Beta thalassemia is a rare genetic blood disorder characterized by a substantial reduction in or lack of β-globin protein, resulting in chronic anemia, failure to thrive, jaundice, pallor, poor musculature, and skeletal deformities. Allogenic hematopoietic stem cell transplantation with a matched (most often sibling) donor is the only cure; however, with the conditional approval of Bluebird Bio’s Zynteglo in Europe and its commercial launch in Germany, patients will soon have an additional potentially curative option. Most patients are currently managed with a combination of blood transfusions and iron chelators. The most severe thalassemia major (TM) patients require chronic blood transfusions to survive, substantially reducing theirquality of life. Acceleron/BMS’s luspatercept, which has launched in the United States, improves erythropoiesis and is expected to be used for bothTM and thalassemia intermedia (TI) patients. Still, a high unmet need exists for therapies to reduce or eliminate the need for transfusions. Drug developers have recognized the commercial opportunity in the beta thalassemia market and are focusing on developing agents that target the underlying genetic defect and reduce the transfusion burden.

Questions Answered:

  • How large is the diagnosed prevalent beta thalassemia population in the United States and EU5 (France, Germany, Italy, Spain, and the United Kingdom)? How will the population change through 2029?
  • What is the current treatment landscape for beta thalassemia patients, and how will it change in the next ten years? How will the launches of Zynteglo in the EU (Germany) and luspatercept in the United States affect the treatment of beta thalassemia?
  • What pipeline products are most promising, and what sales/uptake could they secure in beta thalassemia? How will new therapies affect medical practice?

Product Description

Niche & Rare Disease Landscape & Forecast: Comprehensive market intelligence providing world-class epidemiology, keen insight into current treatment paradigms, in-depth pipeline assessments, and drug forecasts supported by detailed primary and secondary research.

