Beta-thalassemia (B-thal) - Market Insight, Epidemiology And Market Forecast - 2032

The ‘Beta Thalassemia-Market Insights, Epidemiology, and Market Forecast-2032’ report deliver an in-depth understanding of Beta Thalassemia, historical and forecasted epidemiology as well as the market trends in the United States and EU5 (Germany, Spain, Italy, France, and United Kingdom).

The Beta Thalassemia market report provides current treatment practices, emerging drugs, and market share of the individual therapies, current and forecasted Beta Thalassemia symptoms market size from 2019 to 2032 segmented by seven major markets. The report also covers current Beta Thalassemia symptoms treatment practice/algorithm and unmet medical needs to curate the best opportunities. It assesses the underlying potential of the market.

Geography Covered

• The United States
• EU5 (Germany, France, Italy, Spain, and the United Kingdom)

Study Period: 2019-2032

Beta Thalassemia Understanding and Treatment Algorithm

Beta Thalassemia Overview

Beta thalassemia (β-Thalassemia) is a blood disorder characterized by reduced synthesis of the hemoglobin subunit beta (hemoglobin beta chain) that results in microcytic hypochromic anemia, an abnormal peripheral blood smear with nucleated red blood cells, and reduced amounts of hemoglobin A (HbA) on hemoglobin analysis.

Beta Thalassemia is an inherited disorder caused due to genetic modification (deletion/alteration) on Chromosome 11. The major risk factors include “Family history of the disorder” and /or “Asian, Chinese, Mediterranean, or African American ethnicity.”

Beta Thalassemia Diagnosis and Treatment

This section covers the details of diagnostic methods and conventional and current medical therapies available in the Beta Thalassemia market to treat the condition. It also provides the country-wise treatment guidelines and algorithms across the United States and Europe.

The Beta Thalassemia market report gives a holistic understanding of Beta Thalassemia clinical manifestations by including disease definition, symptoms, causes, risk factors, physiology, and diagnosis. It also provides Beta Thalassemia treatment algorithms and treatment guidelines for Beta Thalassemia in the US and Europe.

Patients with severe beta thalassemia (major) require lifelong therapy to prevent and manage the clinical consequences of the disease. Long-term adherence to treatment is essential to maintain blood composition level. Comprehensive management guidelines for both TDT (transfusion dependent) and NTDT (Non transfusion dependent) have been developed by the Thalassemia International Federation and are widely available. Current management strategies for TDT include blood transfusion, iron chelation, splenectomy (less common than in the past), and HSCT for a subset of patients.

Beta Thalassemia Epidemiology

The Beta Thalassemia symptoms epidemiology division provides insights into the historical and current patient pool and the forecasted trend for every six major countries. It helps recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of The report also provides the diagnosed patient pool and their trends and assumptions.

Key Findings

The total prevalent cases of Beta Thalassemia patients in the 6MM are increasing during the study period, i.e., 2019-2032.

The disease epidemiology covered in the report provides historical as well as forecasted Beta Thalassemia symptoms epidemiology segmented as the total Prevalent cases of Beta Thalassemia minor, Diagnosed Prevalence of Beta Thalassemia, Diagnosed Beta Thalassemia patients by disease type. The report includes the prevalent cases scenario of Beta Thalassemia minor in 6MM covering the United States and EU5 countries (Germany, France, Italy, Spain, and the United Kingdom) from 2019 to 2032.

Country-wise Beta Thalassemia Epidemiology

The epidemiology segment also provides the Beta Thalassemia epidemiology data and findings across the United States and EU5 (Germany, France, Italy, Spain, and the United Kingdom).

The total number of prevalent cases of Beta Thalassemia-associated minor in 6MM countries was 12,536 in 2020.

Beta Thalassemia Drug Chapters

The Beta Thalassemia report’s drug chapter segment encloses the detailed analysis of Beta Thalassemia early-stage (Phase I, II, and III) pipeline drugs. It also helps understand the Beta Thalassemia clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug, and the latest news and press releases.

