DelveInsight’s analysis estimate that the beta-thalassemia market is expected to show positive growth, mainly attributed to increased prevalence and the anticipated launch of novel therapies during the forecasted period (2023–2032).
LAS VEGAS, Jan. 17, 2024 /PRNewswire/ — DelveInsight’s Beta-thalassemia Market Insights report includes a comprehensive understanding of current treatment practices, beta-thalassemia emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan].
Key Takeaways from the Beta-thalassemia Market Report
- According to DelveInsight’s analysis, the market size for beta-thalassemia across the 7MM is expected to grow with a significant CAGR by 2032.
- DelveInsight’s analysis reveals that the overall prevalent population of beta-thalassemia in the US was reported as ~3K in 2022.
- Leading beta-thalassemia companies such as Vertex Pharmaceuticals Incorporated, CRISPR Therapeutics, Editas Medicine, Inc., Agios Pharmaceuticals, Inc., Celgene, Secura Bio, Inc., Forma Therapeutics, Inc., Novo Nordisk A/S, and others are developing novel beta-thalassemia drugs that can be available in the beta-thalassemia market in the coming years.
- The promising beta-thalassemia therapies in the pipeline include CTX001, EDIT-301, Mitapivat, ACE-536, Panobinostat, Etavopivat tablets, and others.
- In September 2023, Pharmacosmos has initiated a Phase II trial of SP-420 in patients with transfusion-dependent β-thalassemia.
- In June 2023, FDA accepted the Biologics License Application (BLAs) of exagamglogene autotemcel (exa-cel) for transfusion-dependent beta thalassemia (TDT). The Prescription Drug User Fee Act (PDUFA) target action date for TDT is March 30, 2024.
Discover which therapies are expected to grab the major beta-thalassemia market share @ Beta-thalassemia Market Report
Beta-thalassemia Overview
Beta-thalassemia (β-thalassemia) is a blood disorder marked by a diminished production of the beta subunit of hemoglobin, leading to microcytic hypochromic anemia, an atypical blood smear with nucleated red blood cells, and reduced hemoglobin A (HbA) levels upon analysis. Severe untreated β-thalassemia typically manifests with profound anemia (Hb level of 3−7 g/dL), substantial enlargement of the liver and spleen, significant growth impairment, and skeletal abnormalities. Individuals with beta thalassemia major or intermediate often accumulate excess iron in the body, either due to the condition itself or frequent blood transfusions, potentially causing harm to the heart, liver, and endocrine system. Those with the minor form of Beta Thalassemia may have small red blood cells but remain asymptomatic. Beta-thalassemia diagnosis involves recognizing characteristic symptoms and conducting clinical assessments, often complemented by laboratory tests such as complete blood count (CBC) and hemoglobin electrophoresis. Molecular genetic testing can provide confirmation of a beta thalassemia diagnosis. Newborn screening is commonly employed for early detection in infants.
Beta-thalassemia Epidemiology Segmentation
The beta-thalassemia epidemiology section provides insights into the historical and current beta-thalassemia patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders.
The beta-thalassemia market report proffers epidemiological analysis for the study period 2019–2032 in the 7MM segmented into:
- Total Prevalent Cases of Beta-Thalassemia
- Diagnosed Prevalent Cases of Beta-Thalassemia
- Type-specific Diagnosed Prevalent Cases of Beta-Thalassemia
- Treatable Cases of Beta-Thalassemia
Beta-thalassemia Treatment Market
The beta-thalassemia treatment a person receives is determined by the severity of the disease. β-Thalassemia offers limited treatment options, primarily relying on red blood cell transfusions and iron chelation therapy for management. Long-term transfusion is employed to sustain hemoglobin levels within a target pre-transfusion range of 9–10.5 g/dL. To ensure the quality of blood transfusions, it is essential to leucoreduce packed red blood cells. Individuals with thalassemia major undergo regular blood transfusions, while those with thalassemia minor typically do not necessitate such transfusions. However, individuals undergoing frequent blood transfusions may develop iron overload, necessitating additional iron chelation therapy.
In November 2019, the FDA granted approval for REBLOZYL to be used in treating anemia among adult individuals with beta thalassemia who undergo frequent red blood cell (RBC) transfusions. This medication is a synthetic fusion protein designed to interact with various natural TGF-β superfamily ligands, thereby reducing Smad2/3 signaling. In mice, luspatercept-aamt facilitated the maturation of erythroid cells by promoting the differentiation of advanced-stage erythroid precursors, known as normoblasts.
In August 2022, the FDA granted approval to ZYNTEGLO (betibeglogene autotemcel), marking a significant milestone as the initial cell-based gene therapy sanctioned for addressing beta-thalassemia in both adult and pediatric patients reliant on regular red blood cell transfusions. ZYNTEGLO stands out as a singular gene therapy administered in a single dose. The treatment involves customizing each ZYNTEGLO dose by genetically modifying the patient’s own cells, specifically bone marrow stem cells, to generate functional beta-globin. This breakthrough provides a promising one-time therapeutic approach for individuals with beta-thalassemia.
