Global Beta Thalassemia Market - 2024-2031

Global Beta Thalassemia Market - 2024-2031

The global beta thalassemia market reached US$ 480.32 million in 2023 and is expected to reach US$ 889.04 million by 2031 growing with a CAGR of 8.1% during the forecast period 2024-2031.

Beta thalassemia is a blood disorder that reduces the production of hemoglobin. Hemoglobin is the iron-containing protein in red blood cells that carries oxygen to cells throughout the body.

In people with beta thalassemia, low levels of hemoglobin reduce oxygen levels in the body. Affected individuals also have a shortage of red blood cells (anemia), which can cause pale skin, weakness, fatigue, and more serious complications. People with beta thalassemia are at an increased risk of developing abnormal blood clots.

Market Drivers: Dynamics

Increasing Research & Developments and Rising FDA Approvals for Beta Thalassemia

The global beta thalassemia market has witnessed significant growth due to various recent research and developments. Beta Thalassemia is usually diagnosed based on medical history and clinical and imaging findings. Also, advanced research and developments in treating beta-thalassemia help to accelerate market growth.

For instance, in June 2023, CRISPR Therapeutics announced the positive results from pivotal trials of Exa-cel for transfusion-dependent beta-thalassemia and severe sickle cell disease presented at the 2023 Annual European Hematology Association (EHA) Congress. Exa-cel is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited cell therapy that is being evaluated for patients with transfusion-dependent beta-thalassemia (TDT).

Moreover, the increase in treatment approvals by regulatory bodies helps to drive market growth. The process of getting approval from the FDA generally involves preclinical experiments in an animal model of the disease. If the results of the animal model experiments are favorable then the FDA can approve the proposed structure of an interventional study.

For instance, in August 2022, Bluebird Bio, Inc. announced the U.S. Food and Drug Administration (FDA) has approved ZYNTEGLO (betibeglogene autotemcel) also known as beti-cel, a one-time gene therapy custom-designed to treat the underlying genetic cause of beta‑thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions. Thus, the above factors help to boost the market growth.

Restraints

Factors such as the high cost of treatment, side effects associated with the treatment, stringent regulatory guidelines, limited treatment options, and lack of awareness among individuals are expected to hamper the market.

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Segment Analysis

The global beta thalassemia market is segmented based on type, treatment, route of administration, end users, and region.

The stem cell transplant segment accounted for approximately 38.5% of the global beta thalassemia market share

The stem cell transplant segment is expected to hold the largest market share over the forecast period. A stem cell transplant can cure beta-thalassemia. Bone marrow transplantation (BMT) is a medical procedure during which stem cells are transferred from a healthy individual into the blood-producing tissue in the bone marrow of the individual who has a blood disease such as β-thalassemia.

Moreover, government initiatives such as awareness programs, product launches, and approvals would drive this market growth. For instance, in May 2023, the Coal India CSR-funded Hematopoietic Stem Cell Transplant (HSCT) program is a unique initiative aimed at providing a one-time cure opportunity to underprivileged Thalassemia patients who have a matched sibling donor but do not have the financial resources to cover the cost of the procedure.

The program has completed 356 bone marrow transplants for Thalassemia patients across 10 impaneled hospitals in India during the two phases.

Similarly in January 2024, Vertex Pharmaceuticals Incorporated announced that the U.S. Food and Drug Administration (FDA) has approved CASGEVY (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited cell therapy, for the treatment of transfusion-dependent beta-thalassemia (TDT) in patients 12 years and older.

Geographical Analysis

North America accounted for approximately 41.3% of the global beta thalassemia market share

North America region is expected to hold the largest market share over the forecast period owing to the rising number of thalassemia patients in the UK driving this market growth in this region.

For instance, in December 2023, the United Kingdom’s National Health Service (NHS) will begin funding allogeneic hematopoietic stem cell transplants for adult patients with beta-thalassemia following new guidance from NHS England’s Clinical Priorities Advisory Group.

The NHS estimates that around 600 adults across the U.K. have transfusion-dependent thalassemia – the most severe form of the disease – while around 2,281 people with thalassaemia are registered on the U.K.’s National Haemoglobinopathy. This includes 1,332 patients over the age of 18.

In this region, key player's presence and new product approvals would drive this market growth. For instance, in August 2022, bluebird bio, Inc. announced the FDA has approved ZYNTEGLO (betibeglogene autotemcel), also known as beti-cel, a one-time gene therapy custom-designed to treat the underlying genetic cause of beta-thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions.

Market Segmentation

By Type
• Beta-Thalassemia Major
• Beta-Thalassemia Intermedia
• Beta-Thalassemia Minor

By Treatment
• Iron Chelation Therapy
• Folic Acid Supplements
• Blood Transfusions
• Erythroid Maturation Agents
• Stem Cell Transplant
• Others

By Route of Administration
• Oral
• Intravenous
• Others

By End Users
• Hospital
• Clinics
• Others

By Region
• North America
U.S.
Canada
Mexico
• Europe
Germany
U.K.
France
Italy
Spain
Rest of Europe
• South America
Brazil
Argentina
Rest of South America
• Asia-Pacific
China
India
Japan
South Korea
Rest of Asia-Pacific
• Middle East and Africa

Competitive Landscape

The major global players in the beta thalassemia market include Bristol Myers Squibb, Par Pharmaceuticals, Qilu Pharmaceuticals, Taro Pharmaceuticals, Blue Bird Bio, Cipla Ltd., Novartis A.G, Teva Pharmaceuticals, Piramal Pharma Solutions, and LGM Pharma among others.

Key Developments

 In April 2023, a non-profit organization named ECHO India teamed up with the Post Graduate Institute of Child Health (PGICH) to launch a nationwide program focused on the Prevention and Control of Beta Thalassemia and Other Hemoglobinopathies.

 In April 2022, Vertex Pharmaceuticals Incorporated in collaboration with CRISPR Therapeutics announced the completion of the rolling Biologics License Applications (BLAs) to the U.S. Food and Drug Administration (FDA) for the investigational treatment exagamglogene autotemcel (exa-cel) for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia.

 In March 2022, Bristol Myers Squibb announced that the U.S. Food and Drug Administration (FDA) has extended the review of the supplemental biologics license application (sBLA) for Reblozyl) for the treatment of anemia in adults with non-transfusion-dependent (NTD) beta-thalassemia.

Why Purchase the Report??
• To visualize the global beta thalassemia market segmentation based on type, treatment, route of administration, end users, and region, as well as understand key commercial assets and players.
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The global beta thalassemia market report would provide approximately 69 tables, 70 figures, and 195 Pages.

Target Audience 2023
• Manufacturers/ Buyers
• Industry Investors/Investment Bankers
• Research Professionals
• Emerging Companies