Thalassemia Treatment Market Expected to Reach USD 1.5 billion by 2031, with a Projected CAGR of 7.3%: Transparency Market Research, Inc.

Stem cell therapy is gaining traction for the treatment of thalassemia, especially for severe cases. Gene-edited cell therapy is also gaining popularity for the treatment of transfusion-dependent beta-thalassemia (TDT) in patients 12 years and older.

Wilmington, Delaware, United States, Feb. 20, 2024 (GLOBE NEWSWIRE) -- Transparency Market Research Inc. - The global thalassemia treatment market is slated to be valued at US$ 1.5 billion in 2031, with an expected value CAGR of 7.3%. Thalassemia is one of the inherited blood disorders in which the body produces an inadequate quantity of hemoglobin. Blood transfusion solutions and chelation therapy are among the common treatments.

Chelation therapy makes use of drugs, such as deferasirox, for the treatment of chronic iron overloads that occur during frequent blood transfusions amongst non-transfusion-dependent thalassemia patients. They are administered subcutaneously or intravenously with slow infusion to purify the blood. High treatment costs may compel the population in under-developed economies to give a second thought about resorting to thalassemia treatment. This factor may restrain the thalassemia treatment market development.

According to Boston Children’s Hospital, nearly 300 million individuals across the globe suffer from thalassemia trait. These individuals are at a higher risk of begetting children with thalassemia. The hospital further states that more than 100 thousand people are diagnosed with transfusion-dependent thalassemia.

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Fetal hemoglobin inducers are recommended, such that decitabine and hydroxyurea can be successfully induced to elevate levels of fetal hemoglobin. Rise in need to reduce the number of thalassemia-infected patients (to prevent children from getting affected) is prompting extensive research pertaining to thalassemia treatment. This, in turn, is augmenting the thalassemia treatment market demand. Various studies are focusing on devising erythropoiesis-stimulating agents to treat millions of adults contracting thalassemia due to kidney failure.

Key Takeaways from the Market Study

  • From 2024 to 2031, the market for thalassemia treatment is slated to expand 1.6x.
  • As of 2024, the thalassemia treatment market is expected to attain a valuation of US$ 920 million.
  • By treatment type, blood transfusion is slated to be the most preferred approach to address thalassemia.
  • Players in the thalassemia treatment market landscape are broadening their focus toward the treatment of alpha-thalassemia and beta-thalassemia. They are developing advanced thalassemia treatment strategies to expand their product portfolio.

Prominent Market Drivers of the Thalassemia Treatment Market

  • Beta-thalassemia is prevalent in the Middle East, Mediterranean, South Asia, Africa, and India. It is conventionally managed through red blood cell transfusions at periodic intervals, usually every two to four weeks, followed by chelation therapy with deferoxamine (Novartis; Desferal) or deferasirox for removing excess iron from the bloodstream.
  • Emerging treatments like gene therapy aim to address the genetic mutations causing thalassemia. Gene therapy for thalassemia is still under research and development. Stem cell therapy, particularly hematopoietic stem cell transplantation (HSCT), is one of the primary and potentially curative treatments for thalassemia, especially for severe cases.
  • In 2019, the U.S. FDA approved luspatrcept-aamt (Reblozyl) for the treatment of thalassemia. Betibeglogene autotemcel was approved by the FDA in August 2022 and is the first ex-vivo lentiviral vector gene therapy available in the U.S. for the treatment of people with β-thalassemia.
  • R&D of stem cell-based gene therapies is driving the thalassemia treatment market share. In 2019, Vifor Pharma reported positive phase-I trial results for its oral ferroportin inhibitor. In all treated trial participants, the ferroportin inhibitor was well tolerated with mild to moderate side effects that were transient and self-limiting.

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Regional Profile of the Thalassemia Treatment Market

  • According to the latest thalassemia treatment market forecast, North America is expected to hold largest share from 2023 to 2031. The region accounted for major share in 2022. Rise in awareness regarding thalassemia coupled with governments rendering support to gene therapy is fueling the market dynamics of the region.
  • The thalassemia treatment market growth in Europe and Asia Pacific can be ascribed to high demand for gene therapy for thalassemia treatment and surge in investment in healthcare infrastructure.
  • Increase in awareness regarding thalassemia is also boosting the market statistics in these regions. Healthcare experts state that marriages amongst cousins are likely to cause thalassemia in the progeny, especially in Asia Pacific.

Competitive Landscape

Key players are engaging in extensive R&D activities to expand their product portfolio. Bluebird Bio, Inc., Merck & Co., Inc., Novartis AG, Celgene Corporation, Takeda Pharmaceutical Company Limited, Incyte Corporation, Sanofi, Bellicum Pharmaceuticals, and GSK plc are some of the key players in the thalassemia treatment industry.

Key Developments

  • In January 2024, CRISPR Therapeutics 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
  • In November 2023, The Medicines and Healthcare Regulatory Agency in the U.K. approved Casgevy for patients with sickle cell disease and thalassemia who are 12 years old and over.

Key Segments Covered

Treatment Type

  • Blood Transfusion
  • Chelation Therapy
  • Bone Marrow Transplantation
  • Others


  • North America
  • Europe
  • Asia Pacific
  • Latin America
  • Middle East & Africa

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