Thalassemia Treatment Market Analysis

Thalassemia Treatment Market Overview

The Thalassemia Treatment Market Size was valued at USD 2.85 Billion in 2023. The Global Thalassemia Treatment industry is projected to grow from USD 3 Billion in 2024 to USD 4.46 Billion by 2032, exhibiting a compound annual growth rate (CAGR) of 5.23% during the forecast period (2024 - 2032). Thalassemia, specifically the Beta- thalassemia disorder, is majorly caused by the mutation in the Hemoglobin Subunit Beta (HBB) gene. For instance, at least 5.2% of the world population and over 7% of pregnant women have a genetic mutation.

In May 2024, Although cystic fibrosis has traditionally been the main source of income for Vertex Pharmaceuticals, the firm has been actively seeking to expand its revenue streams in recent years. Vertex is making significant progress towards achieving this aim as multiple patients begin their treatment using the company's CRISPR-partnered gene-editing cell therapy, Casgevy. After receiving clearance for sickle cell disease (SCD) in December and beta thalassemia in the following month, Casgevy has made significant advancements in activating authorized treatment centers, engaging physicians and patients, and holding discussions with payers, according to Vertex. In addition to the activation of treatment centers, Vertex also discussed the significant launch component of cell collection during its first-quarter earnings call. Vertex's Casgevy collection surpasses its SCD competitor Lyfgenia from bluebird bio in terms of statistics, despite both drugs receiving FDA approval in December. Bluebird revealed that it had only gathered cells from one patient for its SCD gene treatment.

Thalassemia is an inherited blood disorder wherein the body makes an inadequate amount of hemoglobin. Hemoglobin disorders were initially prevalent in 60% of 229 countries, that potentially affected 75% of births, but are now sufficiently common in 71% of countries amongst 89% of births.

Thalassemia Treatment Market Trends

Major companies are concentrating on mergers and acquisitions, new product developments, approvals, and regional expansions. For instance, in September 2019, Bluebird Bio, Inc. (US) has approved the first gene therapy for Beta-thalassemia. This gene therapy eliminates the need for blood transfusions in patients with β-thalassemia. Thus, major manufacturers are focusing on approvals in thalassemia treatment, which ultimately drives the growth of the Thalassemia Treatment Market.

Market Drivers

• The rising incidence of thalassemia is expected to drive the growth of the market. Patients with insufficient hemoglobin suffer from thalassemia. For instance, thalassemia affects approximately 4.4 of every 10,000 live births throughout the world.

• Increasing pipeline drugs in thalassemia is expected to drive the growth of the market. There are approximately 15 pipeline products under clinical trials for the treatment of thalassemia. For instance, in December 2016, Kiadis Pharma announced initiation of Phase I/II clinical trial with ATIR201 for thalassemia.

• A rise in awareness regarding available treatment options for the treatment of thalassemia is expected to drive the growth of the Thalassemia Treatment Market.

• The rise in demand for improved curative gene therapy is expected to drive the growth of the market.

Market Restraints

• Stringent regulatory policies


• High treatment costs

Thalassemia Treatment Market Segment Insights

Thalassemia Treatment Type Insights

• Alpha-thalassemia: Fastest growing segment. It is one of the most common autosomal recessive disorders. There is an increased incidence of the alpha-globin disorders in the US and other Western nations which eventually drives the growth of the thalassemia treatment market. However, about 5% of the world’s population is affected by alpha-thalassemia.

• Beta Thalassemia: It is caused by reduced or no production of beta-globin subunits. About 1.5% of the global population is affected by Beta-thalassemia.

Thalassemia Treatment Treatment Insights

• Blood Transfusions: The largest growing segment owing to the low risks involved due to careful blood screening. This treatment gives strong red blood cells along with normal hemoglobin. About 56,000 population have major thalassemia, wherein, at least 30,000 need regular blood transfusions. Thus, rising demand for blood transfusions is expected to drive the growth of the market.

• Iron Chelation Therapy: This includes removing excess iron from the bloodstream. Iron chelator drugs such as desferrioxamine, deferiprone, and deferasirox are majorly used in this treatment.

• Folic Acid Supplements: Folic acid is vitamin B that helps build strong red blood cells. These supplements are majorly consumed during and after pregnancy. However, 4% had started consuming after becoming pregnant. Thus, the increased use of folic acid is expected to drive the growth of this segment.

• Gene therapy: Scientists are investigating genetic techniques to treat thalassemia. Gene therapy named Zynteglo was first launched by Bluebird Bio, Inc. (US) in September 2019.

• Bone Marrow transplants: These transplants are not done very often as significant risks are involved but can be tried if no bone marrow matched donor is available. However, the patient will not need further blood transfusions if this transplantation is done. As a result, it would drive the Thalassemia Treatment Market growth in this segment.

Thalassemia Treatment End-User Insights

• Hospitals & Clinics: Holds the largest market share owing to the increase in the number of hospital admissions for the treatment of thalassemia. Moreover, increasing occurrence of thalassemia is also expected to drive this segment.

• Diagnostic laboratories: This segment holds significant market share owing to the increasing awareness regarding thalassemia treatment.

• Others: This segment includes research laboratories, educational institutes, among others.

Thalassemia Treatment Regional Insights

• Americas: The largest growing regional Thalassemia Treatment Market owing to rising awareness about thalassemia and increasing government efforts to support gene therapy. However, Beta-thalassemia disorder is relatively rare in the US.


• Europe: The introduction of gene therapy for the treatment of thalassemia is expected to boost the growth of market. For instance, in September 2019, Bluebird Bio, Inc. (US) has obtained approval for gene therapy that would remove the need for blood transfusions for people with blood disorder beta-thalassemia.


• Asia-Pacific: The fastest-growing regional market in thalassemia treatment. Increasing investment in healthcare infrastructure is expected to drive the growth of the market in this region.


• Middle East & Africa: Increasing adoption rate of blood transfusion treatment in countries such as Turkey and GCC would drive the demand for thalassemia treatment in this region.

• Novartis AG (Switzerland)


• Bluebird Bio, Inc. (US)


• Kiadis Pharma (Netherlands)


• CELGENE CORPORATION (US)


• Sangamo Therapeutics (US)


• Acceleron Pharma, Inc. (US)


• Gamida Cell (Israel)