Beta Thalassemia Emerging Drugs

Zynteglo (LentiGlobin BB305): Bluebird Bio

LentiGlobin BB305, also known as Zynteglo, is a one-time gene therapy that expands options for patients with transfusion-dependent Beta Thalassemia (TDT) ≥12 years of age who do not have a Beta0/Beta0 genotype and who do not have an HLA-matched related donor. It is used in patients who do not completely lack beta-globin and are eligible for stem cell transplantation but do not have a matching related donor. Its active substance is stem cells taken from patients that have been genetically modified to contain a working gene for beta-globin. Bluebird Bio is manufacturing it. It is a genetically modified autologous CD34+ cell-enriched population that contains cells transduced with LentiGlobin BB305 lentiviral vector encoding human BetaA-T87Q-globin.

Bluebird Bio despite its successful conditional marketing authorization for Beta Thalassemia from European commission in 2019, it has announced to exit German markets, due to reimbursement challenges and is expected to hit the US markets in 2022. This drug is currently in Phase III trial, evaluating the efficacy and safety of gene therapy in subjects with transfusion-dependent beta Thalassemia by transplantation of autologous cd34+ stem cells transduced ex vivo with a lentiviral Betaa-t87q-globin vector in subjects’ ≤50 years of age.

Mitapivat (AG-348): Agios Pharmaceuticals

Mitapivat (AG-348) is an oral, small-molecule, allosteric activator of the RBC-specific form of pyruvate kinase (PK-R). PK-R is a key enzyme for maintaining energy homeostasis in RBCs, as they rely almost exclusively on the process of glycolysis to generate ATP. In healthy adults, mitapivat activates wild-type PK-R and increases ATP levels in RBCs. In preclinical studies, AG-348 reduced ineffective erythropoiesis, extramedullary erythropoiesis, increased Hb levels, reduced reticulocyte count, and circulating erythroblasts, and significantly increased RBC survival. These data support the hypothesis that increased ATP synthesis mediated via PK-R activation by mitapivat may improve the survival of thalassemic RBCs in the bone marrow and/or peripheral circulation.

Beta Thalassemia Market Outlook

The report’s Beta Thalassemia market outlook helps to understand the historic, current better, and forecasted Beta Thalassemia market trends by analyzing the impact of current therapies on the market, unmet needs, SWOT analysis and demand for better technology.

This segment gives a holistic analysis of Beta Thalassemia market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria’s, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. The calculated market data are presented with relevant tables and graphs to view the market at first sight.

According to the publisher, the Beta Thalassemia market in 6MM is expected to grow steadily during the study period 2019-2032.

Blood transfusion and iron chelation are the standard of care for patients with TDT, with the former suppressing ineffective hematopoiesis and their complications and the latter treating and preventing complications from iron overload. The goal of treatment is to maintain a hemoglobin level of 9-10.5 g/dL, which has been shown to promote normal growth, suppress bone marrow activity, and minimize iron accumulation.

Various new therapies are in development, focusing on alleviating the limitations of the currently approved drugs. Some of the most prominent ones include Zynteglo (LentiGlobin BB305; Bluebird Bio), Mitapivat (AG-348; Agios Pharmaceuticals), VIT 2763 (Vifor Pharma), IMR-687 (Imara Inc.), among others. The market is poised to take off with novel candidates like CTX001 (CRISPR/Vertex Pharma) and regulators facilitating drug development with various designations including Breakthrough therapy, PRIME designation, Rare Pediatric indication, etc.,

In addition, market analysis shows immense Go-To-Market challenges such as manufacturing and commercialization strategies, pricing decisions, reimbursement negotiations, making the Beta Thalassemia market, one of the trickiest to navigate through.

ZYNTEGLO was granted conditional marketing authorization by European Commission for the treatment of TDT patients above 12 yrs. of age, in 2019. By 2021, though the company managed the commercial launch of Zynteglo in Germany, it failed to navigate the reimbursement challenges successfully. The company has announced to pull-out of Germany and scale-down operations in EU and focus its resource in the US. Thus it is imperative for upcoming therapies like CTX001 and others to be cautious with pricing decisions and market access strategies for a successful launch, across geographies.

