DelveInsight’s “Beta Thalassemia Market Insights, Epidemiology, and Market Forecast 2032” report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the Beta Thalassemia market size, share, trends, and growth opportunities in the seven major markets (7MM) (i.e., the United States, EU4 (Germany, Spain, Italy, France), the United Kingdom and Japan).
The report covers emerging Beta Thalassemia drugs, current treatment practices, market share of individual therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current Beta Thalassemia treatment practice/algorithm, key drivers & barriers impacting the market growth, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.
Beta Thalassemia: An Overview
Beta thalassemia (β-thalassemia) is a blood disorder characterized by reduced synthesis of the hemoglobin subunit beta (hemoglobin beta chain) that results in microcytic hypochromic anemia, an abnormal peripheral blood smear with nucleated red blood cells, and reduced amounts of hemoglobin A (HbA) on hemoglobin analysis.
Beta thalassemia includes three primary forms: thalassemia major (variably referred to as “Cooley’s Anemia” and “Mediterranean Anemia), thalassemia intermediate, and thalassemia minor (also called “beta thalassemia carrier,” “beta thalassemia trait” or “heterozygous beta thalassemia”).
Most beta thalassemia cases are caused by a mutation in the HBB gene. In extremely rare cases, a loss of genetic material (deletion) that includes the HBB gene causes the disorder. Individuals with beta thalassemia minor have a mutation in one HBB gene and are carriers for the disorder. Individuals with beta thalassemia intermedia or major have mutations in both HBB genes.
General symptoms of severe untreated β-thalassemia include severe anemia, with a Hb level of 3−7 g/dL, massive hepatosplenomegaly, severe growth retardation, and bony deformities. People with beta thalassemia major or intermediate usually have a buildup of iron in the body, either from the disease itself or from repeated blood transfusions. Excess iron can damage the heart, liver, and endocrine system. People with the minor form of Beta Thalassaemia have small red blood cells but no symptoms.
Beta Thalassemia Market Key Facts
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Many studies point towards a similar fact that about 1.5% of the global population (80–90 million people) has been estimated to be the carriers of beta-thalassemia.
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According to Chamberlain et al. (2021), in Europe, the estimated beta-thalassemia prevalence was 0.16/100,000 in Spain and in the United States, it was 5.7/100,000.
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As per Kattamis et al. (2020), the prevalence of β‐thalassemia major was observed to be 0.3 per 100,000 in different parts of the United Kingdom.
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As per the studies conducted by Khaled et al. (2023), the prevalence of beta-thalassemia in France increased from 0.6 in the period 2005–2008 to 0.8 in 2019 per 100,000. Whereas, the prevalence of beta-thalassemia in Spain was observed to be 0.2/100,000 people in the 2014–2017 period duration.
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According to the National Haemoglobinopathy Registry (NHR) annual report 2020, there were >900 beta-thalassemia major patients and >200 beta-thalassemia intermediate patients living in the UK.
The market outlook section of the report helps to build a detailed comprehension of the historical, current, and forecasted market size by analyzing the impact of current and emerging Beta Thalassemia pipeline therapies. It also thoroughly assesses the Beta Thalassemia market drivers & barriers, unmet needs, and emerging technologies set to impact the market dynamics.
The report gives complete details of the market trend for each marketed Beta Thalassemia drug and mid & late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, their Mechanism of Action (MOA), Route of Administration (ROA), molecule types, competition with other therapies, brand value, and their impact on the market.
Beta Thalassemia Epidemiology Assessment
The epidemiology section provides insights into the historical, current, and forecasted Beta Thalassemia epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted Beta Thalassemia epidemiology trends by exploring numerous studies and research. The epidemiology section also provides a detailed analysis of diagnosed and prevalent patient pools, future trends, and views of key opinion leaders.
The Report Covers the Beta Thalassemia Epidemiology, Segmented as –
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Total Prevalent Cases of Beta Thalassemia in the 7MM [2019–2032]
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Diagnosed Prevalent Cases of Beta Thalassemia in the 7MM [2019–2032]
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Type-specific Diagnosed Prevalent Cases of Beta Thalassemia in the 7MM [2019–2032]
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Treatable Cases of Beta Thalassemia in the 7MM [2019–2032]
Beta Thalassemia Drugs Uptake and Pipeline Development Activities
The drug uptake section focuses on the uptake rate of potential drugs recently launched in the Beta Thalassemia market or expected to be launched during the study period. The analysis covers the Beta Thalassemia market uptake by drugs, patient uptake by therapies, and sales of each drug. Moreover, the therapeutics assessment section helps understand the market dynamics by drug sales, the most rapid drug uptake, and the reasons behind the maximal use of particular drugs. Additionally, it compares the Beta Thalassemia drugs based on their sale and market share.
