Beta Thalassemia Market: Insights into Epidemiology, Treatment Options, and Key Companies | DelveInsight | Novartis, Merck, Bristol Myers Squibb, Chiesi Farmaceutici S.p.A, Bluebird Bio, Agios Pharmac

In 2020, there are more than 900 beta-thalassemia major patients and more than 200 beta-thalassemia intermedia patients living in the UK.

Emerging therapies for Beta Thalassemia, including Mitapivat (AG-348) and others, are expected to drive significant growth in the market in the coming years.

DelveInsight has released a new report, "Beta Thalassemia - Market Insights, Epidemiology, and Market Forecast-2034", providing comprehensive insights into the disease, historical and projected epidemiology, and market trends across the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.

Discover about the Beta thalassemia market report @ https://www.delveinsight.com/report-store/beta-thalassemia-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Some of the key facts of the Beta thalassemia Market Report:

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In 2023, the Beta Thalassemia market in the United States was valued at approximately USD 358 million and is projected to expand significantly by 2034, exhibiting a notable Compound Annual Growth Rate (CAGR) over the period from 2020 to 2034. That same year, the US recorded around 1.35 million prevalent cases of Beta Thalassemia. The rising prevalence across the seven major markets (7MM) is attributed to improved diagnostic and screening methods, limited access to genetic counseling and family planning, and low public awareness.

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In the EU4 and the UK, Beta Thalassemia Major had a higher prevalence than Beta Thalassemia Intermedia. In 2023, approximately 7.39 million individuals were affected by Beta Thalassemia Major, with fluctuations expected over the 2020-2034 period. Among the 7MM, Italy reported the highest number of cases in 2023, estimated at 3.68 million, with figures expected to evolve through 2034. In Spain, Beta Thalassemia Major was the most common form, with around 217,000 cases in 2023, a number anticipated to rise during the 2024-2034 forecast period.

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Among current therapies, betibeglogene autotemcel is projected to capture the largest market share in the US by 2034. In 2023, the prevalence of Beta Thalassemia Minor in the US was approximately 1.36 million, with significant changes expected by 2034. Germany reported around 235,000 cases of Beta Thalassemia Minor in the same year.

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In January 2025, Agios Pharmaceuticals announced that the FDA accepted its supplemental new drug application (sNDA) to expand the approved use of Pyrukynd, its marketed therapy, to include treatment for thalassemia.

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Key companies involved in developing treatments for Beta Thalassemia include Novartis, Merck, Bristol Myers Squibb, Chiesi Farmaceutici S.p.A, Bluebird Bio, Agios Pharmaceuticals, Imara Inc., CRISPR Therapeutics, Vertex Pharmaceuticals, Vifor Pharma, Ionis Pharmaceuticals, Forma Therapeutics, DisperSol Technologies, SILENCE Therapeutics, and others. Promising therapies in development include Reblozyl, EXJADE, Mitapivat (AG-348), and additional agents expected to enhance the treatment landscape.

Beta thalassemia Overview

Beta Thalassemia (-Thalassemia) is a genetic blood disorder characterized by reduced synthesis of the beta-globin chain of hemoglobin. This results in microcytic, hypochromic anemia, the appearance of nucleated red blood cells in peripheral blood, and decreased hemoglobin A (HbA) levels on hemoglobin analysis. The disorder is inherited through mutations on Chromosome 11, with key risk factors including a family history of the disease and ethnic backgrounds such as Asian, Chinese, Mediterranean, or African American.

Severe, untreated -Thalassemia cases may present with profound anemia (hemoglobin 3-7 g/dL), hepatosplenomegaly, stunted growth, and skeletal deformities. Patients with Beta Thalassemia Major or Intermedia are at risk of iron overload, either from the disease itself or due to frequent blood transfusions, which can damage organs including the heart, liver, and endocrine glands. Individuals with the minor form generally have smaller red blood cells but are usually asymptomatic.

Beta Thalassemia is classified into three main types: Thalassemia Major (also called "Cooley's Anemia" or "Mediterranean Anemia"), Thalassemia Intermedia, and Thalassemia Minor (also referred to as Beta Thalassemia carrier, Beta Thalassemia trait, or heterozygous Beta Thalassemia).

Beta thalassemia Market Outlook

For patients with transfusion-dependent thalassemia (TDT), standard care involves regular blood transfusions combined with iron chelation therapy. Transfusions help reduce ineffective red blood cell production and associated complications, while iron chelation prevents and manages the accumulation of excess iron. The main goal in Beta Thalassemia management is to maintain hemoglobin levels between 9 and 10.5 g/dL, supporting normal growth, suppressing excessive bone marrow activity, and minimizing iron overload.

