DelveInsight Analysts Expect Beta-Thalassemia Market Size to Expand at a CAGR of 16.3% During the Study Period (2017-2030) in the 6MM

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: 

• The Beta-Thalassemia market outlook appears to be ruled by the iron-chelating agents. 
• Exjade is the first, once-daily oral iron chelator commercially available to treat chronic transfusional iron overload due to a wide range of underlying anemia.
• The Beta-Thalassemia market is witnessing an influx of gene therapies and gene-edited cell therapies to eradicate the lifetime burden of the disease.
• Several of the emerging drugs, including LentiGlobin are receiving or have received Orphan Drug Designation. 
• Key pharma players dynamically driving the growth of the Beta-Thalassemia market size are Bluebird Bio, Protagonist Therapeutics, Acceleron Pharma, Agios Pharmaceuticals, Ionis Pharmaceuticals, and others. 
• The upcoming therapies envisaged transforming the Beta-Thalassemia market scenario are ZYNTEGLO (LentiGlobin BB305), Sotatercept (ACE-011), Mitapivat (AG-348), PTG-300, IONIS-TMPRSS6-LRx, VIT-2763, Emeramide, and several others. 
• Rising global prevalence due to increased rates of human migration, the launch of novel and cutting-edge therapies, improvement in diagnostic techniques, the involvement of patients, major healthcare providers, and governmental programs leading to increased awareness, high-priced gene therapies, and an increase in the R&D activities since the cloning of Beta–globin gene is propelling the growth of the Beta-Thalassemia market size.

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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:

• Total Prevalent Cases of Beta-Thalassemia Minor 
• Diagnosed Prevalence of Beta-Thalassemia 
• Diagnosed Beta-Thalassemia Patients by Disease Type 
• Complications of Beta-Thalassemia Patients 

Get a clear picture of Beta-Thalassemia Epidemiology and its Changing trends @ Beta-Thalassemia Prevalence 

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 

• Desferal: Novartis
• Exjade: Novartis
• Ferrirpox (deferiprone): Apotex
• ACE-536: Acceleron Pharma/Celgene Corporation

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. 

Beta-Thalassemia Market Outlook and Forecast 

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 

• LentiGlobin BB305: BlueBird Bio
• PTG-300: Protagonist Therapeutics
• ACE-011 (Sotatercept): Acceleron Pharma/Celgene Corporation
• Mitapivat: Agios Pharmaceuticals
• IONIS TMPRSS6 LRx: Ionis Pharmaceuticals
• VIT 2763: Vifor Pharma
• OTL-300: Orchard Therapeutics
• ST-400: Sangamo Therapeutics/Sanofi
• CTX001: CRISPR Therapeutics/Vertex Pharmaceuticals
• DST-0509: DisperSol Technologies
• ATIR201: Kiadis
• Ruxolitinib: Incyte Corporation/Novartis
• EDIT-301: Editas Medicine
• Global Blood Therapeutics/Syros Pharmaceuticals

And others.

Beta-Thalassemia Market Outlook: Analysts Comments

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.

Know more about the collaborations, tie-ups, and therapies launch @ Beta-Thalassemia Pipeline Therapies and Forecast 

Table of Contents 

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Scope of the Report

• Geography Coverage: 6MM - The United States, and EU5 (Germany, France, Italy, Spain, and the United Kingdom). 
• Study Period: 3-year historical and 11-year forecasted analysis
• Beta-Thalassemia Market Segmentation: Total Prevalent Cases of Beta-thalassemia Minor, Diagnosed Prevalence of Beta-thalassemia, Diagnosed Beta-thalassemia Patients by Disease Type, Complications associated with Beta-thalassemia Patients
• Key Players Involved: Novartis, Apotex, Acceleron Pharma/Celgene Corporation, BlueBird Bio, Protagonist Therapeutics, Acceleron Pharma/Celgene Corporation, Agios Pharmaceuticals, Ionis Pharmaceuticals, Vifor Pharma, EmeraMed, and others.
• Analysis: Comparative and conjoint analysis of Beta-Thalassemia Emerging therapies
• Tools used: SWOT analysis, Porter's Five Forces, PESTLE analysis, BCG Matrix analysis methods.
• Case Studies
• KOL's Views
• Analyst's Views

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