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(Medical-NewsWire.com, June 30, 2020 ) Market Overview
• The Global Thalassemia Treatment Market is expected to grow at a CAGR of 10.5% during the forecasting period (2020-2027).
• Thalassemia is an inherited blood disorder that reduces the production of functional hemoglobin, the protein in red blood cells that carries oxygen. This causes a shortage of red blood cells and low levels of oxygen in the bloodstream, leading to a variety of health problems. Signs and symptoms vary but may include mild to severe anemia, paleness, fatigue, yellow discoloration of skin, and bone problems. When there are not enough healthy red blood cells, there is also not enough oxygen delivered to all the other cells of the body, which may cause a person to feel tired, weak or short of breath. This is a condition called anemia. People with thalassemia may have mild or severe anemia. Severe anemia can damage organs and lead to death.
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• The thalassemia treatment market growth is driven by several factors, such as an increase in the prevalence of thalassemia, a rise in global awareness and, increasing investment in research and development.
• Inherited hemoglobin disorders (sickle-cell disorders and thalassemias) were initially characteristic of the tropics and subtropics but are now common worldwide due to migration. According to the World Health Organization (WHO), thalassemia is sufficiently common in 71% of countries of the world and among 89% of births. At least 5.2% of the world’s population and over 7% of pregnant women carry a significant variant. Moreover, at least 20% of the world population carries α+ thalassemia. Around 1.1% of couples worldwide are at risk of having children with thalassemia, and 2.7 per 1000 conceptions are affected. Annually there are over 56,000 births that have a major thalassemia, including at least 30,000 who need regular transfusions to survive and 5,500 who die perinatally due to α thalassemia major. Increasing cases of thalassemia require a universal cure for the treatment of the disease, hence boosting the market growth globally.
• An increase in awareness about thalassemia is very important for the prevention of the disease. Many organizations are working towards this cause by initiating awareness campaigns. Governments of countries are also supporting the cause by investing in these programs. For instance, International Thalassaemia Day is celebrated on May 8 every year to raise awareness amongst people concerned with decision and policymaking, health care professionals, patients, and their families and community at large about thalassemia. A vast majority of the population is mostly unaware of the disease and especially those areas where it is more prevalent such as Asia Pacific and Mediterranean. Center for Disease Control and Prevention (CDC) funds one project that monitors blood safety in people with blood disorders. This project is called “Characterizing the Complications Associated with Therapeutic Blood Transfusions for Hemoglobinopathies.” Georgia State University, the University of Florida, and the University of California at San Francisco Benioff Children’s Hospital Oakland have been awarded funding to look at transfusion-related complications in people with hemoglobin disorders (sickle cell disease and thalassemia) and develop approaches for reducing these complications. Besides, CDC funds the Association of Public Health Laboratories to provide public health technical assistance with screening (a test to look for a disease before it is noticeable) activities, including needs assessments for laboratories, as well as education for patients, caregivers, and healthcare workers on hemoglobinopathy screening programs.
Market Segmentation Analysis
• By disease type, the global thalassemia treatment market is segmented as alpha-thalassemia and beta-thalassemia. Alpha thalassemia is expected to hold the largest market share over the period of forecast (2020-2027) owning to higher prevalence of the disease globally. According to National Organization for Rare Disorders, alpha thalassemia occurs in 4-20 individuals per every 1,000 births. Alpha thalassemia is found in most populations worldwide but is most common in the Middle East, Southeast Asia, and certain Mediterranean countries. Doctors in Europe and North America are also increasingly encountering patients with alpha-thalassemia due to increase in migration. Alpha-thalassemia is an inherited disease caused by mutations in a gene required for making a component of hemoglobin. Those mutations either prevent or reduce the production of hemoglobin, which can cause a shortage of mature red blood cells and lead to anemia. The mutations that cause alpha-thalassemia are more common in Asia, India, the Middle East and the Mediterranean where they are found in up to 20% of the population. But the increase in modern migration means that cases are now cropping up more often in other regions. Southern Mediterranean countries recognize the rise in patients with alpha-thalassemia and have increased resources to meet the growing demand appropriately, hence increasing the market for alpha thalassemia treatment globally.
