As per DelveInsight, the Beta-thalassemia Market is anticipated to evolve immensely in the coming years owing to the rise in the prevalence of Beta-thalassemia and the anticipated launch of novel therapies in the market.
Some of the key products in the Beta-thalassemia therapeutics market landscape include Zynteglo (LentiGlobin BB305), Mitapivat, Sapablursen, among others.
Key highlights from the beta-thalassemia market report:
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The Beta-thalassemia market in the United States was valued at approximately USD 358 million in 2023, with projections indicating significant growth during the forecast period (2020–2034), driven by a robust Compound Annual Growth Rate (CAGR).
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In 2023, there were approximately 1.35 million prevalent cases of Beta-thalassemia in the U.S. The rise in the number of prevalent cases across the 7MM (United States, EU4, UK, and Japan) is largely attributed to improvements in diagnosis and screening, along with limited access to genetic counseling, family planning, and a general lack of public awareness.
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In the EU4 and the UK, Beta-thalassemia Major was more common than Beta-thalassemia Intermedia, with approximately 7,391 thousand cases of Beta-thalassemia Major recorded in 2023. These numbers are expected to evolve over the study period (2020–2034).
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Among the 7MM, Italy reported the highest number of Beta-thalassemia prevalent cases, with approximately 3,676 thousand cases in 2023. This figure is expected to increase as the market progresses through 2034. In Spain, Beta-thalassemia Major accounts for the majority of cases, with around 217 thousand cases recorded in 2023. These cases are projected to rise during the forecast period (2024–2034), according to DelveInsight’s analysis.
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Among the currently approved treatments, betibeglogene autotemcel is expected to capture the largest share of the Beta-thalassemia market in the U.S. by 2034.
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With the anticipated approval of new therapies during the forecast period (2024–2034), the overall Beta-thalassemia therapeutic market is set to experience a substantial surge, with significant growth projected at a high CAGR.
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According to DelveInsight, the Beta-thalassemia market is expected to experience significant growth in the coming years, driven by the increasing prevalence of the disease and the anticipated introduction of innovative therapies.
To address the unmet needs in the Beta-thalassemia treatment landscape and provide improved options for patients, several pharmaceutical and biotechnology companies are actively developing novel single-agent molecules, as well as combination therapies and dose modifications of existing treatments. The upcoming launch of several new therapies is expected to further fuel market expansion during the forecast period.
Bluebird Bio, Agios Pharmaceuticals, Ionis Pharmaceuticals, and other leading companies are at the forefront of this market. Some of the key products making an impact in the Beta-thalassemia therapeutics landscape include Zynteglo (LentiGlobin BB305), Mitapivat, and Sapablursen, among others. These therapies are poised to contribute significantly to the growth of the Beta-thalassemia market in the years to come.
DelveInsight’s “Beta-thalassemia (B-thal) Market Insights, Epidemiology, and Market Forecast 2034” report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the Beta-thalassemia (B-thal) 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 Beta-thalassemia (B-thal) market report covers emerging drugs, current treatment practices, market share of individual therapies, and current & forecasted market size from 2020 to 2034. It also evaluates the current 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 Overview
Beta-thalassemia (β-thalassemia) is a hereditary blood disorder characterized by a reduction in the production of the hemoglobin beta chain, leading to microcytic hypochromic anemia, abnormal red blood cell morphology (including nucleated red blood cells), and a decrease in hemoglobin A (HbA) levels.
In its severe, untreated form, β-thalassemia presents with severe anemia (Hb levels ranging from 3−7 g/dL), hepatosplenomegaly, growth retardation, and bone deformities. Individuals with beta-thalassemia major or intermediate forms often experience iron overload due to the disease itself or frequent blood transfusions. Excess iron accumulation can lead to organ damage, particularly in the heart, liver, and endocrine system. Conversely, individuals with beta-thalassemia minor typically exhibit small red blood cells but remain asymptomatic.
Beta-thalassemia is classified into three main forms:
– Thalassemia Major (sometimes referred to as “Cooley’s Anemia” or “Mediterranean Anemia”)
– Thalassemia Intermediate
– Thalassemia Minor (also known as beta-thalassemia carrier, beta-thalassemia trait, or heterozygous beta-thalassemia).
Beta-thalassemia (B-thal) Market Outlook:
Blood transfusion and iron chelation are the cornerstone treatments for patients with Transfusion-Dependent Thalassemia (TDT). Blood transfusions help manage ineffective hematopoiesis and its associated complications, while iron chelation therapies address and prevent complications arising from iron overload. The primary treatment goal for Beta Thalassemia is to maintain a hemoglobin level between 9–10.5 g/dL, which supports normal growth, suppresses abnormal bone marrow activity, and minimizes iron buildup.
A significant complication of frequent blood transfusions is iron overload, as the body lacks a natural mechanism to eliminate excess iron. Iron accumulates in vital organs, such as the heart, liver, and pituitary gland, leading to serious conditions like heart failure, cirrhosis, hepatocellular carcinoma, hypothyroidism, hypoparathyroidism, hypogonadism, diabetes, and growth failure. The primary aim of treatment is to manage and prevent iron overload by maintaining a balance of iron in the body, promoting its excretion through urine or feces.
Since treatment for Beta Thalassemia is primarily symptomatic, the market covered in this report focuses specifically on revenue generated from iron chelation therapies. However, the forecast also includes potential market share for upcoming therapies that address other aspects of disease management.