To know more about beta-thalassemia treatment guidelines, visit @ Beta-thalassemia Management
Beta-thalassemia Pipeline Therapies and Key Companies
- CTX001: Vertex Pharmaceuticals Incorporated/CRISPR Therapeutics
- EDIT-301: Editas Medicine, Inc.
- Mitapivat: Agios Pharmaceuticals, Inc.
- ACE-536: Celgene
- Panobinostat: Secura Bio, Inc.
- Etavopivat tablets: Forma Therapeutics, Inc./Novo Nordisk A/S
Learn more about the FDA-approved drugs for beta-thalassemia @ Drugs for Beta-thalassemia Treatment
Beta-thalassemia Market Dynamics
The dynamics of the beta-thalassemia are expected to change in the coming years. The strong pipeline activity includes potential Phase III and Phase II emerging therapies, driven by the increasing prevalence and awareness of beta thalassemia. Despite the launch of gene therapies, which are subject to patient outcome assessment, there is still a lack of any curative treatment options. The landscape is evolving with improved national blood policies and advancements in genetic engineering, as gene therapy has been hypothesized to serve as an effective cure for monogenic blood disorders for several decades.
Furthermore, many potential therapies are being investigated for the treatment of beta-thalassemia, and it is safe to predict that the treatment space will significantly impact the beta-thalassemia market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate are expected to drive the growth of the beta-thalassemia market in the 7MM.
However several factors may impede the growth of the beta-thalassemia market. The high burden of thalassemia major, or transfusion-dependent thalassemia (TDT), is exacerbated by factors such as regular blood transfusions, poor adherence to chelation therapy, a shortage of voluntary non-remunerated blood donors, and out-of-pocket treatment costs. This burden, as reported by both patients and caregivers, is strongly influenced by the time required for disease management, fatigue, pain symptoms, and the overall impairment to quality of life.
Moreover, beta-thalassemia treatment poses a significant economic burden and disrupts patients’ overall well-being and QOL. Furthermore, the beta-thalassemia market growth may be offset by failures and discontinuation of emerging therapies, unaffordable pricing, market access and reimbursement issues, and a shortage of healthcare specialists. In addition, the undiagnosed, unreported cases and the unawareness about the disease may also impact the beta-thalassemia market growth.
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Beta-thalassemia Market Report Metrics |
Details |
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Study Period |
2019–2032 |
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Coverage |
7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan]. |
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Key Beta-thalassemia Companies |
Vertex Pharmaceuticals Incorporated, CRISPR Therapeutics, Editas Medicine, Inc., Agios Pharmaceuticals, Inc., Celgene, Secura Bio, Inc., Forma Therapeutics, Inc., Novo Nordisk A/S, and others |
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Key Pipeline Beta-thalassemia Therapies |
CTX001, EDIT-301, Mitapivat, ACE-536, Panobinostat, Etavopivat tablets, and others |
Scope of the Beta-thalassemia Market Report
- Therapeutic Assessment: Beta-thalassemia current marketed and emerging therapies
- Beta-thalassemia Market Dynamics: Key Market Forecast Assumptions of Emerging Beta-thalassemia Drugs and Market Outlook
- Competitive Intelligence Analysis: SWOT analysis and Market entry strategies
- Unmet Needs, KOL’s views, Analyst’s views, Beta-thalassemia Market Access and Reimbursement
Discover more about beta-thalassemia drugs in development @ Beta-thalassemia Clinical Trials
Table of Contents
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1. |
Beta-thalassemia Key Insights |
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2. |
Beta-thalassemia Report Introduction |
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3. |
Beta-thalassemia Overview at a Glance |
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4. |
Beta-thalassemia Executive Summary |
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5 |
Beta-thalassemia Key Events |
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6 |
Epidemiology and Market Forecast Methodology |
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6. |
Disease Background and Overview |
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7. |
Beta-thalassemia Treatment and Management |
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8. |
Beta-thalassemia Guidelines |
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9. |
Beta-thalassemia Epidemiology and Patient Population |
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10. |
Patient Journey |
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11. |
Key Endpoints in Beta-thalassemia |
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12. |
Beta-thalassemia Marketed Drugs |
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13. |
Beta-thalassemia Emerging Drugs |
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14. |
7MM Beta-thalassemia Market Analysis |
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15. |
Market Access and Reimbursement |
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16. |
KOL Views |
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17. |
Unmet Needs |
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18. |
SWOT Analysis |
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19. |
Appendix |
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20. |
DelveInsight Capabilities |
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21. |
Disclaimer |
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22. |
About DelveInsight |
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SOURCE DelveInsight Business Research, LLP
