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  • Published On : Nov 2021
  • Code : CMI4793
  • Industry : Pharmaceutical
  • Pages : 192
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Beta thalassemia is an inherited blood disorder in which the patient’s body doesn't make hemoglobin normally. Hemoglobin is the part of red blood cells (RBCs) that carries oxygen throughout the human body. The abnormal hemoglobin can lead to anemia (not enough RBCs in the body) and other medical problems weakness, shortness of breath. Depending on the type of beta thalassemia, symptoms can be mild or very severe. Moreover, clinically there can be two main categories of beta-thalassemia patients based on the patient's need for blood transfusion transfusion-dependent thalassemia (TDT) and non-transfusion dependent thalassemia (NTDT). The diagnosis of beta thalassemia relies on measuring red blood cell indices that reveal microcytic hypochromic anemia, nucleated red blood cells on peripheral blood smear, hemoglobin analysis that reveals decreased amounts of HbA and increased amounts of hemoglobin F (HbF) after age of 12 months.

The global beta-thalassemia (B-thal) market is estimated to be valued at US$ 411.8 million in 2021 and is expected to exhibit a CAGR of 7.8% over the forecast period (2021-2028).

Figure 1. Global Beta-thalassemia (B-thal) Market Share (%), By Region, 2021

Beta-Thalassemia  | Coherent Market Insights

The increasing incidence of beta-thalassemia is expected to drive the growth of the global beta-thalassemia (B-thal) market over the forecast period.

Rising number of patient population suffering from beta-thalassemia globally is anticipated to drive the market growth over the forecast period.

For instance, according to the report published by Bristol-Myers Squibb Company, a global biopharmaceutical company, in November 2018, stated that about 1.5% of the global population (an estimated 80-90 million people) are carriers of β-thalassemia. Although β-thalassemia is relatively rare in the U.S., there are an estimated 1.25 million carriers, making up 0.4% of the population. Moreover, according to the same source, thalassemia affects about 2,000 patients living in the U.S., with 1,000 patients having β-thalassemia major.

For instance, according to the article published in the Journal of European Hematology Association in June 2019, stated that the annual incidence of symptomatic individuals suffering from β-thalassemia is estimated at between one in 100,000 worldwide and one in 10,000 in Europe.

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Beta-thalassemia (B-thal) Market Report Coverage

Report Coverage Details
Base Year: 2020 Market Size in 2021: US$ 411.8 Mn
Historical Data for: 2017 to 2020 Forecast Period: 2021 to 2028
Forecast Period 2021 to 2028 CAGR: 7.8% 2028 Value Projection: US$ 698.3 Mn
Geographies covered:
  • North America: U.S. and Canada
  • Latin America: Brazil, Argentina, Mexico, and Rest of Latin America
  • Europe: Germany, U.K., Spain, France, Italy, Russia, and Rest of Europe
  • Asia Pacific: China, India, Japan, Australia, South Korea, ASEAN, and Rest of Asia Pacific
  • Middle East: GCC Countries, Israel, and Rest of Middle East
  • Africa: South Africa, North Africa, and Central Africa
Segments covered:
  • By Treatment: Iron Chelating Drugs, Erythroid Maturation Agents, Stem Cell Therapy, Others
  • By Disease Indication: Beta Thalassemia Minor, Beta Thalassemia Intermedia, Beta Thalassemia Major
Companies covered:

Celgene Corp., BlueBird Bio, Protagonist Therapeutics, Agios Pharmaceuticals, Ionis Pharmaceuticals, Vifor Pharma, Orchard Therapeutics, Sangamo Therapeutics, CRISPR Therapeutics, DisperSol Technologies, Kiadis Pharma, Incyte Corporation, Editas Medicine, Global Blood Therapeutics, and Syros Pharmaceuticals

Growth Drivers:
  • Increasing incidence of beta-thalassemia 
  • Increasing research and development activities for the development and approval of novel therapeutics for the treatment of beta-thalassemia
Restraints & Challenges:
  • Strict pricing and reimbursement policies
  • Extremely high cost of drugs

Figure 2. Global Beta-thalassemia (B-thal) Market Value (US$ Mn), By Treatment, 2021

Beta-Thalassemia  | Coherent Market Insights

The increasing research and development activities for the development and approval of novel therapeutics for the treatment of beta-thalassemia is expected to drive the market growth over the forecast period.

