Number of pages: 100 | Report Format: PDF | Published date: April 19, 2023
Historical Years – 2021 | Base Year – 2022 | Forecasted Years – 2023-2031
According to the deep-dive market assessment study by Growth Plus Reports, the global beta thalassemia was valued at US$ 442.68 million in 2022 and is expected to register a revenue CAGR of 7.5% to reach US$ 848.73 million by 2031.
Beta Thalassemia Market Fundamentals
Beta-thalassemia (β-thalassemia) is described by reduced synthesis of the hemoglobin subunit beta (hemoglobin beta chain) that results in microcytic hypochromic anemia, an abnormal peripheral blood smear with nucleated red blood cells, and decreased amounts of hemoglobin A (HbA) on hemoglobin analysis. People with thalassemia major have severe anemia and hepatosplenomegaly. They generally come to medical consideration within the first two years of life. Without treatment, affected children have severe failure to thrive and shortened life expectancy. Treatment with a regular transfusion program and chelation therapy, aimed at reducing transfusion iron overload, allows for normal growth and development and may improve the overall prognosis.
Beta-thalassemia is diagnosed by measuring red blood cell indices that reveal microcytic hypochromic anemia, nucleated red blood cells on a peripheral blood smear, hemoglobin analysis that reveals decreased amounts of HbA and increased amounts of hemoglobin F (HbF) after the age of 12 months, and the clinical severity of anemia. Identification of biallelic pathogenic variants in HBB (the gene encoding the hemoglobin subunit beta) on molecular genetic testing may be useful for diagnosis in at-risk children under the age of 12 months with a positive or suggestive newborn screening result and unexplained microcytic hypochromic anemia with anisopoikilocytosis and nucleated red blood cells on a peripheral blood smear.
There are three primary types of thalassemia, i.e., thalassemia minor, thalassemia intermedia, and thalassemia major, with symptoms ranging from asymptomatic or moderate to severe anemia requiring lifelong transfusions. Individuals with beta-thalassemia major (homozygous for thalassemia mutations or inheriting two mutations) typically appear with symptomatic severe anemia, poor growth, and skeletal deformities during the first two years of life. Those with beta-thalassemia intermedia are usually present with mild to severe anemia later in life. Beta thalassemia minor can also manifest as beta thalassemia silent carriers, which are people who inherit a beta thalassemic mutation but have no hematologic or symptomatic problems. Some patients with thalassemia are predisposed to splenic problems (hypersplenism) and gallstones (due to hyperbilirubinemia from peripheral hemolysis). These conditions are more common in people with thalassemia major and intermedia.
Although beta thalassemia is rare in the United States, it is one of the most frequent autosomal recessive illnesses globally. The prevalence of symptomatic cases in the general population is estimated to be 1 in 100,000. The condition is most common in the Mediterranean, the Middle East, Africa, Central Asia, the Indian subcontinent, and the Far East. Individuals in other areas of the world with ancestors from these locations are more likely to have beta thalassemia.
Some drugs, including 5-azacytidine, decytabine, and butyrate derivatives, are being researched as possible therapies for beta thalassemia. These medications stimulate fetal hemoglobin production (synthesis), created in the growing fetus and newborn before the body starts producing adult hemoglobin. These medications attach to the extra alpha protein chains, decreasing the imbalance between alpha and beta protein chains. But more study is needed to evaluate these drugs’ long-term safety and efficacy.
Another medication that stimulates the formation of fetal hemoglobin is hydroxyurea. This medication has been explored as a therapy for beta thalassemia intermedia patients to boost hemoglobin levels, reduce the size of extramedullary masses, and alleviate leg ulcers.
Beta Thalassemia Market Dynamics
The rise in the prevalence of thalassemia will function as the primary driver, resulting in the market’s revenue expansion. The growing healthcare spending is a further factor impacting the revenue CAGR of the beta-thalassemia market. Furthermore, advancements in medical technology, rising activities by public and private groups to raise awareness, and increased government financing will all contribute to the growth of the beta-thalassemia market revenue. Factors such as increased demand for effective medications and an aging population will boost the beta-thalassemia market’s revenue growth. Increased disposable money and a changing lifestyle will result in the rise of the beta-thalassemia market. The increased use of early genetic testing and counseling would boost the revenue growth of the beta-thalassemia market.
