Summary

Market Overview
The Global Alpha Thalassemia Market reached US$ 1.1 billion in 2022 and is expected to reach US$ 1.9 billion by 2030 growing with a CAGR of 7.4% during the forecast period 2023-2030. The alpha thalassemia market trends show rising research owing to the rising technological advancements. When one or more of the four alpha-globin genes—which are responsible for producing hemoglobin—are damaged or absent, alpha thalassemia is the result.
Furthermore, rising adoption of iron chelators for alpha thalassemia due to the rising availability of treatment and increase in the number of alpha thalassemia silent carrier cases in the market, and an increase in the research and development is driving up the alpha thalassemia market size.
The market is experiencing a growth in demand for treatments from North American areas as a result of the rising advancements in this field. With significant competitors like Agios Pharmaceuticals, Inc., Actis, Novartis Pharmaceuticals Corporation, and Taro Pharmaceutical Industries Ltd. actively operating in the market.
Market Dynamics
Growing Advancements and Clinical Trials by Key Players Drive the Growth of the Alpha Thalassemia Market
Several clinical trials are taking place currently and resulting in positive outcomes. On November 15, 2022, a study published in American Society of Hematology Adenosine triphosphate (ATP) levels in thalassemic red blood cells (RBCs) are insufficient to meet their elevated energy requirements. Pyruvate kinase (PK), a crucial enzyme that controls the final stage of glycolysis, which is responsible for producing ATP in RBCs, is activated allosterically by the drug mitapivat.
Earlier, the main findings of an open-label stage 2 study (NCT03692052) of mitapivat in patients with - or -non-transfusion-dependent thalassemia (NTDT) showed that 80.0% of the patients had a reaction in hemoglobin (Hb) of 1.0 g/dL rise from baseline at 1 assessments in between 4–12 weeks (Wk), inclusive. Hemolysis markers also improved, and Hb levels continued to rise, with a mean Hb rising to 1.7 g/dL (0.5) at Wk 72. The market is predicted to take the lead throughout the forecast period as a consequence of the aforementioned claims.
Rising Collaboration by Key Players for Research and Development Creates Opportunities for the Growth of the Market
As a result of years of study, a number of major manufacturers are now developing novel products and releasing them for therapeutic purposes, which is fostering the expansion of the alpha thalassemia market. Several treatments have been approved by the regulatory authorities for management of alpha thalassemia and major players are collaborating for development of products for rare diseases including thalassemia.
For instance, on January 8, 2023, a commercial-stage biopharmaceutical company, Amryt Pharma Plc, committed to acquiring, creating, and marketing novel treatments for rare diseases, and Chiesi Farmaceutici S.p.A., a research-focused healthcare and biopharmaceuticals group, announced that the two companies have come to terms to an official agreement pursuant to which Chiesi will purchase Amryt.
Unavailability of Proper Treatment for Alpha Thalassemia will Hamper the Growth of the Market
There is not a reliable treatment for alpha thalassemia currently on the market. Alpha thalassemia cannot be cured by chelation or blood transfusions. A stem cell transplant can cure certain alpha thalassemia major patients. The technique of a stem cell transplant is serious and fraught with danger.
Other therapies are being developed by medical professionals and researchers to assist those who have alpha thalassemia. The only treatment for thalassemia is a stem cell transplant. Only a small percentage of those with severe thalassemia can locate a donor match and be a good candidate for the operation.
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Segment Analysis
The global alpha thalassemia market is segmented based type, treatment, end user and region.
Owing to High Utilization, the Iron Chelators from Treatment Segment Accounted for Approximately 40.1% of the Alpha Thalassemia Market Share
The iron chelators category from treatment held the largest market share in 2022 and is expected to maintain its dominance over the forecast period due to a growth in alpha thalassemia types worldwide. Iron overload in thalassemias is primarily brought on by two mechanisms: blood transfusions and increased iron absorption as a result of inefficient erythropoiesis. Atypical dietary iron absorption causes severe thalassemia patients who are not receiving blood transfusions to have an annual rise in body iron burden of 2 to 5 g.
Blood transfusion recipients are more likely to develop iron excess. Iron from all the transfusions can accumulate in the body over time since red blood cells have a lot of iron in them. The accumulation of iron can make it difficult for several organs to function effectively, including the heart, liver, and brain. People having thalassemia may require chelation therapy, in which case doctors provide a medication - either a pill or an injection under the skin - to remove surplus iron as it builds up in the organs, to prevent iron overload.
Due to the widespread use of iron chelation therapy in medical centers during the past few decades, the survival rates of patients with iron excess have significantly increased. The three iron chelators that the United States Food and Drug Administration has approved for clinical use are deferoxamine, deferiprone, and deferasirox. It is generally recognized that these iron chelators can all be used to treat iron overload in a variety of clinical circumstances.
