Alpha Thalassemia Market Insight

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

 

The Alpha Thalassemia market report provides current treatment practices, emerging drugs, and market share of the individual drugs, current and forecasted 7MM Alpha Thalassemia market size from 2018 to 2030. The report also covers current Alpha Thalassemia treatment algorithm, market drivers, market barriers and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the market.

Geography Covered

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

Study Period: 2018–2030

Alpha Thalassemia Disease Understanding and Treatment Algorithm

Alpha Thalassemia Overview

Thalassemias are a group of blood disorders that affect the way the body makes hemoglobin. Alpha (α) thalassemia refers specifically to the abnormal or absent manufacturing of alpha-globin chains. These are associated with more than 15 different genetic mutations

 

The condition ranges from asymptomatic to severe. Sometimes, a person can be a carrier (silent α-thalassemia) with no sign of disease. Others may have very mild disease, known as having Alpha Thalassemia trait. Patients with Alpha Thalassemia traits require no treatment or long-term monitoring. While patients with α-thalassemia minor have lifelong mild anemia. Those with more moderate to severe cases may require blood transfusions or chelation therapy.

 

Complications of Alpha Thalassemia include excess iron, bone deformities, and broken bones, enlarged spleen, infections, and slower growth rates. The signs and symptoms of Alpha Thalassemia vary depending on the type that a child has and how severe it is. Children with Alpha Thalassemia trait and those who are silent carriers have no symptoms at all. Some of the more common symptoms of Alpha Thalassemia include fatigue, weakness, or shortness of breath, a pale appearance or yellow color to the skin (jaundice), irritability, and others.

 

Alpha Thalassemia Diagnosis

In order to identify fetuses with hemoglobin Bart’s hydrops fetalis and decrease the hazards to mothers, prenatal diagnostics is essential. The presence of hypochromic microcytic red cells in both parents, along with a normal hemoglobin A2 level, usually leads to the consideration of such a diagnosis; this combination would rule out β-thalassemia, which is characterized by an elevated hemoglobin A2 level. An iron deficiency must also be ruled out. The hematologic evaluation is followed by confirmation of the presence of α0-thalassemia in the parents when facilities for fast DNA diagnosis are available. The fetal diagnosis is usually made early in pregnancy utilizing chorionic-villus sampling. However, fetal anemia may also be diagnosed later during gestation by quantitation of the peak systolic velocity in the middle cerebral artery.

 

In the United States, infants may be diagnosed with α-thalassemia through newborn screening. Newborn screening is a public health program that mandates the evaluation of newborn infants for various treatable disorders, but not readily apparent at birth. Each state’s newborn screening program (and the specific disorders tested) is different, and further testing is required to determine the exact type of α-thalassemia present.

 

Initial laboratory testing should include a complete blood count with red cell indices, HPLC or Hb electrophoresis, and eventually α/β-globin chain synthesis ratio measurement. The latter procedure, however, is sometimes bypassed by DNA analysis as a less complicated method to diagnose α- thalassemia.

 

Alpha Thalassemia Treatment

The current treatment pattern for Alpha Thalassemia is dominated by the use of iron chelation therapies, blood transfusion, and bone marrow transplants.

Alpha Thalassemia Epidemiology

The disease epidemiology covered in the report provides historical as well as forecasted epidemiology segmented by diagnosed prevalent cases, diagnosed prevalent cases of Alpha Thalassemia based on transfusion requirement, and treatment eligible cases of Alpha Thalassemia in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan market from 2018 to 2030.

 

Key Findings

This section provides glimpse of the Alpha Thalassemia epidemiology in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan.