Key Findings

This section includes a glimpse of the Beta Thalassemia market in 6MM.

Beta Thalassemia market size in the six major markets was USD 364.3 million in 2020.

The United States Market Outlook

This section provides the total Beta Thalassemia market size and market size by therapies in the United States.

EU-5 Countries: Market Outlook

The total Beta Thalassemia market size and market size by therapies in Germany, France, Italy, Spain, and the United Kingdom are provided in this section.

Italy accounts for the largest market size of Beta Thalassemia followed by the United States, Germany, France, the United Kingdom and Spain.

Beta Thalassemia Drugs Uptake

This section focuses on the uptake of the potential drugs recently launched or expected to get launched in the market during the study period 2019-2032. The analysis covers the Beta Thalassemia market uptake by drugs, patient uptake by therapies, and sales of each drug.

This helps understand the drugs with the most rapid uptake, reasons behind the maximal use of new drugs, and the comparison of the drugs based on market share and size, which will be useful in investigating factors important in the market uptake and in making financial and regulatory decisions.

Beta Thalassemia Pipeline Development Activities

The report provides insights into the therapeutic candidate in Phase I, II, and III stages. It also analyses Beta Thalassemia key players involved in developing targeted therapeutics.

Major market players include Vifor Pharma, Ionis Pharmaceutical, Agios Pharmaceuticals, and others.

Pipeline Development Activities

The report covers collaborations, acquisition, merger, licensing, and patent details for Beta Thalassemia emerging therapies.

In September 2021, Merck, a publicly-traded biopharmaceutical company, announced that the companies have entered into a definitive agreement under which Merck, through a subsidiary, will acquire Acceleron for USD 180 per share in cash for an approximate total equity value of USD 11.5 billion.

In November 2019, Bristol-Myers Squibb announced that it has completed its acquisition of Celgene Corporation following the receipt of regulatory approval from all government authorities required by the merger agreement and, as announced on April 12, 2019, approval by Bristol-Myers Squibb and Celgene stockholders
In December 2017, Vertex Pharmaceuticals Inc. and CRISPR Therapeutics announced that the companies would co-develop and co-commercialize CTX001 as part of the companies’ previously announced collaboration aimed at discovering and developing new gene-editing treatments that use the CRISPR/Cas9 technology

Reimbursement Scenario in Beta Thalassemia

Proactively approaching reimbursement can positively impact both during the early stages of product development and after product launch. The report considers reimbursement to identify economically attractive indications and market opportunities. When working with finite resources, the ability to select the markets with better market access can be a critical business and price strategy.

KOL Views

To keep up with current market trends, we take KOLs and SMEs’ opinions working in the Beta Thalassemia domain through primary research to fill the data gaps and validate our secondary research. Their opinion helps understand and validate current and emerging therapies treatment patterns or Beta Thalassemia market trends. This will support the clients in potential upcoming novel treatment by identifying the overall scenario of the market and the unmet needs.

Competitive Intelligence Analysis

We perform a Competitive and Market Intelligence analysis of the Beta Thalassemia Market by using various Competitive Intelligence tools: SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies, etc. The inclusion of the analysis entirely depends upon the data availability.

Scope of the Report

• The report covers the descriptive overview of Beta Thalassemia, explaining its causes, signs and symptoms, physiology, and currently available therapies.
• Comprehensive insight has been provided into the Beta Thalassemia epidemiology and treatment in the 6MM.
• Additionally, an all-inclusive account of both the current and emerging therapies for Beta Thalassemia is provided, along with the assessment of new therapies, which will impact the current treatment landscape.
• A detailed review of the Beta Thalassemia market, historical and forecasted, is included in the report, covering drug outreach in the 6MM.
• The report provides an edge while developing business strategies by understanding trends shaping and driving the global Beta Thalassemia market.

