Thalassemia Market

 Market Overview:

The 7 major thalassemia markets reached a value of US$ 2.0 Billion in 2023. Looking forward, IMARC Group expects the 7MM to reach US$ 3.0 Billion by 2034, exhibiting a growth rate (CAGR) of 3.84% during 2024-2034.

The thalassemia market has been comprehensively analyzed in IMARC's new report titled "Thalassemia Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Thalassemia refers to a genetic blood disorder that primarily affects the production of hemoglobin, a protein found in red blood cells responsible for carrying oxygen throughout the body. There are two main types of the ailment: alpha thalassemia and beta thalassemia. The symptoms of the disease can vary depending on the type and severity of the condition. Individuals with thalassemia may experience fatigue, weakness, shortness of breath, pale or yellowish skin, delayed growth and development, an enlarged spleen, etc. In more severe cases, the disorder can cause bone deformities, anemia, and damage to various organs, such as the heart and liver. Thalassemia is diagnosed through a combination of medical history, physical examination, and laboratory tests. A complete blood count (CBC) can reveal low levels of red blood cells and hemoglobin, which are typical indicators of the ailment. Additionally, a hemoglobin electrophoresis test can determine the specific type of thalassemia present. Genetic testing can also be done to confirm the diagnosis and identify carriers of the disease.

The increasing cases of mutations in genes that control the production of hemoglobin in the body are primarily driving the thalassemia market. In addition to this, the rising usage of certain medications, such as erythropoietin and androgens, to stimulate the formation of red blood cells in the bone marrow and alleviate symptoms of anemia in thalassemia patients, is creating a positive outlook for the market. Moreover, the escalating utilization of stem cell transplantation for restoring normal hemoglobin production in the body, thereby eliminating the need for blood transfusions, is also augmenting the market growth. Besides this, the widespread adoption of hemoglobin electrophoresis for diagnosis, since it can determine the quantity of each hemoglobin variant present, which can help to further classify the type and severity of thalassemia, is acting as another significant growth-inducing factor. Additionally, the emerging popularity of various oral iron chelators, including deferoxamine, deferitrin, deferiprone, etc., that preferentially chelate the iron in the heart to lower the risk of thalassemia, is also bolstering the market growth. Furthermore, the inflating utilization of several novel medications, such as hydroxyurea and L-glutamine, which aid in lowering the symptoms of the ailment by increasing the production of fetal hemoglobin, is expected to drive the thalassemia market in the coming years.

IMARC Group's new report provides an exhaustive analysis of the thalassemia market in the United States, EU4 (Germany, Spain, Italy, and France), United Kingdom, and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. According to the report, the United States has the largest patient pool for thalassemia and also represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario, unmet medical needs, etc., have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the thalassemia market in any manner.

Recent Developments:

• In January 2024, Agios Pharmaceuticals announced that the global Phase 3 ENERGIZE study of mitapivat in people with non-transfusion-dependent (NTD) alpha- or beta-thalassemia has met its primary goal of hemoglobin response. Both critical secondary goals linked with change from baseline in FACIT-Fatigue Score and hemoglobin concentration were statistically significant.
• In December 2023, Editas Medicine reported new safety and efficacy results from 17 patients treated with EDIT-301, also known as renizgamglogene autogedtemcel (reni-cel), in the EdiTHAL trial for transfusion-dependent beta thalassemia (TDT) (n=6).
• In November 2023, Vertex Pharmaceuticals and CRISPR Therapeutics disclosed that the UK Medicines and Healthcare Products Regulatory Agency (MHRA) had granted conditional marketing authorization for CASGEVY (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy, for the management of transfusion-dependent beta thalassemia (TDT).

Key Highlights:

• Approximately 5% of the world's population possesses a variant in the alpha or beta part of the hemoglobin molecule, yet not all of them are symptomatic, and some are classified as silent carriers.
• In fact, just 1.7% of the world's population exhibits symptoms caused by gene mutations, often known as thalassemia traits.
• Alpha- and beta-thalassemia are more common in tropical and subtropical regions of the world, particularly where malaria is or has been prevalent.
• In America, 0-5% of the population has a thalassemia trait, whereas up to 40% may carry the gene.
• In the Eastern Mediterranean, 0-2% of the population has thalassemia, with up to 60% of the population possibly carrying the gene.