  • The total diagnosed prevalent cases of α-thalassemia in the 7MM range from 3,973 in 2020 growing at a CAGR of 1.3% during the study period (2018–2030).
  • Epidemiology assessed for the condition showed that the US, in 2020, accounted for approximately 1,415 diagnosed prevalent cases of α-thalassemia.
  • Among the EU-5 countries in 2020, Italy had the highest diagnosed prevalent cases of α-thalassemia with 1,003 cases, followed by Spain (410) and Germany (302). In contrast, France had the lowest cases (196) in 2020.
  • Japan accounted for 362 diagnosed prevalent α-thalassemia cases in 2020.
  • In the United States, in 2020, the proportion of non-transfusion-dependent thalassemia (NTDT) and transfusion-dependent thalassemia (TDT) cases were 859 and 556, respectively.
  • As per the analysis, a higher percentage of non-transfusion-dependent thalassemia (NTDT) was observed in the EU-5 compared to transfusion-dependent thalassemia (TDT) in 2020.
  • Among the EU5 countries, Italy accounted for the highest cases of NTDT cases in 2020, whereas France accounted for the lowest cases of NTDT cases in 2020.
  • Japan accounted for approximately 362 treatment eligible cases of α-thalassemia in 2020.
  • The United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan – Alpha Thalassemia Epidemiology

Alpha Thalassemia Drug Chapters

The drug chapter segment of the Alpha Thalassemia report encloses the detailed analysis of Alpha Thalassemia current treatment pattern and pipeline drugs. It also helps to understand the Alpha Thalassemia clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details of each included drugs and the latest news and press releases.

 

Alpha Thalassemia Emerging Drugs

 

Mitapivat: Agios Pharmaceuticals

Mitapivat is a first-in-class, investigational, oral, small-molecule allosteric activator of wild-type and a variety of mutated pyruvate kinase R (PKR) enzymes.

 

In addition to the Phase II extension study of mitapivat in adults with non-transfusion-dependent α- and β-thalassemia, based on the data from the Phase II proof of concept study, the company reported to advance the development of mitapivat in thalassemia as quickly and efficiently as possible, with the initiation of two Phase III studies of mitapivat, ENERGIZE and ENERGIZE-T, in non-transfusion-dependent and transfusion-dependent adults with thalassemia, respectively. The company anticipates the approval of the drug in 2025 and also plans to conduct trials for pediatric thalassemia.

Product details in the report…

 

Etavopivat: Forma Therapeutics

Etavopivat is an investigational, oral, selective red blood cell pyruvate kinase-R (PKR) activator designed to be a disease-modifying therapy initially being studied for the treatment of SCD. The company is currently evaluating the drug in a Phase II trial for patients with thalassemia TD/NTD.

 

By employing a multimodal approach, etavopivat is designed to work by activating the RBCs’ natural PKR activity to decrease 2,3-DPG levels, which leads hemoglobin to hold on to oxygen molecules longer to reduce polymerization and RBC sickling. Etavopivat-mediated PKR activation also increases ATP levels, the fuel that provides energy to cells, to improve RBC health and survival. Together, these effects are anticipated to increase hemoglobin levels and decrease painful vaso-occlusive crises.

 

In preclinical safety studies, the drug did not inhibit aromatase activity or affect steroidogenesis, important biological processes responsible for sexual development. The drug has been granted Fast Track, Rare Pediatric Disease and Orphan Drug designations from the US FDA, and Orphan Drug Designation from the European Commission based on a positive opinion from the Committee for Orphan Medicinal Products of the EMA for the treatment of patients with SCD.

 

The company further estimates to initiate Phase II trial in December 2021 for the treatment of patients with SCD TD and Thalassemia TD/NTD, with results anticipated in 2022. Moreover, the company anticipates conducting trials for pediatrics (down to age 6 months) in SCD in early 2022.

Product details in the report…

Alpha Thalassemia Market Outlook

Alpha (α)-thalassemia is an inherited disorder in which the body cannot produce enough hemoglobin. The condition ranges from asymptomatic to severe. Sometimes, a person can be a carrier (silent α-thalassemia) with no sign of disease. Others may have very mild disease, known as having Alpha Thalassemia trait.

