Transfusion-dependent Thalassaemia Market Set for Robust Growth Through 2034, Driven by Advances in Gene and Cell Therapies | DelveInsight
DelveInsight’s “Transfusion-dependent Thalassemia Market Insight, Epidemiology and Market Forecast – 2034” report delivers a comprehensive evaluation of the current treatment paradigm, emerging pipeline assets, epidemiological trends, and market projections across the United States, EU4 (Germany, France, Italy, Spain), the United Kingdom, and Japan. The analysis encompasses marketed therapies, late-stage clinical candidates, and forecasted transfusion-dependent thalassaemia market size through 2034.
Transfusion-dependent thalassemia (TDT) is a severe form of the genetic blood disorder thalassemia that requires patients to undergo lifelong, regular blood transfusions to survive. Transfusion-dependent thalassemia is a subtype of beta thalassemia, also known as thalassemia major. The other type is non-transfusion-dependent thalassemia or thalassemia intermedia.
According to DelveInsight’s projections, the transfusion-dependent thalassaemia market is expected to grow at a significant compound annual growth rate (CAGR) during the 2025-2034 forecast period, driven by the maturation of gene-editing approaches, novel pharmacological agents targeting ineffective erythropoiesis, and expanding reimbursement landscapes.
Download the Transfusion-dependent Thalassemia Market report to understand which factors are driving the market @ Transfusion-dependent Thalassemia Market Trends.
DelveInsight’s epidemiological insights show that Beta Thalassemia continues to represent a significant public health concern across major global markets. In the United States, the total prevalent cases of thalassemia intermedia were estimated at approximately 1.4 million in 2023, with expectations of substantial changes by 2034.
In the UK, transfusion-dependent thalassaemia shows a higher prevalence compared to Beta Thalassemia Intermedia. In 2023, approximately 745K Beta Thalassemia major cases were recorded in the UK, and these figures are expected to increase throughout the forecast period spanning from 2025 to 2034. These figures highlight the evolving transfusion-dependent thalassaemia landscape and underscore the need for continued surveillance and targeted healthcare strategies.
According to the market report, transfusion-dependent thalassaemia management is characterized by lifelong red blood cell transfusions to manage severe anaemia, leading to iron overload and complications such as cardiac dysfunction, endocrinopathies, and hepatic fibrosis. Current treatment practices include regular transfusion regimens combined with iron chelation therapies, such as Deferoxamine, Deferiprone, and Deferasirox, offering symptomatic relief but failing to alter disease progression significantly. Hematopoietic stem cell transplant (HSCT) is currently the only curative treatment option, primarily used in younger patients with suitable HLA-matched donors, but is limited by donor availability and potential risks like graft-versus-host disease.
Discover how emerging Transfusion-dependent Thalassemia therapies will impact the market @ Transfusion-dependent Thalassemia Emerging Therapies.
Gene therapy has emerged as a promising alternative, involving the modification of a patient’s own hematopoietic stem cells to produce healthy hemoglobin, thereby reducing or eliminating the need for transfusions and chelation. CASGEVY (exagamglogene autotemcel), by Vertex Pharmaceuticals and CRISPR Therapeutics, was approved in January 2024 for the treatment of transfusion-dependent beta-thalassemia in patients 12 years and older. Other gene editing approaches, such as CRISPR/Cas9 and zinc finger nucleases targeting genes like BCL11A to increase fetal hemoglobin production, are also under investigation in clinical trials. Additionally, erythroid maturation agents like luspatercept are approved to reduce transfusion requirements in adult TDT patients.
Beyond gene therapies, the pipeline includes drugs targeting specific aspects of transfusion-dependent thalassaemia pathophysiology. Luspatercept, an erythroid maturation agent, is approved for adults with TDT to reduce transfusion needs. Other promising agents in clinical trials include mitapivat and etavopivat, which are oral pyruvate kinase activators aimed at improving red blood cell health, and various agents that regulate hepcidin and iron metabolism to address iron overload. Repurposed drugs and novel combinations are also being investigated to potentially enhance the efficacy and safety of existing or noval therapies for transfusion-dependent thalassaemia management.
Long-term follow-up data from the CLIMB THAL-111 and CLIMB-131 trials were presented at the 2025 EHA Congress. CASGEVY continued to demonstrate durable benefits in achieving transfusion independence for most patients with TDT.
Another study presented at the 2025 EHA Congress explored the impact of gene therapy on the physical growth of pediatric patients with beta-thalassemia, observing improvements in height-for-age in some patients and normal BMI-for-age in all after gene therapy, while also noting the presence of vitamin D abnormalities that should be regularly monitored.
In December 2024, a study published highlighted the safety and effectiveness of beti-cel (ZYNTEGLO) in patients with transfusion-dependent non-β0/β0 genotype thalassemia in a real-world setting in Germany, with all patients achieving and maintaining transfusion independence.
Another research published in February 2025 indicated that iron chelation therapy influences the gut microbiome in thalassemia patients, emphasizing the importance of considering microbiome-targeted strategies alongside traditional iron management to improve gut health and potentially optimize iron metabolism.
Discover recent advancements in the Transfusion-dependent Thalassemia landscape @ Transfusion-dependent Thalassemia Recent Developments.
DelveInsight’s analysis underscores that, despite significant therapeutic advances, key opportunities remain in optimizing patient stratification, managing long-term safety of gene therapies, and ensuring equitable access across diverse geographies. As research progresses and clinical data mature, the transfusion-dependent thalassaemia market is set for remarkable expansion and innovation through 2034, offering renewed hope for patients and robust growth prospects for stakeholders.
Table of Contents
1. Key Insights
2. Executive Summary of Transfusion-dependent Thalassemia
3. Competitive Intelligence Analysis for Transfusion-dependent Thalassemia
4. Transfusion-dependent Thalassemia Market Overview at a Glance
5. Transfusion-dependent Thalassemia: Disease Background and Overview
6. Transfusion-dependent Thalassemia Patient Journey
7. Transfusion-dependent Thalassemia Epidemiology and Patient Population
8. Treatment Algorithm, Current Treatment, and Medical Practices
9. Transfusion-dependent Thalassemia Unmet Needs
10. Key Endpoints of Transfusion-dependent Thalassemia Treatment
11. Transfusion-dependent Thalassemia Marketed Products
12. Transfusion-dependent Thalassemia Emerging Therapies
13. Transfusion-dependent Thalassemia: Seven Major Market Analysis
14. Attribute analysis
15. 7MM: Market Outlook
16. Access and Reimbursement Overview of Transfusion-dependent Thalassemia
17. KOL Views
18. Transfusion-dependent Thalassemia Market Drivers
19. Transfusion-dependent Thalassemia Market Barriers
20. Appendix
21. DelveInsight Capabilities
22. Disclaimer
23. About DelveInsight
About DelveInsight
DelveInsight is a leading market research and consulting firm specializing in disease-specific insights and therapeutic market analysis. Their reports integrate real-world data, clinical trial findings, and expert interviews to deliver comprehensive industry intelligence.
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