LAS VEGAS, Feb. 8, 2021
The modest increase in the Beta-Thalassemia market size can be attributed to a combination of factors such as an overall increase in prevalence, better diagnosis, enhanced R&D, and emerging therapies
LAS VEGAS, Feb. 8, 2021 /PRNewswire/ -- DelveInsight's Beta-Thalassemia Market report offers a holistic picture of the market landscape of the Beta Thalassemia key companies, emerging pipeline therapies, Beta-Thalassemia market share occupied by individual therapies, current and forecasted Beta-thal market share in the 6MM (the US, and EU5 (the UK, Germany, France, Italy, and Spain) for the study period.
The report highlights the drivers and constraints shaping the present Beta-Thalassemia Market along with the unmet medical needs that offer opportunities to the key players to explore the underlying potential of the market.
Some of the key highlights from the Beta-Thalassemia Market Report:
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Beta-Thalassemia is a rare, genetic disorder, which is inherited in an autosomal, recessive fashion. The primary hallmark of the condition is a lack of haemoglobin in RBCs due to mutations in the HBB gene on chromosome 11. The severity of Beta-Thalassemia varies based on the nature of the mutation and the presence of mutations in one or both alleles.
Clinically, there can be two main categories of Beta-Thalassemia patients based on the patient's need for blood transfusion—transfusion-dependent thalassemia (TDT) and non-transfusion dependent thalassemia (NTDT).
Beta-Thalassemia Epidemiological Analysis
According to DelveInsight, the total Beta-Thalassemia prevalent population in the 6MM was 14,954 in 2020. Italy accounted for the highest prevalent Beta-Thalassemia patient pool, followed by the US with Spain reporting the least size of the Beta-Thalassemia prevalent patient pool.
DelveInsight's Beta-Thalassemia Market Report puts forward historical as well as forecasted epidemiological analysis for the 6MM for the study period 2017-30 segmented into:
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Beta-Thalassemia Treatment Market Outlook
Beta Thalassemia is a genetic disorder that is inherited in an autosomal, recessive fashion. The clinical manifestations seen in patients are severe anemia, abnormal hemoglobin, and build-up of iron in the body. The treatment market mainly focuses on delaying the progression of the disease and relieving the symptoms.
Blood transfusion and iron chelation remain the gold-standard of care for patients with TDT, with the former suppressing ineffective hematopoiesis and its complications, and the latter treating and preventing complications from iron overload. The treatment aims to maintain the levels of haemoglobin of 9-10.5 g/dL. However, excess accrual of iron due to frequent blood transfusions is a common complication in patients. Therefore, the present Beta-Thalassemia therapeutic market is dominated by iron binders/chelators, which helps in maintaining iron balance in the body by excreting excess iron through urine or faeces.
Desferal (deferoxamine mesylate USP) is an iron-chelating agent of Novartis and is available in vials for intramuscular, subcutaneous, and intravenous administration. It is indicated for the treatment of acute iron intoxication and chronic iron overload due to transfusion-dependent anaemia. Novartis also Exjade (Deferasirox), another oral iron chelator, which got FDA nod in 2005. It primarily helps in reducing chronic iron overload in patients who are receiving long-term blood transfusions for conditions such as beta-thalassemia and other chronic anaemias. Recently, Luspatercept (ACE-536), a candidate of Acceleron received an FDA nod for the treatment of anaemia in adult patients with beta-thalassemia who require regular red blood cell transfusions. Moreover, in May 2020, the Committee for Medicinal Products for Human Use (CHMP) has also recommended approval of Luspatercept to treat anaemia in both the rare blood disorder beta-thalassemia and haematological cancer myelodysplastic syndrome (MDS).
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Beta-Thalassemia Marketed Therapies
However, these drugs are mainly curative in nature. Potential curative treatment options present in the Beta-Thalassemia market include bone marrow transplant in selected patients and gene therapies that aim to partially, if not fully, correct the disorder.