 

We assessed the α-thalassemia market based on demand, prescription analysis, and the annual cost of therapy of current and forecasted market value of the current regimen suitable for the patient pool. We found that the current market of α-thalassemia lacks approved therapies, and the treatment depends on the severity of the condition. Alpha globin is made by four genes and one or more can be mutated or missing, so there are four kinds of Alpha Thalassemia. Types of Alpha Thalassemia include silent Alpha Thalassemia (one missing or abnormal gene, no signs or symptoms of the disease, but are able to pass thalassemia on to their children), Alpha Thalassemia minor (two missing or mutated genes, children with this condition may have red blood cells that are smaller than normal (microcytosis) and sometimes very slight anemia), hemoglobin H disease (three missing or mutated genes, signs and symptoms will be moderate to severe), Alpha Thalassemia major or hydrops fetalis (four missing or mutated genes, almost always leads to a fetus dying before delivery or a newborn baby dying shortly after birth).

 

  • Patients with Alpha Thalassemia traits require no treatment or long-term monitoring. They usually do not have iron deficiency, so iron supplements will not improve their anemia.
  • Patients with the α-thalassemia minor will have lifelong mild anemia. Those with more moderate to severe cases may require blood transfusions or chelation therapy.

 

Transfusions: Patients with HbH disease usually have moderate anemia that is well-tolerated. However, transfusions are occasionally needed during illnesses with a fever due to accelerated RBCs breakdown. Adults may require more regular transfusions. Those with a more severe form of HbH disease (called Hemoglobin H-Constant Spring disease) can have significant anemia and require frequent transfusions during their lifetime.

 

Iron chelation therapy: Patients with HbH disease may develop iron overload. This may occur even in the absence of blood transfusions secondary to increased absorption of iron in the small intestine. Medications called chelators can help rid the body of excess iron.

 

Two drugs are used for chelation therapy:

  • Deferoxamine: It is a liquid medicine given slowly under the skin, usually overnight, 5–6 nights a week, with a small, portable pump. This treatment takes time and can be mildly painful, and side effects may include problems with hearing and vision.
  • Deferasirox: It is an oral drug taken by mouth once a day. Side effects may include headache, nausea, vomiting, diarrhea, tiredness, and joint pain.

 

A bone marrow transplant has helped cure a small number of individuals with severe α-thalassemia.

 

Key Findings

  • The market size of α-thalassemia in the seven major markets is expected to rise from USD 153.11 million in 2020 during the study period (2018–2030).
  • Current market dynamics are dominated by the use of blood transfusions, iron chelation, and others (bone marrow or stem cell transplant, surgery, etc.)
  • The therapies with the potential to get launched in the forecast period include mitapivat (AG-348), etavopivat (FT-4202), and SLN124. The launch of these therapies may increase market size in the coming years, assisted by an increasing patient pool of α-thalassemia patients.
  • The United States accounts for the largest market size of Alpha Thalassemia compared to EU5 (the United Kingdom, Germany, Italy, France, and Spain) and Japan. In the United States, the market size of α-thalassemia is anticipated to rise from USD 74.64 million in 2020 during the forecast period.
  • Among the EU5 countries, Italy had the largest market size (USD 30.76 million) in 2020, while France had the smallest with USD 6.01 million.
  • In 2020, Japan accounted for a market size of USD 11.11 million.

