen throughout the body. There are two main types of the ailment: alpha thalassemia and beta thalassemia. The symptoms of the disease can vary depending on the type and severity of the condition. Individuals with thalassemia may experience fatigue, weakness, shortness of breath, pale or yellowish skin, delayed growth and development, an enlarged spleen, etc. In more severe cases, the disorder can cause bone deformities, anemia, and damage to various organs, such as the heart and liver. Thalassemia is diagnosed through a combination of medical history, physical examination, and laboratory tests. A complete blood count (CBC) can reveal low levels of red blood cells and hemoglobin, which are typical indicators of the ailment. Additionally, a hemoglobin electrophoresis test can determine the specific type of thalassemia present. Genetic testing can also be done to confirm the diagnosis and identify carriers of the disease.
The increasing cases of mutations in genes that control the production of hemoglobin in the body are primarily driving the thalassemia market. In addition to this, the rising usage of certain medications, such as erythropoietin and androgens, to stimulate the formation of red blood cells in the bone marrow and alleviate symptoms of anemia in thalassemia patients, is creating a positive outlook for the market. Moreover, the escalating utilization of stem cell transplantation for restoring normal hemoglobin production in the body, thereby eliminating the need for blood transfusions, is also augmenting the market growth. Besides this, the widespread adoption of hemoglobin electrophoresis for diagnosis, since it can determine the quantity of each hemoglobin variant present, which can help to further classify the type and severity of thalassemia, is acting as another significant growth-inducing factor. Additionally, the emerging popularity of various oral iron chelators, including deferoxamine, deferitrin, deferiprone, etc., that preferentially chelate the iron in the heart to lower the risk of thalassemia, is also bolstering the market growth. Furthermore, the inflating utilization of several novel medications, such as hydroxyurea and L-glutamine, which aid in lowering the symptoms of the ailment by increasing the production of fetal hemoglobin, is expected to drive the thalassemia market in the coming years.
IMARC Group's new report provides an exhaustive analysis of the 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, 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 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 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 thalassemia market
Historical, current, and future performance of various therapeutic categories in the market
Sales of various drugs across the thalassemia market
Reimbursement scenario in the market
In-market and pipeline drugs
Competitive Landscape:
This report also provides a detailed analysis of the current 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 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 thalassemia market across the seven major markets in 2023 and what will it look like in 2034?
What is the growth rate of the 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 thalassemia across the seven major markets?
What is the number of prevalent cases (?2018-2034?) of thalassemia by age across the seven major markets?
What is the number of prevalent cases (?2018-2034?) of thalassemia by gender across the seven major markets?
What is the number of prevalent cases (?2018-2034?) of thalassemia by type across the seven major markets?
How many patients are diagnosed (?2018-2034?) with thalassemia across the seven major markets?
What is the size of the 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 thalassemia?
What will be the growth rate of patients across the seven major markets?
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 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 thalassemia market?
What are the key regulatory events related to the thalassemia market?
What is the structure of clinical trial landscape by status related to the thalassemia market?
What is the structure of clinical trial landscape by phase related to the thalassemia market?
What is the structure of clinical trial landscape by route of administration related to the thalassemia market?
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 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 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 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 THALASSEMIA - TREATMENT ALGORITHM, GUIDELINES, AND MEDICAL PRACTICES
8.1 Guidelines, Management and Treatment
8.2 Treatment Algorithm
9 THALASSEMIA - UNMET NEEDS
10 THALASSEMIA - KEY ENDPOINTS OF TREATMENT
11 THALASSEMIA - MARKETED PRODUCTS
11.1 List of Thalassemia Marketed Drugs Across the Top 7 Markets
11.1.1 Reblozyl (Luspatercept) - Acceleron Pharma/Celgene Corporation
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 (Betibeglogene autotemcel) - 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 Exjade (Deferasirox) - Novartis
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
11.1.4 Ferriprox (Deferiprone) - Apotex
11.1.4.1 Drug Overview
11.1.4.2 Mechanism of Action
11.1.4.3 Regulatory Status
11.1.4.4 Clinical Trial Results
11.1.4.5 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 THALASSEMIA - PIPELINE DRUGS
12.1 List of Thalassemia Pipeline Drugs Across the Top 7 Markets
12.1.1 Mitapivat - Agios Pharmaceuticals
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 SLN 124 - Silence Therapeutics plc