Table of contents

  • Beta-Thalassemia - Landscape & Forecast - Disease Landscape & Forecast
    • COVID-19
      • Key Findings
        • Commercial Outlook
          • Key Findings
            • Regional Sales of Key Emerging Therapies to Treat Beta Thalassemia: 2029
            • Population Positioning of Therapies in Beta Thalassemia
            • Beta Thalassemia SWOT Analysis
          • Drivers and Constraints
            • What Factors Are Driving the Market for Beta Thalassemia?
            • What Factors Are Constraining the Market for Beta Thalassemia?
          • Gene Therapy-Specific Trends
            • Major-Market Sales of Gene Therapies in Beta Thalassemia: 2019-2029
            • Major-Market: Zynteglo Sales by Genotype vs. Cumulative TM Patients
            • Major-Market: Yearly and Cumulative TM Patients Treated with Zynteglo
          • Ligand Trap Receptor-Specific Trends
            • Major-Market Sales of Ligand Trap Receptors in Beta Thalassemia: 2019-2029
          • Alternative Market Scenarios
            • Alternative Scenarios for the Beta Thalassemia Market Through 2029
            • U.S. Sales of Zynteglo by Scenario: 2019-2029
        • Forecast
          • Zynteglo Sales to Treat Beta Thalassemia in the Major Pharmaceutical Markets: 2019-2029
          • Luspatercept Sales to Treat Beta Thalassemia in the Major Pharmaceutical Markets: 2019-2029
        • Etiology and Pathophysiology
          • Disease Overview
            • Etiology
              • The HBB Gene and β-Globin
              • Notable Transcription Factors Involved in Globin Gene Activation and Switching
              • Genotype/Phenotype Relationship
              • Genotype / Phenotype Relationship in Beta Thalassemia
            • Pathophysiology
              • Ineffective Erythropoiesis
              • Iron Overload
              • Symptoms of Beta Thalassemia
              • Symptomology Prominent in Thalassemia Intermedia Resulting from Extramedullary Hematopoiesis
            • Key Pathways and Drug Targets
              • Hemoglobin Subunit Development
              • Key Pathways and Targets in Beta Thalassemia
              • Key Pathways and Targets in Beta Thalassemia
          • Epidemiology
            • Key Findings
              • Epidemiology Populations
                • Disease Definition
                • Methods
                • Sources Used for Diagnosed Prevalence of Beta Thalassemia
                • Number of Diagnosed Prevalent Cases of Beta Thalassemia in the Major Pharmaceutical Markets: 2019-2029
                • Disease Definition
                • Methods
                • Sources Used for Severity of Beta Thalassemia
                • Number of Diagnosed Prevalent Cases of Beta Thalassemia in the Major Pharmaceutical Markets by Severity: 2019-2029
                • Disease Definition
                • Methods
                • Sources Used for Prevalent Cases of Beta Thalassemia Major by Genotype
                • Number of Diagnosed Prevalent Cases of Beta Thalassemia Major in the Major Pharmaceutical Markets by Genotype: 2019-2029
                • Disease Definition
                • Methods
                • Sources Used for Matched Related HSCTs Among Prevalent Cases of Beta Thalassemia Major
                • Number of Diagnosed Prevalent Cases of Beta Thalassemia Major Genotype β0/β0 in the Major Pharmaceutical Markets by Status of Matched Related HSCT: 2019-2029
                • Number of Diagnosed Prevalent Cases of Beta Thalassemia Major Genotype β+/β+ in the Major Pharmaceutical Markets by Status of Matched Related HSCT: 2019-2029
                • Number of Diagnosed Prevalent Cases of Beta Thalassemia Major Genotype β0/β+ in the Major Pharmaceutical Markets by Status of Matched Related HSC Transplantation: 2019-2029
                • Disease Definition
                • Methods
                • Sources Used for Prevalent Cases of Beta Thalassemia Intermedia by Transfusion Requirement
                • Number of Diagnosed Prevalent Cases of Beta Thalassemia Intermedia in the Major Pharmaceutical Markets by Transfusion Requirement: 2019-2029
                • Methods
                • Sources Used for Drug-Treatment Rates of Beta Thalassemia
            • Current Treatment
              • Key Findings
                • Diagnosis
                  • Treatment Providers and Referral Patterns
                  • Expert Insight: Diagnosis of Beta Thalassemia
                • Treatment Goals
                  • Key Endpoints Used in Clinical Trials for Beta Thalassemia
                • Key Current Therapies
                  • Overview
                  • Mechanism of Action of Key Treatments Used for Beta Thalassemia
                  • Current Treatments Used for Beta Thalassemia
                  • Expert Insight: Iron Chelators
                  • Clinical Trial Outcomes for Deferoxamine
                  • Advantages and Disadvantages of Deferoxamine
                  • Expert Insight: Deferoxamine
                  • Clinical Trial Outcomes for Deferasirox
                  • Advantages and Disadvantages of Deferasirox
                  • Key Results from Select Clinical Trials Investigating Deferasirox for the Treatment of Beta Thalassemia
                  • Expert Insight: Deferasirox
                  • Clinical Trial Outcomes for Deferiprone
