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

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

PR Newswire

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.

DelveInsight Logo

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: 

Request for Sample @ Beta-Thalassemia Market Outlook and Forecast

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:

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).

Got Queries? Contact @ Beta-Thalassemia Marketed Therapies and Treatment Approaches and get a comprehensive understanding of the Beta-Thalassemia therapeutic outlook

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. 

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 

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 

1

Key Insights

2

Executive Summary of Beta-thalassemia

3

SWOT Analysis for Beta-Thalassemia

4

Beta-Thalassemia Market Overview at a Glance

5

Disease Background and Overview

6

Beta-Thalassemia Epidemiology and Patient Population

7

Country-Wise Beta-Thalassemia Epidemiology

8

Beta-Thalassemia Treatment and Management

9

Case Study

10

Beta-Thalassemia Market Unmet Needs

11

Beta-Thalassemia Marketed Products

12

Beta-Thalassemia Emerging Therapies

13

Beta-Thalassemia Promising Candidates

14

Beta-Thalassemia Therapies in the News

15

Beta-Thalassemia Discontinued Therapies

16

Beta-Thalassemia: Six Major Market Analysis

17

Beta-Thalassemia Market Outlook by Country

18

Beta-Thalassemia Market Access and Reimbursement Landscape

19

Beta-Thalassemia Market Drivers

20

Beta-Thalassemia Market Barriers

21

Appendix

22

DelveInsight Capabilities

23

Disclaimer

24

About DelveInsight

Know more of what we are offering @ Beta-Thalassemia Market Outlook

Scope of the Report

Related Reports Beta Thalassemia Pipeline Insight

"Beta-thalassemia (B-thal) Pipeline Insight, 2020" report by DelveInsight outlays comprehensive insights of present clinical development scenario and growth prospects across the Beta-thalassemia (B-thal) market.

Beta Thalassemia Epidemiology Forecast

DelveInsight's 'Beta-thalassemia Epidemiology Forecast to 2030' report delivers an in-depth understanding of the disease, historical and forecasted Beta-thalassemia epidemiology in the 6MM, i.e., the United States and EU5 (Germany, Spain, Italy, France, and the United Kingdom).

Thalassemia Market

DelveInsight's "Thalassemia - Market Insights, Epidemiology, and Market Forecast-2030" report delivers an in-depth understanding of the Thalassemia, historical and forecasted epidemiology as well as the Thalassemia market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

Essential Thrombocythemia Market Insights

DelveInsight's 'Essential Thrombocythemia (ET) Market Insights, Epidemiology, and Market Forecast–2030' report deliver an in-depth understanding of the Essential Thrombocythemia (ET), historical and forecasted epidemiology as well as the Essential Thrombocythemia (ET) market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

Insulin Resistance Market

DelveInsight's "Insulin Resistance - Market Insights, Epidemiology, and Market Forecast-2030" report delivers an in-depth understanding of the Insulin Resistance, historical and forecasted epidemiology as well as the Insulin Resistance market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

Hairy Cell Leukemia Market

DelveInsight's "Hairy Cell Leukemia Market Insights, Epidemiology, and Market Forecast-2030" report delivers an in-depth understanding of the Hairy Cell Leukemia (HCL), historical and forecasted epidemiology as well as the Hairy Cell Leukemia (HCL) market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

Acute Lymphoblastic Leukemia Market

DelveInsight's "Acute Lymphoblastic Leukemia Market Insights, Epidemiology, and Market Forecast-2030" report delivers an in-depth understanding of the Acute Lymphoblastic Leukemia (ALL), historical and forecasted epidemiology as well as the Acute Lymphoblastic Leukemia market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

Angelman Syndrome Market Forecast

DelveInsight's 'Angelman Syndrome Market Insights, Epidemiology, and Market Forecast – 2030' report deliver an in-depth understanding of the disease, historical and forecasted epidemiology as well as the market trends of Angelman Syndrome in the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan. 

Congenital Adrenal Hyperplasia Market

DelveInsight's "Congenital Adrenal Hyperplasia - Market Insights, Epidemiology, and Market Forecast-2030" report delivers an in-depth understanding of the Congenital Adrenal Hyperplasia, historical and forecasted epidemiology as well as the Congenital Adrenal Hyperplasia market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

Von Willebrand Disease Market

DelveInsight's "Von Willebrand disease Market Insights, Epidemiology, and Market Forecast-2030" report delivers an in-depth understanding of the Von Willebrand disease, historical and forecasted epidemiology as well as the Von Willebrand disease market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

About DelveInsight

DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing end to end comprehensive solutions to improve their performance.  Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve.

Contact Us
Shruti Thakur 
info@delveinsight.com  
+1(919)321-6187  
www.delveinsight.com 

Logo: https://mma.prnewswire.com/media/1082265/DelveInsight_Logo.jpg

Voltar noticias em Inglês