A key challenge with frequent transfusions is iron overload, as the body lacks a natural mechanism to eliminate excess iron. Iron accumulation, particularly in the heart, liver, and pituitary gland, can lead to serious complications such as heart failure, liver cirrhosis, hepatocellular carcinoma, endocrine disorders (including hypothyroidism, hypogonadism, and hypoparathyroidism), diabetes, and growth impairment. Iron chelation therapy helps restore iron balance by promoting its excretion through urine or feces.

Currently, Beta Thalassemia treatment is largely symptomatic, so the market is primarily driven by iron chelators, although future projections include potential revenue from emerging therapies.

In the six major markets, physicians typically prescribe monotherapy with chelators such as deferiprone (DFP), deferasirox (DFX), or desferrioxamine (DFO). Oral agents like DFP and DFX are preferred due to their convenience and cost-effectiveness, whereas DFO requires subcutaneous, intravenous, or occasionally intramuscular administration.

Desferal was the first FDA-approved iron chelation therapy in 1968. More recently, in 2019, the FDA approved Reblozyl (luspatercept) to treat anemia in adults with Beta Thalassemia who require regular red blood cell transfusions.

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Beta thalassemia Marketed Drugs

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Reblozyl: Merck/Bristol Myers Squibb

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EXJADE: Novartis

Beta thalassemia Emerging Drugs

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Mitapivat (AG-348): Agios Pharmaceuticals

Scope of the Beta thalassemia Market Report

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Study Period: 2020-2034

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Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]

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Key Beta thalassemia Companies: Novartis, Merck, Bristol Myers Squibb, Chiesi Farmaceutici S.p.A, Bluebird Bio, Agios Pharmaceuticals, Imara Inc., CRISPR Therapeutics, Vertex Pharmaceuticals, Vifor Pharma, Ionis Pharmaceuticals, Forma Therapeutics, DisperSol Technologies, SILENCE Therapeutics, and others

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Key Beta thalassemia Therapies: Reblozyl, EXJADE, Mitapivat (AG-348), and others

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Beta thalassemia Therapeutic Assessment: Beta thalassemia current marketed and Beta thalassemia emerging therapies

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Beta thalassemia Market Dynamics: Beta thalassemia market drivers and Beta thalassemia market barriers

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Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies

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Beta thalassemia Unmet Needs, KOL's views, Analyst's views, Beta thalassemia Market Access and Reimbursement

To know what's more in our Beta thalassemia report, visit https://www.delveinsight.com/report-store/beta-thalassemia-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Key benefits of the Beta thalassemia Market Report:

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Beta thalassemia market report covers a descriptive overview and comprehensive insight of the Beta thalassemia Epidemiology and Beta thalassemia market in the 7MM (the United States, EU5 (Germany, Spain, France, Italy, UK) & Japan).

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The Beta thalassemia market report provides insights into the current and emerging therapies.

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The Beta thalassemia market report provides a global historical and forecasted market covering drug outreach in 7MM.

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The Beta thalassemia market report offers an edge that will help in developing business strategies by understanding trends shaping and driving the Beta thalassemia market.

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Table of Contents

1. Report Introduction

2. Executive Summary

3. SWOT analysis

4. Beta thalassemia Patient Share (%) Overview at a Glance

5. Beta thalassemia Market Overview at a Glance

6. Beta thalassemia Disease Background and Overview

7. Beta thalassemia Epidemiology and Patient Population

8. Country-Specific Patient Population of Beta thalassemia

9. Beta thalassemia Current Treatment and Medical Practices

10. Unmet Needs

11. Beta thalassemia Emerging Therapies

12. Beta thalassemia Market Outlook

13. Country-Wise Beta thalassemia Market Analysis (2020-2034)

14. Market Access and Reimbursement of Therapies

15. Market drivers

16. Market barriers

17. Appendix

18. Beta thalassemia Report Methodology

19. DelveInsight Capabilities

20. Disclaimer

21. About DelveInsight

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Beta thalassemia Pipeline Insights, DelveInsight

"Beta thalassemia Pipeline Insight, 2024" report by DelveInsight outlines comprehensive insights of present clinical development scenarios and growth prospects across the Beta thalassemia market. A detailed picture of the Beta thalassemia pipeline landscape is provided, which includes the disease overview and Beta thalassemia treatment guidelines.

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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