Beta Thalassemia Market Trends Point to Steady Growth Ahead by 2034, DelveInsight Analysis Reveals

DelveInsight's "Beta Thalassemia Market Insights, Epidemiology, and Market Forecast-2034′′ report offers an in-depth understanding of the Beta Thalassemia, historical and forecasted epidemiology as well as the Beta Thalassemia market trends in the United States, EU4 (Germany, Spain, Italy, France) the United Kingdom and Japan.

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Some of the key facts of the Beta Thalassemia Market Report:
• The Beta Thalassemia market size is anticipated to grow with a significant CAGR during the study period (2020-2034)
• In January 2025, Agios Pharmaceuticals (AGIO) announced that the FDA has accepted its supplemental new drug application (sNDA) to expand the label of Pyrukynd, its sole marketed drug, to include the treatment of thalassemia.
• In January 2025, YolTech Therapeutics is preparing to launch a clinical trial for YOLT-204, its in vivo gene-editing therapy designed to treat transfusion-dependent beta-thalassemia (TDT). TDT is a genetic blood disorder caused by mutations in the beta-globin gene, leading to reduced or absent hemoglobin production. Patients with this condition require regular blood transfusions to manage anemia and prevent future complications.
• In 2023, the Beta-thalassemia market size in the US was around USD 358 million, with expectations of significant growth by 2034, driven by a substantial Compound Annual Growth Rate (CAGR) during the study period (2020-2034).
• The Beta-thalassemia market size in EU4 and the UK was around USD 356 million and is anticipated to grow during the forecast period.
• Among European countries, Italy had the largest Beta-thalassemia market size, reaching approximately USD 241 million in 2023.
• The Beta-thalassemia market size in the EU4 and the UK was roughly USD 356 million, with projections indicating further growth during the forecast period.
• Italy had the largest Beta-thalassemia market size among European countries, reaching around USD 241 million in 2023.
• In September 2023, Pharmacosmos initiated a Phase IItrial 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 FeeAct (PDUFA) target action date for TDT is March 30, 2024.
• The Centers for Disease Control and Prevention (2023) state that beta thalassemia is comparatively uncommon in the US
• In 2023, the United States had 1,359,000 prevalent cases of Beta-thalassemia Minor. These numbers are anticipated to change considerably by 2034.
• Germany had approximately 235,000 prevalent cases of Beta-thalassemia Minor in 2023.
• In the UK, a significant difference was seen in the prevalence of Beta-thalassemia types, with Beta Thalassemia Major being more common than Beta Thalassemia Intermedia. In 2023, there were about 745,000 cases of Beta-thalassemia Major recorded, and these numbers are anticipated to fluctuate further during the study period from 2020 to 2034.
• Italy had the highest number of prevalent Beta-thalassemia minor cases among the EU4 countries and the UK, with approximately 3,676,000 cases, followed by Spain with around 772,000 cases. These numbers are projected to change significantly by 2034.
• In 2023, France had approximately 678,000 diagnosed prevalent cases of Beta-thalassemia, with these numbers anticipated to change further by 2034.
• Key Beta Thalassemia Companies: Agios Pharmaceuticals, Vertex Pharmaceuticals, Pharmacosmos, Editas Medicine, HemaQuest Pharmaceuticals, Acceleron Pharma, Bristol-Myers Squibb, Hoffmann-La Roche, Sanofi, Novartis, EmeraMed, Pharmacosmos A/S, Celgene, DisperSol Technologies, LLC, Agios Pharmaceuticals, Shire, and others
• Key Beta Thalassemia Therapies: PYRUKYND (mitapivat), CTX001, SP-420, EDIT-301, dimethylbutyrate, ACE-536, Luspatercept, Bitopertin, Deferitrin (GT56-252), ICL670, Emeramide, SP-420, Luspatercept, DST-0509, Mitapivat, SPD602, and others
• The Beta Thalassemia market is expected to surge due to the disease's increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Beta Thalassemia pipeline products will significantly revolutionize the Beta Thalassemia market dynamics.

Beta Thalassemia Overview
Beta thalassemia is a genetic blood disorder characterized by reduced or absent production of hemoglobin beta chains, leading to abnormal red blood cells. This results in anemia, fatigue, and other complications due to insufficient oxygen delivery throughout the body. Beta thalassemia can range from mild to severe, with the severe form, known as thalassemia major, requiring regular blood transfusions and iron chelation therapy to manage iron overload. The condition is inherited in an autosomal recessive manner, and while there is no universal cure, treatments focus on managing symptoms and preventing complications. Stem cell or bone marrow transplants offer potential cures for certain cases.

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Beta Thalassemia Epidemiology
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 trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends.

Beta Thalassemia Epidemiology Segmentation:
The Beta Thalassemia market report proffers epidemiological analysis for the study period 2020-2034 in the 7MM segmented into:
• Total Prevalence of Beta Thalassemia
• Prevalent Cases of Beta Thalassemia by severity
• Gender-specific Prevalence of Beta Thalassemia
• Diagnosed Cases of Episodic and Chronic Beta Thalassemia

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Beta Thalassemia Drugs Uptake and Pipeline Development Activities
The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Beta Thalassemia market or expected to get launched during the study period. The analysis covers Beta Thalassemia market uptake by drugs, patient uptake by therapies, and sales of each drug.
Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on 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 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.

Beta Thalassemia Therapies and Key Companies
• PYRUKYND (mitapivat): Agios Pharmaceuticals
• CTX001: Vertex Pharmaceuticals
• SP-420: Pharmacosmos
• EDIT-301: Editas Medicine
• dimethylbutyrate: HemaQuest Pharmaceuticals
• ACE-536: Acceleron Pharma
• Luspatercept: Bristol-Myers Squibb
• Bitopertin: Hoffmann-La Roche
• Deferitrin (GT56-252): Sanofi
• ICL670: Novartis
• Emeramide: EmeraMed
• SP-420: Pharmacosmos A/S
• Luspatercept: Celgene
• DST-0509: DisperSol Technologies, LLC
• Mitapivat: Agios Pharmaceuticals
• SPD602: Shire

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Beta Thalassemia Market Strengths
• Strong pipeline activity with potential Phase III and phase II emerging therapies.
• Increasing prevalence and awareness of beta thalassemia

Beta Thalassemia Market Opportunities
• Lack of any curative treatment options despite the launch of gene therapies which are subject to patient outcome assessment
• Improved national blood policies
• Advancement in genetic engineering because gene therapy has been hypothesized to serve as an effective cure for monogenic blood disorders for several decades

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: Agios Pharmaceuticals, Vertex Pharmaceuticals, Pharmacosmos, Editas Medicine, HemaQuest Pharmaceuticals, Acceleron Pharma, Bristol-Myers Squibb, Hoffmann-La Roche, Sanofi, Novartis, EmeraMed, Pharmacosmos A/S, Celgene, DisperSol Technologies, LLC, Agios Pharmaceuticals, Shire, and others
• Key Beta Thalassemia Therapies: PYRUKYND (mitapivat), CTX001, SP-420, EDIT-301, dimethylbutyrate, ACE-536, Luspatercept, Bitopertin, Deferitrin (GT56-252), ICL670, Emeramide, SP-420, Luspatercept, DST-0509, Mitapivat, SPD602, 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

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About DelveInsight
DelveInsight is a leading Healthcare 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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