Geographical Penetration
North America Accounted for Approximately 44.7% of the Market Share in 2022, Owing to the Strong Presence of Major Players and Increasing Healthcare Infrastructure Investment
Due to the rising need for alpha thalassemia treatment in healthcare, manufacturers in North America have chances of increasing their operations. There are many producers and suppliers in North America and owing to the quick economic growth of the region, industrial production has expanded, driving the demand for iron chelators for treatment.
Increasing expenditure on healthcare and rising adoption among patients, advancement of technologies for tests or treatment, and increase in pharmaceutical business establishment across the region are also contributing to the growth of alpha thalassemia market share of this region.
It is also anticipated that the main healthcare organizations and enterprises collaborative research projects as well as new product development, which constantly seeks to improve available alternatives, will contribute to the expanding demand. Individuals are becoming more aware of various types of tests for alpha thalaessemia and their treatment that are being utilized for management purposes, leading to the expansion of the market in this region. The above-mentioned factors further proves the dominance of North America on a global scale.
Competitive Landscape
The major global players in the alpha thalassemia market include Agios Pharmaceuticals, Inc., Actis, Novartis Pharmaceuticals Corporation, Global Calcium PVT LTD, CHIESI Farmaceutici S.p.A., Taro Pharmaceutical Industries Ltd., Amgen Inc., Sun Pharmaceutical Industries Ltd., FRESENIUS SE & Co. KGaA and Acceleron Pharma among others.
COVID-19 Impact Analysis
Russia Ukraine Conflict Analysis
Due to the low prevalence as well as absence of major market participants in this region, it is predicted that the Russia-Ukraine conflict will not have a significant effect on the worldwide alpha thalassemia market. The growth of the global alpha thalassemia market however, is anticipated to be relatively unaffected by the import and export of raw materials over the forecast period.
By Type
• Alpha Thalassemia Silent Carrier
• Alpha Thalassemia Carrier
• Hemoglobin H Disease
• Alpha Thalassemia Major
By Treatment
• Folic Acid Supplement
• Iron Chelators
o Deferoxamine (Desferal)
o Deferasirox
 Exjade
 Jadenu
o Deferiprone or L1 (Ferriprox)
• Blood Transfusion
• Stem Cell Transplant
By End User
• Hospitals
• Ambulatory Surgical Centers
• Others
By Region
• North America
o U.S.
o Canada
o Mexico
• Europe
o Germany
o U.K.
o France
o Spain
o Italy
o Rest of Europe
• South America
o Brazil
o Argentina
o Rest of South America
• Asia-Pacific
o China
o India
o Japan
o Australia
o Rest of Asia-Pacific
• Middle East and Africa
Key Developments
• On March 31, 2023, FERRIPROX MR deferiprone extended-release tablets, 1000 mg, have been approved by Health Canada for the treatment of individuals with transfusional iron overload resulting from thalassemia syndromes if current chelation therapy is insufficient, or sickle cell disease (SCD), or other anemias, according to Chiesi Global Rare Diseases, a subsidiary of the Chiesi Group created to deliver novel treatments and solutions to individuals affected by rare diseases.
• On August 11, 2022, data from the core time of the open-label, stage 2 investigation regarding PYRUKYND (mitapivat) in individuals having non-transfusion-dependent - or -thalassemia was released on August 11, 2022, in The Lancet, according to a release from Agios Pharmaceuticals, Inc., an innovator in the field of cellular metabolism and treatments for genetically defined diseases. The 2021 Annual Congress of the European Hematology Association (EHA) featured an earlier presentation of study data. PYRUKYND is an experimental, oral, first-in-class small molecule wild-type allosteric activator and other PK enzyme mutations.
• On April 23, 2021, through its affiliate Novadoz, the Indian company MSN Labs has introduced four recently-approved generic drugs into the US market: albendazole, droxidopa, deferasirox, and pregabalin. For the upcoming ten years, the corporation claims to have a "deep pipeline" of launches planned.
Why Purchase the Report?
• To visualize the global alpha thalassemia market segmentation based on type, treatment, end user and region as well as understand key commercial assets and players.
• Identify commercial opportunities by analyzing trends and co-development.
• Excel data sheet with numerous data points of alpha thalassemia market-level with all segments.
• PDF report consists of a comprehensive analysis after exhaustive qualitative interviews and an in-depth study.
• Product mapping available as excel consisting of key products of all the major players.
The global alpha thalassemia market report would provide approximately 53 tables, 54 figures and 195 Pages.
Target Audience 2023
• Manufacturers/ Buyers
• Industry Investors/Investment Bankers
• Research Professionals
• Emerging Companies

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