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
12.1.3 CTX001 - Vertex Pharmaceuticals/ CRISPR Therapeutics
12.1.3.1 Drug Overview
12.1.3.2 Mechanism of Action
12.1.3.3 Clinical Trial Results
12.1.3.4 Safety and Efficacy
12.1.3.5 Regulatory Status
12.1.4 Etavopivat - Forma therapeutics
12.1.4.1 Drug Overview
12.1.4.2 Mechanism of Action
12.1.4.3 Clinical Trial Results
12.1.4.4 Safety and Efficacy
12.1.4.5 Regulatory Status
12.1.5 EDIT-301 - Editas Medicine, Inc.
12.1.5.1 Drug Overview
12.1.5.2 Mechanism of Action
12.1.5.3 Clinical Trial Results
12.1.5.4 Safety and Efficacy
12.1.5.5 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. THALASSEMIA - ATTRIBUTE ANALYSIS OF KEY MARKETED AND PIPELINE DRUGS
14. 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 THALASSEMIA - MARKET SCENARIO
15.1 Market Scenario - Key Insights
15.2 Market Scenario - Top 7 Markets
15.2.1 Thalassemia - Market Size
15.2.1.1 Market Size (2018-2023)
15.2.1.2 Market Forecast (2024-2034)
15.2.2 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 Thalassemia - Market Size
15.3.1.1 Market Size (2018-2023)
15.3.1.2 Market Forecast (2024-2034)
15.3.2 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 Thalassemia - Access and Reimbursement Overview
15.4 Market Scenario - Germany
15.4.1 Thalassemia - Market Size
15.4.1.1 Market Size (2018-2023)
15.4.1.2 Market Forecast (2024-2034)
15.4.2 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 Thalassemia - Access and Reimbursement Overview
15.5 Market Scenario - France
15.5.1 Thalassemia - Market Size
15.5.1.1 Market Size (2018-2023)
15.5.1.2 Market Forecast (2024-2034)
15.5.2 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 Thalassemia - Access and Reimbursement Overview
15.6 Market Scenario - United Kingdom
15.6.1 Thalassemia - Market Size
15.6.1.1 Market Size (2018-2023)
15.6.1.2 Market Forecast (2024-2034)
15.6.2 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 Thalassemia - Access and Reimbursement Overview
15.7 Market Scenario - Italy
15.7.1 Thalassemia - Market Size
15.7.1.1 Market Size (2018-2023)
15.7.1.2 Market Forecast (2024-2034)
15.7.2 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 Thalassemia - Access and Reimbursement Overview
15.8 Market Scenario - Spain
15.8.1 Thalassemia - Market Size
15.8.1.1 Market Size (2018-2023)
15.8.1.2 Market Forecast (2024-2034)
15.8.2 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 Thalassemia - Access and Reimbursement Overview
15.9 Market Scenario - Japan
15.9.1 Thalassemia - Market Size
15.9.1.1 Market Size (2018-2023)
15.9.1.2 Market Forecast (2024-2034)
15.9.2 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 Thalassemia - Access and Reimbursement Overview
16 THALASSEMIA - RECENT EVENTS AND INPUTS FROM KEY OPINION LEADERS
17 THALASSEMIA MARKET - SWOT ANALYSIS
17.1 Strengths
17.2 Weaknesses
17.3 Opportunities
17.4 Threats
18 THALASSEMIA MARKET – STRATEGIC RECOMMENDATIONS
19 APPENDIX
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 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 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 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 THALASSEMIA - TREATMENT ALGORITHM, GUIDELINES, AND MEDICAL PRACTICES
8.1 Guidelines, Management and Treatment
8.2 Treatment Algorithm
9 THALASSEMIA - UNMET NEEDS
10 THALASSEMIA - KEY ENDPOINTS OF TREATMENT
11 THALASSEMIA - MARKETED PRODUCTS
11.1 List of Thalassemia Marketed Drugs Across the Top 7 Markets
11.1.1 Reblozyl (Luspatercept) - Acceleron Pharma/Celgene Corporation