Drugs:

ZYNTEGLO is a one-time gene therapy that addresses beta-thalassemia (also known as beta-thalassemia major or Cooley's Anemia) in those who need regular transfusions. Beta-thalassemia develops when a mutation in the beta-globin gene leads the body to produce little or no beta-globin. ZYNTEGLO is created exactly for each patient by utilizing the patient's blood stem cells and putting functional copies of the beta-globin gene into them.

CTX001 is an ex vivo CRISPR gene-edited therapy being tested for patients with transfusion-dependent beta thalassemia or severe sickle cell disease. The therapy involves engineering a patient's hematopoietic stem cells to form high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. For individuals with transfusion-dependent beta thalassemia, the rise of HbF by CTX001 may reduce the need for transfusions.

EDIT-301 is an investigational gene editing drug being tested for the treatment of transfusion-dependent beta thalassemia (TDT). EDIT-301 is made up of patient-derived CD34+ hematopoietic stem and progenitor cells that have been edited at the gamma globin gene (HBG1 and HBG2) promoters, which include naturally occurring fetal hemoglobin (HbF) generating mutations, by a highly precise and efficient proprietary manufactured AsCas12a nuclease. Red blood cells formed from EDIT-301 CD34+ cells show a sustained increase in fetal hemoglobin synthesis, indicating a one-time, long-term therapeutic benefit for people with TDT.

Time Period of the Study

• Base Year: 2023
• Historical Period: 2018-2023
• Market Forecast: 2024-2034

Countries Covered

• United States
• Germany
• France
• United Kingdom
• Italy
• Spain
• Japan
   

Analysis Covered Across Each Country

• Historical, current, and future epidemiology scenario
• Historical, current, and future performance of the thalassemia market
• Historical, current, and future performance of various therapeutic categories in the market
• Sales of various drugs across the thalassemia market
• Reimbursement scenario in the market
• In-market and pipeline drugs

Competitive Landscape:

This report also provides a detailed analysis of the current thalassemia marketed drugs and late-stage pipeline drugs.

In-Market Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance
   

Late-Stage Pipeline Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance
   

*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.

Key Questions Answered in this Report:

Market Insights

• How has the thalassemia market performed so far and how will it perform in the coming years?
• What are the markets shares of various therapeutic segments in 2023 and how are they expected to perform till 2034?
• What was the country-wise size of the thalassemia market across the seven major markets in 2023 and what will it look like in 2034?
• What is the growth rate of the thalassemia market across the seven major markets and what will be the expected growth over the next ten years?
• What are the key unmet needs in the market?
   

Epidemiology Insights

• What is the number of prevalent cases (​2018-2034​) of thalassemia across the seven major markets?
• What is the number of prevalent cases (​2018-2034​) of thalassemia by age across the seven major markets?
• What is the number of prevalent cases (​2018-2034​) of thalassemia by gender across the seven major markets?
• What is the number of prevalent cases (​2018-2034​) of thalassemia by type across the seven major markets?
• How many patients are diagnosed (​2018-2034​) with thalassemia across the seven major markets?
• What is the size of the thalassemia patient pool (2018-2023) across the seven major markets?
• What would be the forecasted patient pool (2024-2034) across the seven major markets?
• What are the key factors driving the epidemiological trend of thalassemia?
• What will be the growth rate of patients across the seven major markets?
   

Thalassemia: Current Treatment Scenario, Marketed Drugs and Emerging Therapies

• What are the current marketed drugs and what are their market performance?
• What are the key pipeline drugs and how are they expected to perform in the coming years?
• How safe are the current marketed drugs and what are their efficacies?
• How safe are the late-stage pipeline drugs and what are their efficacies?
• What are the current treatment guidelines for thalassemia drugs across the seven major markets?
• Who are the key companies in the market and what are their market shares?
• What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the thalassemia market?
• What are the key regulatory events related to the thalassemia market?
• What is the structure of clinical trial landscape by status related to the thalassemia market?
• What is the structure of clinical trial landscape by phase related to the thalassemia market?
• What is the structure of clinical trial landscape by route of administration related to the thalassemia market?