• By treatment type, the global market is segmented into blood transfusion, iron chelation, bone marrow transplant, gene therapy, and surgery. Blood transfusion is expected to be the largest market holder over the period of forecast (2020-2027).
Market Geographical Analysis
• North America is dominating the thalassemia treatment market in 2018 and estimated to hold the largest market size over the forecast period (2020-2027), owing to an increase in thalassemia incidences, highly aware population, advanced healthcare infrastructure and, favorable government initiatives. The cases of thalassemia disorder are increasing in the region due to an increase in migration. Thalassemia, once a rarity in the US, is increasingly encountered in clinical practice due to shifts in immigration. Thalassemia is becoming more prevalent in the US due to immigration of people from affected regions, with an approximately 7.5% increase over the last five decades. According to National Institute of Health, the overall prevalence of Thalassemia is around 5.4% in US.
• Thalassemia is a major public health concern in South Asia, and the situation is expected to deteriorate over the period of forecast (2020-2027); hence Asia Pacific is expected to grow at the highest CAGR over the period of forecast. Most information on thalassemia in South Asia comes from studies conducted in India. Due to extreme heterogeneity, an uneven frequency of thalassemia heterozygote or carrier in the range of 1% and 10% has been reported throughout different parts of India. However, the overall prevalence of thalassemia carriers has been estimated to be between 2.78% and 4% in India. This number translates to approximately 30–48 million beta-thalassemia carriers in India with a carrier rate of 5–7%. Many companies are launching their products in the market targeting the large population. Moreover, government and many other organizations are working in the region to create awareness among the population. Asian Network for Thalassemia Control is one such organization that focus on fact-finding regarding the extent of the problem in individual countries together with an account of the facilities that exist for the diagnosis and management of the different forms of thalassemia in each country. Guangxi, south China, where thalassemia is very common launched a prevention program in 2010. They have set a “one-stop service unit” for thalassemia screening at Marriage Registration Centers (in China, all couples must be registered at these centers). This is where they educate newly married couples about birth control, HIV, and other issues including thalassemia, after which blood is drawn and sent to the hospital for a test.
Market Competitive Trends
• Some of the major key players in the market are Vifor Pharma, Kiadis Pharma, Gamida Cell, ApoPharma, bluebird bio, Inc., Novartis AG, Celgene Corporation, La Jolla Pharmaceutical, Sangamo Biosciences, and Acceleron Pharma.
• In the thalassemia treatment market, bluebird bio Inc. is going to dominate the market, owning to portfolio expansion, mergers, collaborations, and acquisitions. The key players are adopting various growth strategies such as mergers & acquisitions, partnerships, and collaborations and increasing research and development and high investment, which are contributing to the growth of the thalassemia treatment market globally.
• In June 2019, bluebird bio, Inc. announced updated results from the completed Phase 1/2 Northstar (HGB-204) study, and new data from the Phase 3 Northstar-2 (HGB-207) and Phase 3 Northstar-3 (HGB-212) clinical studies of its LentiGlobin® gene therapy for patients with transfusion-dependent β-thalassemia, at the 24th European Hematology Association Congress in Amsterdam, the Netherlands.
• In June 2019, Celgene Corporation and Acceleron Pharma Inc. announced that the U.S. Food and Drug Administration (FDA) accepted Celgene’s Biologics License Application (BLA) for luspatercept, an investigational erythroid maturation agent, for the treatment of adult patients with beta-thalassemia-associated anemia who require RBC transfusions.
• In January 2019, Vifor Pharma is pleased to report positive phase-I trial results for its oral ferroportin inhibitor. Trial subjects received single oral doses of VIT-2763 (ferroportin inhibitor) for the treatment of beta-thalassemia.
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