In the six major markets, physicians typically recommend monotherapy with iron chelators such as deferiprone (DFP), deferasirox (DFX), and desferrioxamine (DFO). DFX and DFP are preferred due to their cost-effectiveness and ease of administration (oral), while DFO requires more complex administration via subcutaneous, intravenous, or occasional intramuscular routes.
Desferal (DFO) was the first iron chelator approved by the FDA in 1968 for the treatment of iron overload. More recently, Reblozyl (luspatercept) received FDA approval in 2019 for the treatment of anemia in adult patients with Beta Thalassemia who require regular blood transfusions.
Beta-thalassemia (B-thal) Epidemiology Assessment
The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. It helps to recognize the causes of current and forecasted epidemiology trends by exploring numerous studies and research. The epidemiology section also provides a detailed analysis of diagnosed and prevalent patient pool, future trends, and views of key opinion leaders.
The Beta-thalassemia market in the United States was valued at approximately USD 358 million in 2023 and is projected to grow significantly during the forecast period (2020–2034), driven by a strong Compound Annual Growth Rate (CAGR). The U.S. holds the largest share of the global Beta-thalassemia market, representing around 50% of the total market size across the 7MM (US, EU4, UK, Japan).
Emerging therapies for Beta-thalassemia, such as Mitapivat (AG-348) and others, are expected to drive substantial growth in the market, potentially reshaping treatment paradigms and further expanding market size.
In the EU4 and UK, the Beta-thalassemia market was valued at approximately USD 356 million in 2023 and is also expected to experience growth over the forecast period. Among European countries, Italy stands out with the largest market size, estimated at USD 241 million in 2023.
The anticipated launch of innovative therapies, along with an increase in the prevalence of Beta-thalassemia, is expected to drive further expansion in the market, particularly in the coming years.
The Report Covers the Beta-thalassemia (B-thal) Epidemiology, Segmented as –
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Total Prevalent Population of Beta Thalassemia (2020–2034)
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Total Prevalent Cases of Beta Thalassemia Minor (2020–2034)
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Diagnosed Prevalence of Beta Thalassemia (2020–2034)
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Diagnosed Beta-Thalassemia Patients by Disease Type (2020–2034)
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Leading Companies in the Beta Thalassemia Therapeutics Market Include:
Acceleron Pharma, Agios Pharmaceuticals, Inc., Allife Medical Science and Technology, Aruvant Sciences, Beam Therapeutics, Bioray Laboratories, Bluebird Bio, Bristol Myers Squibb, Chiesi Farmaceutici S.p.A, CRISPR Therapeutics, Disc Medicine, DisperSol Technologies, EdiGene (GuangZhou) Inc., Editas Medicine, EmeraMed, Forma Therapeutics, Fulcrum Therapeutics, Global Blood Therapeutics, Inc, Graphite Bio, Imara, Inc., Ionis Pharmaceuticals, Inc., Merck, Novartis, Orchard Therapeutics, Phoenicia Biosciences, Regenacy Pharmaceuticals, San Rocco Therapeutics, Sangamo Therapeutics, Shanghai BDgene, Silence Therapeutics plc, Vertex Pharmaceuticals, Vifor Pharma, and many others.
Beta Thalassemia Emerging and Marketed Drugs Covered in the Report Include:
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Benserazide: Phoenicia Biosciences
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CTX001: CRISPR Therapeutics
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EDIT-301: Editas Medicine
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ET-01: EdiGene Inc
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GPH 102: Graphite Bio
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IMR-687: Imara
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Mitapivat: Agios Pharmaceutical
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Sapablursen: Ionis Pharmaceutical
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SLN124: Silence Therapeutics
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Vamifeport: Vifor Pharma
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Zynteglo (LentiGlobin BB305): Bluebird Bio
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And Many More
Report Covers the In-depth Assessment of the Emerging Drugs & Key Companies. Download the Sample Report to Learn More @ https://www.delveinsight.com/sample-request/beta-thalassemia-market
Table of Contents
1. Key Insights
2. Executive Summary
3. Beta-thalassemia (B-thal) Competitive Intelligence Analysis
4. Beta-thalassemia (B-thal) Market Overview at a Glance
5. Beta-thalassemia (B-thal) Disease Background and Overview
6. Beta-thalassemia (B-thal) Patient Journey
7. Beta-thalassemia (B-thal) Epidemiology and Patient Population (In the US, EU5, and Japan)
8. Beta-thalassemia (B-thal) Treatment Algorithm, Current Treatment, and Medical Practices
9. Beta-thalassemia (B-thal) Unmet Needs
10. Key Endpoints of Beta-thalassemia (B-thal) Treatment
11. Beta-thalassemia (B-thal) Marketed Products
12. Beta-thalassemia (B-thal) Emerging Drugs and Latest Therapeutic Advances
13. Beta-thalassemia (B-thal) Seven Major Market Analysis
14. Attribute Analysis
15. Beta-thalassemia (B-thal) Market Outlook (In US, EU5, and Japan)
16. Beta-thalassemia (B-thal) Access and Reimbursement Overview
17. KOL Views on the Beta-thalassemia (B-thal) Market
18. Beta-thalassemia (B-thal) Market Drivers
19. Beta-thalassemia (B-thal) Market Barriers
20. Appendix
21. DelveInsight Capabilities
22. Disclaimer
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