The report also covers the Beta Thalassemia pipeline development activities. It provides valuable insights about different therapeutic candidates in various stages and the key Beta Thalassemia companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.
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Beta Thalassemia Therapeutics Analysis
Blood transfusions are the primary therapy for β-thalassemia, but the frequency depends upon the severity of the disease. Blood transfusions expose patients to other complications and risks, including iron overload, infections, cardiac disease, etc. Other treatment strategies include iron chelation therapy, splenomegaly/splenectomy, growth abnormalities, bone deformities treatment, and more.
To counter unmet market needs and provide better treatment choices for beta-thalassemia, several market players are working robustly either on single-agent novel molecules or on combination and dose modification of standard therapies. Several therapies are expected to be launched in the study period, likely to drive market growth during the study period. Currently, CRISPR Therapeutics is leading the therapeutics market with its Beta-thalassaemia drug candidates in the most advanced stage of clinical development.
In November 2019, the FDA approved REBLOZYL for the treatment of anemia in adult patients with beta-thalassemia who require regular red blood cell (RBC) transfusions. It is a recombinant fusion protein that binds several endogenous TGF-β superfamily ligands, diminishing Smad2/3 signaling. Luspatercept-aamt promoted erythroid maturation through differentiation of late-stage erythroid precursors (normoblasts) in mice.
In August 2022, the FDA approved ZYNTEGLO (betibeglogene autotemcel), the first cell-based gene therapy for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions. ZYNTEGLO is a one-time gene therapy product administered as a single dose. Each dose of ZYNTEGLO is a customized treatment created using the patient’s own cells (bone marrow stem cells) that are genetically modified to produce functional beta-globin.
Beta Thalassemia Companies Actively Working in the Therapeutics Market Include
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Novartis
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Merck
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Bristol Myers Squibb
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Chiesi Farmaceutici S.p.A
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Bluebird Bio
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Agios Pharmaceuticals
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Imara Inc.
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CRISPR Therapeutics
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Vertex Pharmaceuticals
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Vifor Pharma
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Ionis Pharmaceuticals
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Forma Therapeutics
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DisperSol Technologies
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SILENCE Therapeutics
And Many Others
Emerging and Marketed Beta Thalassemia Therapies Covered in the Report Include:
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Zynteglo (LentiGlobin BB305): Bluebird Bio
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Mitapivat (AG-348): Agios Pharmaceuticals
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CTX001: CRISPR Therapeutics
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VIT-2763: CSL Vifor
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Emeramide: EmeraMed
And Many More
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Table of Content (TOC)
1. Key Insights
2. Executive Summary
3. Beta Thalassemia Competitive Intelligence Analysis
4. Beta Thalassemia Market Overview at a Glance
5. Beta Thalassemia Disease Background and Overview
6. Beta Thalassemia Patient Journey
7. Beta Thalassemia Patient Population and Epidemiology Trends (In the US, EU5, and Japan)
8. Beta Thalassemia Treatment Algorithm, Current Treatment, and Medical Practices
9. Beta Thalassemia Unmet Needs
10. Key Endpoints of Beta Thalassemia Treatment
11. Beta Thalassemia Marketed Therapies
12. Beta Thalassemia Emerging Drugs and Latest Therapeutic Advances
13. Beta Thalassemia Seven Major Market Analysis
14. Attribute Analysis
15. Beta Thalassemia Market Outlook (In US, EU5, and Japan)
16. Beta Thalassemia Companies Active in the Market
17. Beta Thalassemia Access and Reimbursement Overview
18. KOL Views on the Beta Thalassemia Market
19. Beta Thalassemia Market Drivers
20. Beta Thalassemia Market Barriers
21. Appendix
22. DelveInsight Capabilities
23. Disclaimer
*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.
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About DelveInsight
DelveInsight is a leading Business Consultant and Market Research Firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance.
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