Key players operating in the market are focusing on submitting application for approval of drugs therapy for the treatment of beta-thalassemia, which is expected to drive growth of the global beta-thalassemia (B-thal) market over the forecast period.

For instance, in September 2021, Bluebird bio, a U.S. based biotechnology company, announced that its beti-cel gene therapy had been officially submitted for approval in the U.S., for the treatment of beta-thalassemia. Beti-cel was tested in multiple early- and late-stage clinical trials as a one-time treatment for beta-thalassemia, a rare condition that impairs the production of hemoglobin, the oxygen-carrying protein in red blood cells. Bluebird bio, a biopharmaceutical company is submitted the approval letter to the Food and Drug Administration to approve its therapy for adults and children with beta-thalassemia who need regular transfusions of red blood cells. Beti-cel is already cleared for market in Europe, where it's sold under the brand name Zynteglo.

Furthermore, in November 2019, the U.S. Food and Drug Administration granted approval to Reblozyl (luspatercept–aamt) for the treatment of anemia (lack of red blood cells) in adult patients with beta thalassemia who require regular red blood cell (RBC) transfusions.

Global Beta-thalassemia (B-thal) Market – Impact of Coronavirus (Covid-19) Pandemic

During this COVID-19 pandemic, pharmaceutical business, clinical tool companies and likewise biotechnology firms are facing difficulties that are occurring from the interruption in supply chains and the demand to transform service processes. As the researches reveal that there is no relationship in between beta-thalassemia as well as coronavirus, hence beta-thalassemia market is being affected by the pandemic. If COVID-19 pandemic proceeds for a medium/long duration, it may impact the supply of product and also active ingredients around the world along with the import as well as export of drugs. There has been a disturbance in the treatment market. As coronavirus has actually ended up being the major emphasis on beta-thalassemia market and all study institutes, biotech as well as pharmaceutical companies are selected in cooperation job to manage COVID-19, who are previously engaged in research and development of therapeutics for the treatment of beta-thalassemia.

Global Beta-thalassemia (B-thal) Market Restraint

The strict pricing and reimbursement policies, high cost of drugs used in the treatment of beta-thalassemia are the factors that are expected to hinder growth of the global beta-thalassemia market over the forecast period.

Key Players

Major players operating in the global beta-thalassemia (B-thal) market include Celgene Corp., BlueBird Bio, Protagonist Therapeutics, Agios Pharmaceuticals, Ionis Pharmaceuticals, Vifor Pharma, Orchard Therapeutics, Sangamo Therapeutics, CRISPR Therapeutics, DisperSol Technologies, Kiadis Pharma, Incyte Corporation, Editas Medicine, Global Blood Therapeutics, and Syros Pharmaceuticals.

Thalassemia is a term for a group of disorders in which there is reduced levels of hemoglobin, decreased red blood cell production and anemia. There are two main forms – alpha thalassemia and beta thalassemia, each with various subtypes. Beta thalassemia minor, also known as beta thalassemia trait, is a common condition. The symptoms and severity of beta thalassemia varies greatly from one person to another. Individuals with beta thalassemia minor do not develop symptoms of the disorder but may have a mild anemia. Several individuals with beta thalassemia minor go through life never knowing they carry an altered gene for the disorder. A beta thalassemia major diagnosis is usually made during the first two years of life and individuals require regular blood transfusions and lifelong medical care to survive. When the disorder develops later during life, a diagnosis of beta thalassemia intermedia is given; individuals may only require blood transfusions on rare, specific instances. Most beta thalassemia cases are caused by a mutation in the HBB gene. In extremely rare cases, a loss of genetic material (deletion) that includes the HBB gene causes the disorder. Genes provide instructions for creating proteins that play a critical role in many body functions. When a gene mutation occurs, the protein product may be faulty, inefficient, or absent. Depending upon the functions of the particular protein, this can affect several organ systems of the body. Individuals with beta thalassemia minor have a mutation in one HBB gene and are carriers for the disorder. Individuals with beta thalassemia intermedia or major have mutations in both HBB genes.