Analyst views

  • Most of the attention in the field of thalassemia is given to the research and development of drugs for patients with (β)-thalassemia even though (α)-thalassemia is almost universally fatal in utero and represents an orphan disease with an unmet need for effective therapies. The current market does not hold any FDA or EMA-approved drugs for patients with α-thalassemia. Despite the lack of current treatment options, only a handful of drugs are in development. Any medication, if approved, may capture a major share of the market due to the lack of treatment options for this patient pool.
  • After years of scarcity, Agios initiated a Phase II trial for patients with this condition. Based on the positive results presented at ASH 2021, the company has initiated Phase III trials for patients with NTDT and TDT in 2021. According to the company reports, the drug may be launched in 2025 in the US. We believe that the drug holds the potential to address the unmet needs in the field. The drug may prove to be a blockbuster considering the scarcity of any approved therapy for α-thalassemia patients, provided that the drug demonstrates promising efficacy and safety data in the ongoing Phase III trials.
  • We expect other therapies in Phase II and Phase I stages of development by Forma and Silence Therapeutics, respectively, to change the market dynamics after analyzing the data presented by both companies in ASH 2021.
  • Forma Therapeutics has recently initiated the Phase II trial in patients with thalassemia TD/NTD, with results anticipated in 2022. We took into account the results presented by the company from the ongoing trial in SCD patients.
  • Silence Therapeutics has recently initiated a Phase I trial in patients with MDS and thalassemia. The drug in development is based on the company’s mRNAi GOLD platform. The therapy is a gene ‘silencing’ therapy aimed to silence TMPRSS6 temporarily, thereby preventing the liver from producing hepcidin. Levels of hepcidin are inversely proportional to iron levels in the blood, which may increase the production of healthy RBCs, thereby reducing anemia. Thus, the therapy has the potential to reduce the burden on patients with this condition. However, it is in the early stages of development, with data anticipated in the third quarter of 2022.
  • According to an analysis by DelveInsight, mitapivat (AG-348) has the potential to grab a major patient pool during the forecast period. The oral route of administration and the company’s visibility around the product may set the drug for approval by the US FDA by 2025, EMA by 2026, and PMDA by 2026. The drug’s potential is attributed to the strong safety and efficacy demonstrated by the drug during clinical development.

Alpha Thalassemia Drug Uptake

  • This section focusses on the rate of uptake of the potential drug launched in the Alpha Thalassemia market or expected to get launched in the market during the study period 2018–2030. The analysis covers Alpha Thalassemia market uptake by drug; patient uptake by drug; and sales of each drug.
  • This helps in understanding the drug with the most rapid uptake, reasons behind the maximal use of new drug and allow the comparison of the drug on the basis of market share and size which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.
  • Alpha Thalassemia Development Activities
  • The report provides insights into different candidates in phase II, and phase III stage. It also analyzes key players involved in developing targeted drug.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing and patent details for Alpha Thalassemia emerging drug.

Competitive Intelligence Analysis

We perform competitive and market intelligence analysis of the Alpha Thalassemia market by using various competitive intelligence tools that include–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 Alpha Thalassemia, explaining its causes, signs and symptoms, pathogenesis and currently available drug.
  • Comprehensive insight has been provided into the Alpha Thalassemia epidemiology and treatment.
  • Additionally, an all-inclusive account of both the current and emerging drug for Alpha Thalassemia are provided, along with the assessment of new drug, which will have an impact on the current Alpha Thalassemia treatment landscape.
  • A detailed review of Alpha Thalassemia market; historical and forecasted is included in the report, covering the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan drug outreach.
  • The report provides an edge while developing business strategies, by understanding trends shaping and driving the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan Alpha Thalassemia market.

Report Highlights

  • In the coming years, Alpha Thalassemia market is set to change due to the rising awareness of the disease, and incremental healthcare spending across the world; which would expand the size of the market to 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 Alpha Thalassemia R&D. The drug under development are focused on novel approaches to treat/improve the disease condition.
  • Delvelnsight has analysed the total age-specific and total vaccinated population of Alpha Thalassemia.

Alpha Thalassemia Report Insights

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

Alpha Thalassemia Report Key Strengths

  • Ten Years Forecast
  • The United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan Coverage
  • Alpha Thalassemia Epidemiology Segmentation
  • Key Cross Competition
  • Highly Analyzed Market
  • Drug Uptake

Alpha Thalassemia Report Assessment

  • Current Treatment Practices
  • Unmet Needs
  • Pipeline Product Profiles
  • Market Attractiveness
  • Market Drivers and Barriers

Key Questions

Market Insights:

  • What was the Alpha Thalassemia market share (%) distribution in 2018 and how it would look like in 2030?
  • What would be the Alpha Thalassemia total market size as well as market size by drug in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan during the forecast period (2021–2030)?
  • What are the key findings pertaining to the market in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan?
  • At what CAGR, the Alpha Thalassemia market is expected to grow at the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan level during the forecast period (2021–2030)?
  • What would be the Alpha Thalassemia market outlook in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), aduring the forecast period (2021–2030)?
  • What would be the Alpha Thalassemia market growth till 2030 and what will be the resultant market size in the year 2030?
  • How would the market drivers, barriers and future opportunities affect the market dynamics and subsequent analysis of the associated trends?