The increased R&D efforts and the emergence of innovative medicines would give advantageous possibilities for the beta-thalassemia market during the forecast period. Furthermore, the high unmet demand for existing therapy, ongoing clinical studies, and advancements in healthcare technology will accelerate the future beta-thalassemia market’s revenue CAGR. Additionally, government initiatives to improve thalassemia awareness were expected to expand the market revenue during the forecast period. CIL, for instance, signed an MoU with Kokilaben Dhirubhai Ambani in March 2022 for its symbol program “Thalassemia Bal Seva Yojana,” which will support bone marrow transplants for Thalassemia and Aplastic Anaemia patients from low-income families. This yojana provides financial assistance of up to ten lakh rupees to children from low-income households with Thalassemia and Aplastic Anaemia. This, in turn, is likely to substantially influence the revenue growth of the global market during the forecast period.
However, the high cost of therapy and a lack of infrastructure in low-income nations may slow the development rate of the beta-thalassemia industry. Additionally, comorbidities associated with beta-thalassemia, including bone abnormalities, an enlarged spleen, an increased risk of infection, and cardiac difficulties, impede beta-thalassemia market revenue expansion. During the forecast period, less awareness will further challenge the market revenue.
Beta Thalassemia Market Ecosystem
The global beta thalassemia market is analyzed from three perspectives: treatment, indication, and region.
Beta Thalassemia Market by Treatment
The global beta thalassemia market is segmented based on treatment into erythroid maturation agents, iron-chelating drugs, stem cell therapy, and others.
The iron-chelating drugs segment is expected to show significant revenue growth during the forecast period. At present, the usual approach to treating thalassemia is to treat the symptoms rather than seeking to develop a cure. Most treatments for the illness combat iron excess caused by blood transfusions and fetal hemoglobin-raising drugs, vitamin supplements, and indicaxanthin, an antioxidant that prevents hemoglobin breakdown. If the FDA authorizes drugs now in phase II or phase III studies, the market for iron-chelating agents is expected to offer several potentials for corporate growth. The development of novel treatment techniques and a deeper awareness of the various treatment options that are already available are driving market revenue in developed regions such as North America and Europe. Because of the huge number of young people in developing countries such as India and China, there is expected to be an increase in demand for thalassemia drugs and treatments in the future.
Beta Thalassemia Market by Indication
Based on indication, the global beta thalassemia market is segmented into beta thalassemia minor, beta thalassemia intermedia, and beta thalassemia major.
The beta thalassemia minor segment’s revenue is expected to grow faster during the forecast period. People with beta thalassemia minor have low hemoglobin levels, resulting in an insufficient oxygen supply. Those who are affected also have a lack of red blood cells, which is known as anemia in medicine. This deficiency can cause various symptoms, including pale skin, weakness, fatigue, and more serious effects. Beta thalassemia patients are more likely than the general population to have abnormal blood clots.
Beta Thalassemia Market by Region
Based on region, the global beta thalassemia market is segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa.
The existence of significant companies, the region’s greater incidence of thalassemia patients, and the region’s well-established healthcare infrastructure are all crucial factors contributing to the market revenue share. Furthermore, good government initiatives and a rise in research collaborations are expected to boost market revenue expansion. Because of greater awareness of the illness, a growing proportion of persons with the thalassemia carrier gene, and a rise in fertility rates due to genetic heterogeneity, the United States has the highest percentage.
There has been an increase in same-community marriage patterns and a large proportion of alpha-type patients in the Asia Pacific. Asia Pacific is expected to grow greatly in terms of revenue share due to huge variability and an uneven frequency of heterozygotes or carriers. Furthermore, the government and other organizations strive to promote public awareness.
Governments in developing nations are investing heavily in healthcare infrastructure and the life sciences industry to expand access to healthcare. This is expected to increase demand for cutting-edge technology. Governments and non-governmental organizations are also initiating public awareness efforts in impoverished countries to promote public knowledge about the need for early detection. This boosts the attraction of testing for diagnosis and, as a result, opens up massive business opportunities.
Beta Thalassemia Competitive Landscape
The prominent market players operating in the global beta thalassemia market include,
Beta Thalassemia Strategic Development
Beta-thalassemia (β-thalassemia) is described by reduced synthesis of the hemoglobin subunit beta (hemoglobin beta chain) that results in microcytic hypochromic anemia, an abnormal peripheral blood smear with nucleated red blood cells, and decreased amounts of hemoglobin A (HbA) on hemoglobin analysis.
The Asia Pacific is the key growth region in the global beta thalassemia market.
The major driving factors are the rising prevalence of thalassemia, increased R&D efforts, and the emergence of innovative medicines.
The ion-chelating drugs segment will likely dominate the market during the forecast period.
The high treatment costs and lack of infrastructure in countries with low incomes may slow the development pace of the beta-thalassemia market.
*Insights on financial performance are subject to the availability of information in the public domain