Beta thalassemia Market: Epidemiology, Therapies, Companies, DelveInsight | Novartis, Merck, Bristol Myers Squibb, Chiesi Farmaceutici S.p.A, Bluebird Bio, Agios Pharmaceuticals, Imara Inc., CRISPR Therapeutics, Vertex Pharmaceuticals, Vifor Pharma, Ionis

Beta thalassemia emerging therapies, such as Mitapivat (AG-348), and others, are expected to boost the Beta thalassemia Market in the upcoming years.

DelveInsight has launched a new report on "Beta thalassemia - Market Insights, Epidemiology, and Market Forecast-2034" that delivers an in-depth understanding of the Beta thalassemia, historical and forecasted epidemiology as well as the Beta thalassemia market trends in the United States, EU5 (Germany, Spain, Italy, France, and 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:

In 2023, the Beta Thalassemia market in the US was valued at approximately USD 358 million and is projected to grow significantly by 2034, with a notable Compound Annual Growth Rate (CAGR) over the study period (2020-2034).
That same year, the US reported around 1.35 million prevalent cases of Beta Thalassemia. The increase in prevalence across the 7MM (seven major markets) can be attributed to enhanced diagnostic and screening methods, limited access to genetic counseling and family planning services, and a general lack of public awareness.
In the EU4 and the UK, when prevalence was categorized by type, Beta Thalassemia Major had a higher number of cases compared to Beta Thalassemia Intermedia. In 2023, approximately 7.39 million individuals were affected by Beta Thalassemia Major, with this number expected to fluctuate over the study period (2020-2034).
Among the 7MM, Italy recorded the highest number of Beta Thalassemia cases in 2023, with an estimated 3.68 million cases-figures that are also anticipated to evolve through 2034.
In Spain, Beta Thalassemia Major was the most prevalent form, with around 217,000 cases in 2023. According to DelveInsight's analysis, this number is expected to rise during the forecast period (2024-2034).
Of the currently available treatments, betibeglogene autotemcel is projected to achieve the largest market share in the US by 2034.
In 2023, the total number of Beta Thalassemia Minor prevalent cases in the US was estimated at approximately 1.36 million. This figure is projected to undergo notable changes by 2034.
Germany reported around 235,000 prevalent cases of Beta Thalassemia Minor in the same year.
In January 2025, Agios Pharmaceuticals (AGIO) reported that the FDA had accepted its supplemental new drug application (sNDA) seeking to expand the approved use of Pyrukynd-its sole marketed therapy-to include the treatment of thalassemia.
Key Beta thalassemia companies such as 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 are evaluating new drugs for Beta thalassemia to improve the treatment landscape.
Promising Beta thalassemia therapies include Reblozyl, EXJADE, Mitapivat (AG-348), and others.

Beta thalassemia Overview

Beta Thalassemia (β-Thalassemia) is a genetic blood disorder marked by reduced production of the beta-globin subunit of hemoglobin. This leads to microcytic, hypochromic anemia, the presence of nucleated red blood cells in the peripheral blood smear, and decreased levels of hemoglobin A (HbA) on hemoglobin analysis. The condition is inherited and results from mutations on Chromosome 11. Key risk factors include a family history of the disorder and ethnic backgrounds such as Asian, Chinese, Mediterranean, or African American.

Severe, untreated cases of β-Thalassemia can present with symptoms like profound anemia (hemoglobin levels between 3-7 g/dL), significant enlargement of the liver and spleen (hepatosplenomegaly), severe growth delays, and skeletal abnormalities. Individuals with Beta Thalassemia major or intermediate forms often experience iron overload, either from the disease itself or due to frequent blood transfusions, which can harm vital organs such as the heart, liver, and endocrine glands. Those with the minor form typically have smaller red blood cells but usually show no symptoms.

Beta Thalassemia is categorized into three primary types: Thalassemia Major (also known as "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), the standard treatment involves regular blood transfusions and iron chelation therapy. Blood transfusions help reduce ineffective red blood cell production and associated complications, while iron chelation is used to manage and prevent issues caused by iron overload. The primary treatment goal in Beta Thalassemia is to maintain hemoglobin levels between 9 and 10.5 g/dL, which supports normal growth, suppresses bone marrow hyperactivity, and limits iron buildup.

A major concern with frequent transfusions is iron overload, as the human body lacks a natural mechanism to eliminate excess iron. Excess iron, particularly in organs such as the heart, liver, and pituitary gland, can result in severe complications including heart failure, liver cirrhosis, hepatocellular carcinoma, endocrine disorders (e.g., hypothyroidism, hypogonadism, hypoparathyroidism), diabetes, and growth impairment. Iron chelation therapy helps maintain iron homeostasis by promoting iron excretion through urine or feces.

Since treatment for the target patient population is largely symptomatic, the current Beta Thalassemia market primarily reflects revenue from iron chelators. However, projections also account for potential revenue from emerging therapies.

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

Desferal was the first iron chelation therapy approved by the FDA in 1968. More recently, in 2019, the FDA approved Reblozyl (luspatercept) for treating anemia in adults with Beta Thalassemia who need regular red blood cell transfusions.

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Beta thalassemia Marketed Drugs
Reblozyl: Merck/Bristol Myers Squibb
EXJADE: Novartis

Beta thalassemia Emerging Drugs
Mitapivat (AG-348): Agios Pharmaceuticals

Scope of the Beta thalassemia Market Report
Study Period: 2020-2034
Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
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
Key Beta thalassemia Therapies: Reblozyl, EXJADE, Mitapivat (AG-348), and others
Beta thalassemia Therapeutic Assessment: Beta thalassemia current marketed and Beta thalassemia emerging therapies
Beta thalassemia Market Dynamics: Beta thalassemia market drivers and Beta thalassemia market barriers
Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies
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:
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).
The Beta thalassemia market report provides insights into the current and emerging therapies.
The Beta thalassemia market report provides a global historical and forecasted market covering drug outreach in 7MM.
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.

Contact Info:

Kritika Rehani, Assistant Manager, Marketing & Branding
krehani@delveinsight.com
info@delveinsight.com
www.delveinsight.com
+14699457679

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.

This release was published on openPR.