 

Epidemiology Insights:

  • What is the disease risk, burden and unmet needs of Alpha Thalassemia?
  • What is the historical Alpha Thalassemia patient pool in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan?
  • What would be the forecasted vaccinated pool of Alpha Thalassemia at the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), level?
  • What will be the growth opportunities in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan with respect to the patient population pertaining to Alpha Thalassemia?
  • At what CAGR the population is expected to grow in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan during the forecast period (2021–2030)?

 

Current Treatment Scenario, Marketed Drug and Emerging Drug:

  • What are the current options for the treatment of Alpha Thalassemia along with the approved drug?
  • What are the current treatment guidelines for the prevention of Alpha Thalassemia in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan?
  • What are the Alpha Thalassemia marketed drug and their MOA, regulatory milestones, product development activities, advantages, disadvantages, safety and efficacy, etc.?
  • How many companies are developing drug for the treatment of Alpha Thalassemia?
  • How many drug are developed by each company for the treatment of Alpha Thalassemia?
  • How many emerging drug are in the mid-stage and late stage of development for the treatment of Alpha Thalassemia?
  • What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Alpha Thalassemia drug?
  • What are the recent novel drug, targets, mechanisms of action and technologies developed to overcome the limitation of existing drug?
  • What are the clinical studies going on for Alpha Thalassemia and their status?
  • What are the key designations that have been granted for the emerging drug for Alpha Thalassemia?
  • What is the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan historical and forecasted market of Alpha Thalassemia?

Reasons to buy

  • The report will help in developing business strategies by understanding trends shaping and driving the Alpha Thalassemia.
  • To understand the future market competition in the Alpha Thalassemia market and Insightful review of the key market drivers and barriers.
  • Organize sales and marketing efforts by identifying the best opportunities for Alpha Thalassemia in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan.
  • Identification of strong upcoming players in the market will help in devising strategies that will help in getting ahead of competitors.
  • Organize sales and marketing efforts by identifying the best opportunities for Alpha Thalassemia market.
  • To understand the future market competition in the Alpha Thalassemia market.

1. Key Insights

2. Report Introduction

3. Alpha Thalassemia Market Overview at a Glance

3.1. Market Share (%) Distribution of Alpha Thalassemia in 2020

3.2. Market Share (%) Distribution of Alpha Thalassemia in 2030

4. Executive Summary of Alpha Thalassemia

5. Key Events

6. Epidemiology and Market Forecast Methodology

7. SWOT Analysis

8. Disease Background and Overview

8.1. Signs and Symptoms

8.2. Causes

8.3. Clinical Manifestations

8.4. Pathophysiology

8.5. Diagnosis

8.5.1. Hematology

8.5.2. Alpha/beta-globin chain synthesis

8.5.3. Molecular analysis

8.6. Differential diagnosis and Genetic Counseling

9. Treatment and Management of Alpha-thalassemia

10. Guidelines for the Management of Transfusion Dependent Thalassemia (TDT) (Thalassemia International Federation, 2021)

