As per DelveInsight, there are approx. 22+ key companies which are developing therapies for Beta thalassemia (B-thal). Currently, Imara has its Beta thalassemia (B-thal) drug candidates in the most advanced stage of clinical development.
DelveInsight’s “Beta-thalassemia (B-thal) Market Insights, Epidemiology, and Market Forecast 2032” report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the Beta-thalassemia Market size, share, and trends in the seven major market (7MM) (i.e., the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan).
The Beta-thalassemia market report covers emerging drugs, current treatment practices, market share of the individual therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current treatment practice/algorithm, key drivers & barriers impacting the market growth, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.
Beta-thalassemia (B-thal): An Overview
Beta-thalassemia (β-thal) is a blood-related disorder and occurs when the body cannot make enough of the protein called hemoglobin (present in red blood cells; RBCs). Due to the lack of hemoglobin in RBCs, they cannot function properly and persist for shorter periods. It occurs due to mutations in the HBB gene on chromosome 11, also inherited in an autosomal, recessive fashion.
The thalassemia spectrum is clinically divided into two main categories based on the patient’s need for blood transfusion—transfusion-dependent thalassemia (TDT) and non-transfusion-dependent thalassemia
According to the study by Hickman et al., In England, approximately 2,800 (0.44%) carry beta-thalassemia trait annually, and 43 (0.07/1,000) are affected by beta-thalassemia major/intermedia.
Beta-thalassemia (B-thal) Market Key Facts
Endocrine complications and Osteoporosis was found in the majority of patients with Beta-Thalassemia. As per DelveInsight estimates, the trend would remain the same during the forecast period.
The United States accounts for the largest market size for Beta Thalassemia compared to EU5 (the United Kingdom, Germany, Italy, France, and Spain).
According to analysts at Delveinsight, among EU5 countries, Italy had the highest market size, with USD 77.6 million in 2017, while Spain had the smallest market size for Beta Thalassemia, with USD 2.2 million in 2017.
Beta-thalassemia (B-thal) Market
The market outlook section of the report helps to build a detailed comprehension of the historical, current, and forecasted Beta-thalassemia market size by analyzing the impact of current and emerging pipeline therapies. It also provides a thorough assessment of the market drivers & barriers, unmet needs, and emerging technologies set to impact the market dynamics.
The report gives complete detail of the Beta-thalassemia market trend for each marketed drug and mid & late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, their Mechanism of Action (MoA), Route of Administration (RoA), molecule types, competition with other therapies, brand value, and their impact on the market.
Beta-thalassemia (B-thal) Epidemiology
The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted epidemiology trends by exploring numerous studies and research. The epidemiology section also provides a detailed analysis of diagnosed and prevalent patient pool, future trends, and and views of key opinion leaders.
Beta-thalassemia (B-thal) Drugs Uptake and Pipeline Development Activities
The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Beta-thalassemia market or expected to get launched during the study period. The analysis covers Beta-thalassemia market uptake by drugs, patient uptake by therapies, and sales of each drug.
Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.
The report also covers the Beta-thalassemia Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyses recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.
Key Developments in the Beta-thalassemia (B-thal) Therapeutics Market
Companies across the globe are diligently working toward the development of new treatment therapies for β-thal treatment. Many of them are in the early clinical development phase and are expected to launch in the coming years.
On August 17, 2022, bluebird bio, Inc. (Nasdaq: BLUE) announced that the U.S. Food and Drug Administration (FDA) has approved ZYNTEGLO® to treat the underlying genetic cause of beta‑thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions. ZYNTEGLO® (betibeglogene autotemcel) also known as beti-cel, is a first-in-class, one-time ex-vivo LVV gene therapy. Each dose of Zynteglo is a customized treatment. It is created using the patient’s own cells (bone marrow stem cells) that are genetically modified to produce functional beta-globin (a hemoglobin component).
On May 12, 2022, Editas Medicine, Inc. (Nasdaq: EDIT) announced that the US FDA granted Orphan Drug Designation to EDIT-301 for the treatment of beta-thalassemia. EDIT-301 is an investigational gene editing medicine. The FDA previously granted Rare Pediatric Disease designation to EDIT-301 for treating beta-thalassemia.
In the coming days, the competitive landscape for the Beta-thalassemia (β-thal) market is set to change due to improvements in the diagnosis methodologies, raising awareness of the diseases, and incremental healthcare spending worldwide.
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Majro Players in the Beta-thalassemia (B-thal) Therapeutics Market:
Novartis, Apotex, Celgene Corporation, Bluebird Bio, Protagonist Therapeutics, Acceleron Pharma, Agios Pharmaceuticals, Ionis Pharmaceuticals, Orchard Therapeutics, Sangamo Therapeutics, CRISPR Therapeutics, DisperSol Technologies, Kiadis, Editas Medicine, La Jolla Pharmaceutical Company, Vertex Pharmaceuticals, Sanofi, and many others.
Beta-thalassemia (B-thal) Therapies covered in the report include:
Desferal, Exjade, Ferrirpox (deferiprone), ACE-536, LentiGlobin BB305, PTG-300, ACE-011 (Sotatercept), Mitapivat, IONIS TMPRSS6 LRx, VIT 2763, Emeramide, OTL-300, ST-400, CTX001, DST-0509, ATIR201, Ruxolitinib, LJPC-401, and many more.
Further Emerging Drugs & Key Companies are Covered in the Report. Download the Sample PDF to Learn More:
Table of Content (TOC)
1. Key Insights
2. Executive Summary
3. Beta-thalassemia Competitive Intelligence Analysis
4. Beta-thalassemia Market Overview at a Glance
5. Beta-thalassemia Disease Background and Overview
6. Beta-thalassemia Patient Journey
7. Beta-thalassemia Epidemiology and Patient Population (In US, EU5, and Japan)
8. Beta-thalassemia Treatment Algorithm, Current Treatment, and Medical Practices
9. Beta-thalassemia Unmet Needs
10. Key Endpoints of Beta-thalassemia Treatment
11. Beta-thalassemia Marketed Products
12. Beta-thalassemia Emerging Drugs and Latest Therapeutic Advances
13. Beta-thalassemia Seven Major Market Analysis
14. Attribute Analysis
15. Beta-thalassemia Market Outlook (In US, EU5, and Japan)
16. Beta-thalassemia Access and Reimbursement Overview
17. KOL Views on the Beta-thalassemia Market
18. Beta-thalassemia Market Drivers
19. Beta-thalassemia Market Barriers
20. Appendix
21. DelveInsight Capabilities
22. Disclaimer
*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.
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About DelveInsight
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