Report Highlights

• In the coming years, the Beta Thalassemia market is set to change due to the rising awareness of the disease, development of diagnostic techniques, and the favorable environment from regulators for innovative first-in-class curative drugs that are likely to expand the market’s size and enable the drug manufacturers to penetrate more into the market.
• The companies and academics are working to assess challenges and seek opportunities that could influence Beta Thalassemia R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition.
• Major players are involved in developing therapies for Beta Thalassemia. The launch of emerging therapies will significantly impact the Beta Thalassemia market.
• Our in-depth analysis of the pipeline assets across different stages of development (Phase I, II, and III), different emerging trends, and comparative analysis of pipeline products with detailed clinical profiles, key cross-competition, and launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities.

Beta Thalassemia Report Insights

• Patient Population
• Therapeutic Approaches
• Beta Thalassemia Pipeline Analysis
• Beta Thalassemia Market Size and Trends
• Market Opportunities
• Impact of upcoming Therapies

Beta Thalassemia Report Key Strengths

• 11 Years Forecast
• 6MM Coverage
• Beta Thalassemia Epidemiology Segmentation
• Key Cross Competition
• Highly Analyzed Market

Drugs Uptake

• Beta Thalassemia Report Assessment
• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness

Key Questions

Market Insights:

• What was the Beta Thalassemia Market share (%) distribution in 2019, and how would it look in 2032?
• What would be the Beta Thalassemia total market size and market size by therapies across the 6MM during the forecast period (2019-2032)?
• What are the key findings of the market across 6MM, and which country will have the largest Beta Thalassemia market size during the forecast period (2019-2032)?
• At what CAGR, the Beta Thalassemia market is expected to grow by 6MM during the forecast period (2019-2032)?
• What would be the Beta Thalassemia market outlook across the 6MM during the forecast period (2019-2032)?
• What would be the Beta Thalassemia market growth till 2032, and what will be the resultant market Size in the year 2032?

Epidemiology Insights:

• What are the disease risk, burdens, and unmet needs of Beta Thalassemia?
• What is the historical Beta Thalassemia patient pool in six major markets covering the United States and EU5 (Germany, Spain, France, Italy, and UK)?
• What would be the forecasted patient pool of Beta Thalassemia in six major markets covering the United States and EU5 (Germany, Spain, France, Italy, and UK)?
• What will be the growth opportunities in the 6MM concerning the patient population about Beta Thalassemia?
• Out of all 6MM countries, which country would have the largest prevalent population of Beta Thalassemia during the forecast period (2019-2032)?
• At what CAGR is the population expected to grow by 6MM during the forecast period (2019-2032)?

Current Treatment Scenario, Marketed Drugs, and Emerging Therapies:

• What are the current options for the treatment of Beta Thalassemia along with the approved therapy?
• What are the current treatment guidelines for treating Beta Thalassemia in the USA and Europe?
• What is the Beta Thalassemia marketed drugs and their MOA, regulatory milestones, product development activities, advantages, disadvantages, safety, and efficacy, etc.?
• How many companies are developing therapies for the treatment of Beta Thalassemia?
• How many therapies are developed by each company for the treatment of Beta Thalassemia?
• How many emerging therapies are in the mid-stage and late stages of development to treat Beta Thalassemia?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to Beta Thalassemia therapies?
• What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Beta Thalassemia and its status?
• What key designations have been granted for the emerging therapies for Beta Thalassemia?
• What are the global historical and forecasted markets of Beta Thalassemia?

Reasons to buy

• The report will help develop business strategies by understanding trends shaping and driving the Beta Thalassemia market.
• To understand the future market competition in the Beta Thalassemia market and an Insightful review of the key market drivers and barriers.
• Organize sales and marketing efforts by identifying the best opportunities for Beta Thalassemia in the US and Europe (Germany, Spain, Italy, France, and the United Kingdom).
• Identifying strong upcoming players in the market will help devise strategies that will help get ahead of competitors.
• Organize sales and marketing efforts by identifying the best opportunities for the Beta Thalassemia market.
• To understand the future market competition in the Beta Thalassemia market.