11. Guidelines for the Management of Non-transfusion-dependent Thalassemia (NTDT)

12. Centers for Disease Control and Prevention (CDC) - 2016

13. Epidemiology and Patient Population

13.1. Key Findings

13.2. Total Diagnosed Prevalent Population of Alpha Thalassemia in the 7MM

13.3. Diagnosed Prevalent Cases of Alpha Thalassemia based on Transfusion Requirement in the 7MM

13.4. Treatment Eligible Cases of Alpha Thalassemia in the 7MM

13.5. Assumptions and Rationale

13.6. The United States

13.6.1. Total Diagnosed Prevalent Population of Alpha Thalassemia in the United States

13.6.2. Diagnosed Prevalent Cases of Alpha Thalassemia based on Transfusion Requirement in the United States

13.6.3. Treatment Eligible Cases of Alpha Thalassemia in the United States (2018–2030) in the United States

13.7. EU5

13.7.1. Total Diagnosed Prevalent Population of Alpha Thalassemia in EU5

13.7.2. Diagnosed Prevalent Cases of Alpha Thalassemia based on Transfusion Requirement in EU5

13.7.3. Treatment eligible Cases of Alpha Thalassemia in EU5

13.8. Japan

13.8.1. Total Diagnosed Prevalent Population of Alpha Thalassemia in Japan

13.8.2. Diagnosed Prevalent Cases of Alpha Thalassemia based on Transfusion Requirement in Japan

13.8.3. Treatment eligible Cases of Alpha Thalassemia in Japan

14. Organizations contributing towards Alpha Thalassemia

15. Patient Journey

16. Emerging Therapies

16.1. Key Competitors

16.2. Mitapivat: Agios Pharmaceuticals

16.2.1. Drug description

16.2.2. Other development activities

16.2.3. Clinical development

16.2.4. Safety and Efficacy

16.3. Etavopivat: Forma Therapeutics

16.3.1. Drug description

16.3.2. Clinical development

16.3.3. Safety and Efficacy

16.4. SLN124: Silence Therapeutics

16.4.1. Drug description

16.4.2. Other development activities

16.4.3. Clinical development

16.4.4. Safety and Efficacy

17. Alpha Thalassemia: Seven Major Market Analysis

17.1. Key Findings

17.2. Total Market Size of Alpha Thalassemia in the 7MM

17.3. Market Size of Alpha Thalassemia by therapies in the 7MM

17.4. Market Outlook

17.5. Key Market Forecast Assumptions

17.6. United States Market Size

17.6.1. Total Market Size of Alpha Thalassemia in the United States

17.6.2. Market Size of Alpha Thalassemia by Therapies in the United States

17.7. EU-5 Market Size

17.7.1. Total Market size of Alpha Thalassemia in EU5

17.7.2. Market Size of Alpha Thalassemia by Therapies in EU5

17.8. Japan

17.8.1. Total Market size of Alpha Thalassemia in Japan

17.8.2. Market Size of Alpha Thalassemia by Therapies in Japan

18. Market Drivers

19. Market Barriers

20. Unmet Needs

21. Reimbursement and Market Access

21.1. Reimbursement of Rare Diseases Therapies

21.2. United States Reimbursement Policies

21.3. Europe Reimbursement Policies

21.4. Japan reimbursement policies

21.5. Co-pay Program

22. Appendix

23. Report Methodology

24. DelveInsight Capabilities

25. Disclaimer

26. About DelveInsight

List of Table

Table 1: Summary of Alpha Thalassemia Market, and Epidemiology (2018–2030)

Table 2: Key Events

Table 3: Red Blood Cell Indices in Individuals with Hb Bart Syndrome and HbH Disease

Table 4: Genotype-phenotype associations in α-thalassemia

Table 5: Total Diagnosed Prevalent Population of Alpha Thalassemia in the 7MM (2018–2030)

Table 6: Diagnosed Prevalent Cases of Alpha Thalassemia based on Transfusion Requirement in the 7MM (2018–2030)

Table 7: Treatment Eligible Cases of Alpha Thalassemia in the 7MM (2018–2030)

Table 8: Total Diagnosed Prevalent Population of Alpha Thalassemia in the US (2018–2030)

Table 9: Diagnosed Prevalent Cases of Alpha Thalassemia based on Transfusion Requirement in the US (2018–2030)

Table 10: Treatment Eligible Cases of Alpha Thalassemia in the United States (2018–2030)

Table 11: Total Diagnosed Prevalent Population of Alpha Thalassemia in EU5 (2018–2030)

Table 12: Diagnosed Prevalent Cases of Alpha Thalassemia based on Transfusion Requirement in EU5 (2018–2030)

Table 13: Treatment Eligible Cases of Alpha Thalassemia in EU5 (2018–2030)

Table 14: Total Diagnosed Prevalent Population of Alpha Thalassemia in the Japan (2018–2030)

Table 15: Diagnosed Prevalent Cases of Alpha Thalassemia based on Transfusion Requirement in Japan (2018–2030)

