Beta Thalassemia – Landscape & Forecast – Disease Landscape & Forecast

Beta thalassemia is a rare genetic blood disorder characterized by a substantial reduction in or lack of beta-globin protein, resulting in chronic anemia, failure to thrive, jaundice, pallor, poor musculature, and skeletal deformities. Until 2019, allogenic hematopoietic stem cell transplantation (HSCT) with a matched (most often sibling) donor was the only curative therapy. However, with the launch of Bluebird Bio’s Zynteglo, some beta thalassemia patients have the possibility of a transfusion-free future. Zynteglo is a lentiglobin vector-based gene therapy that launched in Europe for transfusion-dependent patients aged 12 or older who do not have a β00 genotype and for whom HSCT is appropriate but cannot be administered owing to a lack of an HLA-matched related donor. Luspatercept is another treatment for beta thalassemia; it was launched in 2019 in the United States and in 2020 in Europe to treat the anemia in adult beta thalassemia patients who require regular red blood cell transfusions. Most patients are managed with a combination of blood transfusions and iron chelators. Patients with the most severe form of the disease require chronic blood transfusions to survive, substantially affecting their quality of life. Thus, therapies that reduce or eliminate the transfusion burden are greatly needed. Recognizing the commercial opportunity in this market, drug developers have focused on agents targeting the underlying genetic defect and reducing the transfusion burden.

Questions Answered:

  • How large is the diagnosed prevalent beta thalassemia population in the United States and EU5? How will the population change through 2030?
  • What is the current treatment for beta thalassemia patients, and how will it change in the next ten years? How will the launches of Zynteglo and luspatercept impact beta thalassemia treatment?
  • What pipeline products are most promising, and what sales / uptake could they secure in beta thalassemia? How will new therapies impact medical practice?

GEOGRAPHIES

United States, EU5

PRIMARY RESEARCH

Six country-specific interviews with thought-leading hematologists

Supported by survey data collected for this study

EPIDEMIOLOGY

Diagnosed prevalent and drug-treatable cases of beta thalassemia by country, segmented by severity, age cohort, genotype, and availability of HLA-matched sibling HSCT donor.

FORECAST

Drug-level sales and patient shares of key beta thalassemia therapies through 2030, including Zynteglo and luspatercept .

EMERGING THERAPIES

Phase II and Phase I/II : 6 drugs. Coverage of select preclinical and Phase I products.

Marketed drugs: 2

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
    • Key Findings
      • Beta Thalassemia- Key Findings Deck
    • Commercial Outlook
      • Key Findings
        • Regional Sales of Key Emerging Therapies to Treat Beta Thalassemia: 2030
        • 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
        • Ligand Trap Receptor-Specific Trends
          • Alternative Market Scenarios
            • Alternative Scenarios for the Beta Thalassemia Market Through 2030
        • Forecast
          • Zynteglo Sales to Treat Beta Thalassemia in the Major Pharmaceutical Markets: 2020-2030
          • Luspatercept Sales to Treat Beta Thalassemia in the Major Pharmaceutical Markets: 2020-2030
        • Etiology and Pathophysiology
          • Disease Overview
            • Etiology
              • The HBB Gene and u03b2-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: 2020-2030
                • 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: 2020-2030
                • 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: 2020-2030
                • 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 u03b20/u03b20 in the Major Pharmaceutical Markets by Status of Matched Related HSCT: 2020-2030
                • Number of Diagnosed Prevalent Cases of Beta Thalassemia Major Genotype u03b2+/u03b2+ in the Major Pharmaceutical Markets by Status of Matched Related HSCT: 2020-2030
                • Number of Diagnosed Prevalent Cases of Beta Thalassemia Major Genotype u03b20/u03b2+ in the Major Pharmaceutical Markets by Status of Matched Related HSC Transplantation: 2020-2030
                • 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: 2020-2030
                • 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
                  • Advantages and Disadvantages of Transfusion Therapy
                  • Expert Insight: Transfusion Therapy
                  • 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 HSCT
                  • Expert Insight: Hematopoietic Stem Cell Transplantation
                  • Luspatercept Clinical Development
                  • Clinical Trial Outcomes for Luspatercept
                  • Key Ongoing Clinical Trials of Luspatercept in the Treatment of Beta Thalassemia
                  • Advantages and Disadvantages of Luspatercept
                  • Expert Insight: Luspatercept
                • Medical Practice
                  • Overview
                  • Country and Regional Beta Thalassemia Treatment Guidelines
              • 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
                  • 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
                  • 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
                    • Appendix
                      • Beta Thalassemia Bibliography