                  • Advantages and Disadvantages of Deferiprone
                  • Key Results from Select Clinical Trials Investigating Deferiprone for the Treatment of Beta Thalassemia
                  • Expert Insight: Deferiprone
                  • Advantages and Disadvantages of Transfusion Therapy
                  • Expert Insight: Transfusion Therapy
                  • Expert Insight: Hematopoietic Stem Cell Transplantation
                • Medical Practice
                  • Overview
                  • Country and Regional Beta Thalassemia Treatment Guidelines
                  • Treatment Decision Tree for Beta Thalassemia
              • Unmet Need Overview
                • Current and Future Attainment of Unmet Needs in Beta Thalassemia
                • Top Unmet Needs in Beta Thalassemia: Current and Future Attainment
                • Expert Insight: Unmet Needs in Beta Thalassemia
              • Emerging Therapies
                • Key Findings
                  • Expert Insight
                  • Pipeline Trends in Beta Thalassemia
                • Key Emerging Therapies
                  • Key Therapies in Late-Phase Development for Beta Thalassemia
                  • Estimated Launch Dates of Key Emerging Therapies for the Treatment of Beta Thalassemia
                  • Zynteglo Administration Process
                  • Zynteglo Profile
                  • Zynteglo Clinical Development
                  • Key Clinical Trials of Zynteglo in the Treatment of Beta Thalassemia
                  • Expert Insight: Zynteglo
                  • Expectations for Launch and Sales Opportunity of Zynteglo in Beta Thalassemia
                  • Select Clinical Trial Results of Luspatercept for the Treatment of Beta Thalassemia
                  • Luspatercept Clinical Development
                  • Key Ongoing Clinical Trials of Luspatercept in the Treatment of Beta Thalassemia
                  • Expert Insight: Luspatercept
                  • Expectations for Launch and Sales Opportunity of Luspatercept in Beta Thalassemia
                • Early-Phase Pipeline Analysis
                  • Notable Developments in the Early-Phase Pipeline for Beta Thalassemia
                  • Select Compounds in Early-Phase Development for Beta Thalassemia
                  • Expert Insight: Early-Phase Pipeline
                • Patient Registries
                  • Patient Registries for Beta Thalassemia
                  • Prominent Beta Thalassemia Patient Organizations
                • Orphan-Drug Designation
                • Access and Reimbursement Overview
                  • Region-Specific Reimbursement Practices
                  • Methodology
                    • Bottom-Up Forecasting Overview
                      • Patient Populations
                      • Drug-Specific Assumptions
                    • Bottom-Up Forecast Assumptions
                      • General Sources of Data
                      • Patients Eligible for Zynteglo and Luspatercept
                      • Zynteglo Patient Flow at Launch Year
                      • Zynteglo Patient Flow at Out Year 2029
                      • Drug Treatment Rate in Beta Thalassemia
                      • Drug-Treatable Rate in Beta Thalassemia
                      • Zynteglo Patient-Share Assumptions in Beta Thalassemia
                      • Luspatercept Patient-Share Assumptions in Beta Thalassemia
                      • General Statements About Pricing
                      • Sources for Drug Prices
                      • 2019 Exchange Rates
                      • Price per Treated Day: 2029
                      • Dosing, Days of Therapy, and Compliance
                      • Out-Year Forecasting
                      • Emerging Therapy Prices
                      • Estimated Treatment Failure Rates on Zynteglo
                    • Primary Market Research
                      • Experts Interviewed
                  • Appendix
                    • Key Abbreviations Related to Beta Thalassemia
                    • Brands, Marketers, and Generic Availability of Key Therapies for Beta Thalassemia by Market
                    • Beta Thalassemia Bibliography

                Author(s): Natasha Bardhan, M.Pharm; Narendra Parihar

                Natasha Bardhan is a Research Associate for the Infectious, Niche, & Rare Diseases (INRD) team at Decision Resources.

                Natasha Bardhan holds a bachelor’s degree in Pharmacy from DIPSAR, New Delhi and has completed her master’s in Pharmaceutics from Amity University, Noida. Prior to joining DRG, she worked as a Senior Associate at WNS (Healthcare) for two and a half years. She has worked on multiple client projects involving patents, sales and forecasting, pipeline analysis, keywords research and product/company profiling.

                Narendra is an associate epidemiologist within the epidemiology team at Decision Resources Group. Narendra specializes in developing epidemiological forecasts for multiple indications within the DRG syndicated portfolio.

                His qualifications include an MPH with specialization in Health Policy, Economics and Finance from the Tata Institute of Social Sciences, Mumbai, and a Bachelor’s degree in Dentistry from the Rajasthan University of Health Sciences.