Table 16: Treatment Eligible Cases of Alpha Thalassemia in Japan (2018–2030)

Table 17: Organizations Contributing Toward the Fight Against Alpha thalassemia

Table 18: Emerging Drugs in Development

Table 19: Mitapivat, Clinical Trial Description, 2021

Table 20: Etavopivat, Clinical Trial Description, 2021

Table 21: SLN124, Clinical Trial Description, 2021

Table 22: Market Size of Alpha Thalassemia in the 7MM in USD Million (2018–2030)

Table 23: Market Size of Alpha Thalassemia by Therapies in the 7MM in USD Million (2018–2030)

Table 24: Key Market Forecast Assumptions for mitapivat

Table 25: Key Market Forecast Assumptions for Etavopivat

Table 26: Key Market Forecast Assumptions for SLN124

Table 27: Market Size of Alpha Thalassemia in the US, USD Million (2018–2030)

Table 28: Market Size of Alpha Thalassemia by Therapies in the US, USD Million (2018–2030)

Table 29: EU5 Market Size of Alpha Thalassemia in USD Million (2018–2030)

Table 30: EU5 Market Size of Alpha Thalassemia by Therapies in USD Million (2018–2030)

Table 31: Japan Market Size of Alpha Thalassemia in Japan, USD Million (2018–2030)

Table 32: Market Size of Alpha Thalassemia by Therapies in Japan, USD Million (2018–2030)

Table 33: Available Infrastructure for Patients in Thalassemia Countries of Priority

List of Figures

Figure 1: Phenotype–Genotype Relationship in α-thalassemia

Figure 2: The Clinical Spectrum of Thalassemia Syndromes Based on Their Requirement of Regular Blood Transfusions into NTDT and TDT

Figure 3: Total Diagnosed Prevalent Population of Alpha Thalassemia in the 7MM (2018–2030)

Figure 4: Diagnosed Prevalent Cases of Alpha Thalassemia based on Transfusion Requirement in the 7MM (2018–2030)

Figure 5: Treatment Eligible Cases of Alpha Thalassemia in the 7MM (2018–2030)

Figure 6: Total Diagnosed Prevalent Population of Alpha Thalassemia in the US (2018–2030)

Figure 7: Diagnosed Prevalent Cases of Alpha Thalassemia based on Transfusion Requirement in the US (2018–2030)

Figure 8: Treatment Eligible Cases of Alpha Thalassemia in the United States (2018–2030)

Figure 9: Total Diagnosed Prevalent Population of Alpha Thalassemia in EU5 (2018–2030)

Figure 10: Diagnosed Prevalent Cases of Alpha Thalassemia based on Transfusion Requirement in EU5 (2018–2030)

Figure 11: Treatment Eligible Cases of Alpha Thalassemia in EU5 (2018–2030)

Figure 12: Total Diagnosed Prevalent Population of Alpha Thalassemia in Japan (2018–2030)

Figure 13: Diagnosed Prevalent Cases of Alpha Thalassemia based on Transfusion Requirement in Japan (2018–2030)

Figure 14: Treatment Eligible Cases of Alpha Thalassemia in Japan (2018–2030)

Figure 15: Patient Journey

Figure 16: Market Size of Alpha Thalassemia in the 7MM in USD Million (2018–2030)

Figure 17: Market Size of Alpha Thalassemia by Therapies in the 7MM in USD Million (2018–2030)

Figure 18: Market Size of Alpha Thalassemia in the US, USD Millions (2018–2030)

Figure 19: Market Size of Alpha Thalassemia by Therapies in the US, USD Million (2018–2030)

Figure 20: Market Size of Alpha Thalassemia in EU5, USD Million (2018–2030)

Figure 21: EU5 Market Size of Alpha Thalassemia by Therapies in USD Million (2018–2030)

Figure 22: Market Size of Alpha Thalassemia in Japan, USD Million (2018–2030)

Figure 23: Japan Market Size of Alpha Thalassemia by Therapies, USD Million (2018–2030)

Agios Pharmaceuticals
Forma Therapeutics
Silence Therapeutics

 

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