Several emerging therapies including LentiGlobin, PTG-300, ACE-011, Mitapivat, and others are on the horizon anticipated to impact the overall market domain. Among the emerging therapies, PTG-300 is an injectable hepcidin peptide to treat iron overload in patients with Beta-Thalassemia by Protagonist Therapeutics. The drug is expected to be launched within the forecast period [2021-2030] as it has been granted Fast Track designation as well as Orphan Drug designation by FDA and EMA. siRNA targeting Tmprss6, both of which act by increasing the production of endogenous hepcidin, have shown to improve erythropoiesis and anemia in mouse models of Beta-Thalassemia. Ionis Pharmaceuticals, a leader in RNA-targeted drug discovery and development has developed IONIS-TMPRSS6-LRx.
However, the drug which is being labeled as the potential game-changer is LentiGlobin BB305 (Zynteglo) developed by bluebird bio. It is the first-ever gene therapy to be approved for the management of Beta Thalassemia and was given conditional marketing approval by the EU in June 2019 and was first launched in Germany. This will be followed by a large-scale European rollout. Apart from FDA granting LentiGlobin BB305 an Orphan Drug designation, it has also been granted Breakthrough Therapy designation for Beta-Thalassemia. bluebird bio has set a price of 1.6 million euros ($1.8 million) for its gene therapy Zynteglo, which so far has only been approved for a rare blood disorder in the European Union. The cost will be spread over five years based on its continued effectiveness, at 315,000 euros a year following an outcome-based payment model. The COVID-19 pandemic has taken its toll by delaying the first commercial treatment using Zynteglo in Germany. The US launch has also been delayed to 2021 due to disagreement with the FDA about providing additional data for the agency's review. Henceforth, the overall regulatory environment and the global crisis are expected to make large inroads on the overall pipeline environment.
Reblozyl (luspatercept-aamt), an activin type II receptor fusion protein developed by Acceleron Pharma/Celgene, on the other hand, became the first drug to receive FDA approval for the treatment of anemia in adults suffering from beta-thalassemia. It has also received a positive CHMP (Committee for Medicinal Products for Human Use) opinion in April this year and is expected to enter the European arena by 2021. Another novel therapy that is expected to make an impact on the treatment landscape is VIT-2763 being developed by Vifor Pharma.
Beta-Thalassemia Pipeline Therapies
Beta-Thalassemia Therapeutic Market is witnessing an increment in the investment, thereby, R&D, with pharmaceutical companies exploring and exploiting novel cutting-edge technologies for the development of drugs. Further, well-designed, randomized controlled trials have contributed to building a robust pipeline that shall propel the Beta-Thalassemia market size growth. Pharma and biotech companies such as Bluebird Bio, Protagonist Therapeutics, Acceleron Pharma, Agios Pharmaceuticals, Ionis Pharmaceuticals among several others are working to optimize available treatment approaches as well as bringing out novel therapies to address the unmet needs. Moreover, the market space is booming with the major collaborations, tie-ups, and licensing agreements taking place.
However, the already availability of gene therapies is expected to give a strong competition to the Beta-Thalassemia emerging therapies. Besides tough competition, the timelines of ongoing clinical trials have faced a major backlash, and owing to the uncertainty that looms, it is still hard to predict the normalcy.
Nevertheless, the Beta-Thalassemia Market Outlook lacks a curative approach despite the stronghold of gene therapies in the market, which DelveInsight estimates to be a significant driving force behind the market size growth. An increasing trend in the prevalence of the disease also appears to add to the patient pool thus the demand for curative approaches. In a nutshell, it can be summarized that as many potential therapies are being investigated for the management of Beta-Thalassemia, it is safe to predict that the treatment space will experience significant reconstitution during the forecast period of 2021-2030.
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Table of Contents
Executive Summary of Beta-thalassemia
SWOT Analysis for Beta-Thalassemia
Beta-Thalassemia Market Overview at a Glance
Disease Background and Overview
Beta-Thalassemia Epidemiology and Patient Population
Country-Wise Beta-Thalassemia Epidemiology
Beta-Thalassemia Treatment and Management
Beta-Thalassemia Market Unmet Needs
Beta-Thalassemia Marketed Products
Beta-Thalassemia Emerging Therapies
Beta-Thalassemia Promising Candidates
Beta-Thalassemia Therapies in the News
Beta-Thalassemia Discontinued Therapies
Beta-Thalassemia: Six Major Market Analysis
Beta-Thalassemia Market Outlook by Country
Beta-Thalassemia Market Access and Reimbursement Landscape
Beta-Thalassemia Market Drivers
Beta-Thalassemia Market Barriers
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Scope of the Report
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