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 (Betibeglogene autotemcel) - 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 Exjade (Deferasirox) - Novartis
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
11.1.4 Ferriprox (Deferiprone) - Apotex
11.1.4.1 Drug Overview
11.1.4.2 Mechanism of Action
11.1.4.3 Regulatory Status
11.1.4.4 Clinical Trial Results
11.1.4.5 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 THALASSEMIA - PIPELINE DRUGS
12.1 List of Thalassemia Pipeline Drugs Across the Top 7 Markets
12.1.1 Mitapivat - Agios Pharmaceuticals
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 SLN 124 - Silence Therapeutics plc
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
12.1.3 CTX001 - Vertex Pharmaceuticals/ CRISPR Therapeutics
12.1.3.1 Drug Overview
12.1.3.2 Mechanism of Action
12.1.3.3 Clinical Trial Results
12.1.3.4 Safety and Efficacy
12.1.3.5 Regulatory Status
12.1.4 Etavopivat - Forma therapeutics
12.1.4.1 Drug Overview
12.1.4.2 Mechanism of Action
12.1.4.3 Clinical Trial Results
12.1.4.4 Safety and Efficacy
12.1.4.5 Regulatory Status
12.1.5 EDIT-301 - Editas Medicine, Inc.
12.1.5.1 Drug Overview
12.1.5.2 Mechanism of Action
12.1.5.3 Clinical Trial Results
12.1.5.4 Safety and Efficacy
12.1.5.5 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. THALASSEMIA - ATTRIBUTE ANALYSIS OF KEY MARKETED AND PIPELINE DRUGS
14. 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 THALASSEMIA - MARKET SCENARIO
15.1 Market Scenario - Key Insights
15.2 Market Scenario - Top 7 Markets
15.2.1 Thalassemia - Market Size
15.2.1.1 Market Size (2018-2023)
15.2.1.2 Market Forecast (2024-2034)
15.2.2 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 Thalassemia - Market Size
15.3.1.1 Market Size (2018-2023)
15.3.1.2 Market Forecast (2024-2034)
15.3.2 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 Thalassemia - Access and Reimbursement Overview
15.4 Market Scenario - Germany
15.4.1 Thalassemia - Market Size
15.4.1.1 Market Size (2018-2023)
15.4.1.2 Market Forecast (2024-2034)
15.4.2 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 Thalassemia - Access and Reimbursement Overview
15.5 Market Scenario - France
15.5.1 Thalassemia - Market Size
15.5.1.1 Market Size (2018-2023)
15.5.1.2 Market Forecast (2024-2034)
15.5.2 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 Thalassemia - Access and Reimbursement Overview
15.6 Market Scenario - United Kingdom
15.6.1 Thalassemia - Market Size
15.6.1.1 Market Size (2018-2023)
15.6.1.2 Market Forecast (2024-2034)
15.6.2 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 Thalassemia - Access and Reimbursement Overview
15.7 Market Scenario - Italy
15.7.1 Thalassemia - Market Size
15.7.1.1 Market Size (2018-2023)
15.7.1.2 Market Forecast (2024-2034)
15.7.2 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 Thalassemia - Access and Reimbursement Overview
15.8 Market Scenario - Spain
15.8.1 Thalassemia - Market Size
15.8.1.1 Market Size (2018-2023)
15.8.1.2 Market Forecast (2024-2034)
15.8.2 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 Thalassemia - Access and Reimbursement Overview
15.9 Market Scenario - Japan
15.9.1 Thalassemia - Market Size
15.9.1.1 Market Size (2018-2023)
15.9.1.2 Market Forecast (2024-2034)
15.9.2 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 Thalassemia - Access and Reimbursement Overview
16 THALASSEMIA - RECENT EVENTS AND INPUTS FROM KEY OPINION LEADERS
17 THALASSEMIA MARKET - SWOT ANALYSIS
17.1 Strengths
17.2 Weaknesses
17.3 Opportunities
17.4 Threats
18 THALASSEMIA MARKET – STRATEGIC RECOMMENDATIONS
19 APPENDIX