Market Dynamics

The increasing incidence of beta thalassemia, rising research and development activities for the development of novel therapeutics for the treatment of beta thalassemia, and increasing adoption of inorganic growth strategies such as acquisitions and collaborations by key players operating in the market, are the major factors that are expected to drive growth of the global beta-thalassemia (B-thal) market over the forecast period.

For instance, in April 2018, GSK, a global pharmaceutical company and Orchard Therapeutics, a pharmaceutical company, entered into a strategic agreement under which GSK will transfer its portfolio of approved and investigational rare disease gene therapies to Orchard. This acquisition strengthens Orchard’s position as a global leader in gene therapy for rare diseases. GSK will continue to invest in the development of its platform capabilities in cell and gene therapies, with a focus on oncology.

Moreover, the portfolio of gene therapy programs Orchard had acquired includes, Strimvelis, the first autologous ex vivo gene therapy for children with adenosine deaminase severe combined immunodeficiency (ADA-SCID), approved by the EMA in 2016, two late-stage clinical programs in ongoing registration studies for metachromatic leukodystrophy (MLD) and Wiskott Aldrich syndrome (WAS), and one clinical program for beta thalassemia.

Key features of the study:

  • This report provides in-depth analysis of the global beta-thalassemia (B-thal) market, and provides market size (US$ Mn) and compound annual growth rate (CAGR%) for the forecast period (2021–2028), considering 2020 as the base year
  • It elucidates potential revenue opportunities across different segments and explains attractive investment proposition matrices for this market
  • This study also provides key insights about market drivers, restraints, opportunities, new product launches or approval, market trends,  regional outlook, and competitive strategies adopted by key players
  • It profiles key players in the global beta-thalassemia (B-thal) market based on the following parameters – company overview, financial performance, product portfolio, market presence, distribution strategies, key developments, strategies, and future plans
  • Key companies covered as a part of this study include Celgene Corp., BlueBird Bio, Protagonist Therapeutics, Agios Pharmaceuticals, Ionis Pharmaceuticals, Vifor Pharma, Orchard Therapeutics, Sangamo Therapeutics, CRISPR Therapeutics, DisperSol Technologies, Kiadis Pharma, Incyte Corporation, Editas Medicine, Global Blood Therapeutics, and Syros Pharmaceuticals
  • Insights from this report would allow marketers and the management authorities of the companies to make informed decisions regarding their future product launches, type up-gradation, market expansion, and marketing tactics
  • The global beta-thalassemia (B-thal) market report caters to various stakeholders in this industry including investors, suppliers, product manufacturers, distributors, new entrants, and financial analysts
  • Stakeholders would have ease in decision-making through various strategy matrices used in analyzing the global beta-thalassemia (B-thal) market

Detailed Segmentation:

  • Global Beta-thalassemia (B-thal) Market, By Treatment:
    • Iron Chelating Drugs
    • Erythroid Maturation Agents
    • Stem Cell Therapy
    • Others
  • Global Polycythemia Vera Therapeutics Market, By Disease Indication:
    • Beta Thalassemia Minor
    • Beta Thalassemia Intermedia
    • Beta Thalassemia Major
  • Global Polycythemia Vera Therapeutics Market, By Region:
    • North America
      • By Treatment
        • Iron Chelating Drugs
        • Erythroid Maturation Agents
        • Stem Cell Therapy
        • Others
      • Disease Indication
        • Beta Thalassemia Minor
        • Beta Thalassemia Intermedia
        • Beta Thalassemia Major
      • By Country
        • U.S.
        • Canada
    • Latin America
      • By Treatment
        • Iron Chelating Drugs
        • Erythroid Maturation Agents
        • Stem Cell Therapy
        • Others
      • Disease Indication
        • Beta Thalassemia Minor
        • Beta Thalassemia Intermedia
        • Beta Thalassemia Major
      • By Country
        • Brazil
        • Mexico
        • Argentina
        • Rest of Latin America
    • Europe
      • By Treatment
        • Iron Chelating Drugs
        • Erythroid Maturation Agents
        • Stem Cell Therapy
        • Others
      • Disease Indication
        • Beta Thalassemia Minor
        • Beta Thalassemia Intermedia
        • Beta Thalassemia Major
      • By Country
        • Germany
        • U.K.
        • France
        • Italy
        • Spain
        • Russia
        • Rest of Europe
    • Asia Pacific
      • By Treatment
        • Iron Chelating Drugs
        • Erythroid Maturation Agents
        • Stem Cell Therapy
        • Others
      • Disease Indication
        • Beta Thalassemia Minor
        • Beta Thalassemia Intermedia
        • Beta Thalassemia Major
      • By Country
        • China
        • India
        • Japan
        • Australia
        • South Korea
        • ASEAN
        • Rest of Asia Pacific
    • Middle East
      • By Treatment
        • Iron Chelating Drugs
        • Erythroid Maturation Agents
        • Stem Cell Therapy
        • Others
      • Disease Indication
        • Beta Thalassemia Minor
        • Beta Thalassemia Intermedia
        • Beta Thalassemia Major
      • By Country
        • GCC
        • Israel
        • Rest of Middle East
    • Africa
      • By Treatment
        • Iron Chelating Drugs
        • Erythroid Maturation Agents
        • Stem Cell Therapy
        • Others
      • Disease Indication
        • Beta Thalassemia Minor
        • Beta Thalassemia Intermedia
        • Beta Thalassemia Major
      • By Region/Country
        • South Africa
        • Central Africa
        • North Africa
  • Company Profiles
    • Celgene Corp. *
      • Company Overview
      • Material Portfolio
      • Financial Performance
      • Key Highlights
      • Market Strategies
    • BlueBird Bio
    • Protagonist Therapeutics
    • Agios Pharmaceuticals
    • Ionis Pharmaceuticals
    • Vifor Pharma
    • Orchard Therapeutics
    • Sangamo Therapeutics
    • CRISPR Therapeutics
    • DisperSol Technologies
    • Kiadis Pharma
    • Incyte Corporation
    • Editas Medicine
    • Global Blood Therapeutics
    • Syros Pharmaceuticals

“*” marked represents similar segmentation in other categories in the respective section.

Frequently Asked Questions

The global beta-thalassemia (B-thal) market size is estimated to be valued at US$ 411.8 million in 2021 and is expected to exhibit a CAGR of 7.8% between 2021 and 2028.
Increasing incidence of beta-thalassemia and increasing research and development activities for the development and approval of novel therapeutics for the treatment of beta-thalassemia are expected to drive the market growth over the forecast period.
Erythroid Maturation Agents segment is expected to hold the major market share, owing to increasing product approvals and launches during the forecast period.
North America holds the largest market share in the market.
Major factors hampering growth of the market include the strict pricing and reimbursement policies, and extremely high cost of the drugs.
Major players operating in the market include Celgene Corp., BlueBird Bio, Protagonist Therapeutics, Agios Pharmaceuticals, Ionis Pharmaceuticals, Vifor Pharma, Orchard Therapeutics, Sangamo Therapeutics, CRISPR Therapeutics, DisperSol Technologies, Kiadis Pharma, Incyte Corporation, Editas Medicine, Global Blood Therapeutics, and Syros Pharmaceuticals.

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