알파 지중해빈혈 : 시장 인사이트, 역학, 시장 예측(2034년)

Key Highlights:

• The Alpha thalassemia Market Size in the 7MM was ~ 150 Million in 2023.
• The current Alpha Thalassemia Drugs Market lacks approved therapies, and the treatment depends on the severity of the condition.
• After years of scarcity, there has been an increase in research and development regarding a-thalassemia. Moreover, organizations such as Cooley 's Anemia Foundation, Thalassemia International Federation, and others are working to increase awareness of the condition and its underlying factors.
• The current treatment pattern for alpha thalassemia is dominated by the use of iron chelation therapies, blood transfusion, and bone marrow transplants.
• Even though a-thalassemia is a disease with a high rate of fetal mortality in severe conditions, most of the attention in the field of thalassemia is restricted to patients with B-thalassemia.
• Total Alpha thalassemia Prevalence Cases in the 7MM in 2023 was more than 4,000.
• The condition is more prevalent in the eastern and developing nations. These individuals usually die at an early age due to poor quality of life and lack of supportive healthcare knowledge. Thus, the market may face a major threat in these regions.
• Promising Alpha thalassemia Drugs in the pipeline include Mitapivat (Agios Pharmaceuticals), Etavopivat (Novo Nordisk), Luspatercept (Bristol-Myers Squibb).

DelveInsight's " Alpha thalassemia Drugs Market Insights, Epidemiology, and Market Forecast - 2034" report delivers an in-depth understanding of Alpha thalassemia, historical and forecasted epidemiology as well as the Alpha thalassemia market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

The Alpha thalassemia Drugs Market Report provides current treatment practices, emerging drugs, Alpha thalassemia market share of individual therapies, and current and forecasted Alpha thalassemia market size from 2020 to 2034, segmented by seven major markets. The report also covers current Alpha thalassemia treatment market practices/algorithms and Alpha thalassemia unmet needs to curate the best of the opportunities and assess the underlying potential of the Alpha Thalassemia drugs market.

Alpha Thalassemia Treatment Market

Alpha (a) thalassemia refers specifically to the abnormal or absent manufacturing of alpha-globin chains. These are associated with more than 15 different genetic mutations. The severity of the clinical condition is based on the mutation type. The severity of mutation is based on which of the two alpha-globin loci is affected. Mutations can also be deletions or non-deletion. In deletion mutation, there is an inheritance of a single alpha-globin gene. With the non-deletion type, a patient has inherited two a-globin genes, but one gene carries a nondeletion abnormality, for example, point mutation.

In nondeletion, the severity of clinical expression is also affected depending on whether the mutation blocks the production of the remaining normal alpha chains partially or fully. Hemoglobin H disease occurs when only one normal alpha gene has been inherited. One of these most common non-deletion subtypes of Hemoglobin H is Hemoglobin Constant Spring. Hemoglobin H (HbH) disease tends to be more severe in patients with nondeletion type, likely due to interference with the transcription of the normal a chain gene by the abnormal one.

Alpha thalassemia Diagnosis

Diagnosing alpha thalassemia requires a thorough assessment, including medical history, physical exam, and lab tests like CBC and hemoglobin electrophoresis. Genetic testing is crucial for confirmation, pinpointing mutations in alpha-globin genes. Prenatal screening may be considered for families with a history of the condition. This comprehensive approach ensures accurate diagnosis and effective management for those with alpha thalassemia.

Alpha Thalassemia treatment depends on the severity and type of the condition. In Transfusion-dependent thalassemia (TDT), individuals relying on regular blood transfusions for survival, management aims to address complications and maintain optimal haemoglobin levels. This includes closely monitoring iron levels and managing iron overload through chelation therapy. In non-transfusion-dependent cases, where regular transfusions are not needed for survival but may be required during periods of stress, treatment focuses on managing symptoms and complications. This may involve blood transfusions during acute crises and measures to support overall health and well-being. Early detection and appropriate management are essential for improving outcomes and quality of life for individuals with alpha thalassemia.

Alpha Thalassemia Epidemiology

The Alpha thalassemia epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by the total diagnosed prevalent cases of alpha thalassemia, diagnosed prevalent cases of alpha thalassemia based on transfusion requirement, treatment eligible cases of alpha thalassemia in the 7MM covering the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan from 2020 to 2034.

• Among the 7MM, the US accounted for the highest alpha thalassemia prevalence cases in 2023, with more than 1400 cases; these cases are expected to increase during the forecast period.
• Among EU4 and the UK, the total Alpha Thalassemia prevalent cases was maximum in Italy, while the lowest number of cases were in France in 2023.
• As per our analysis, the case of non-transfusion-dependent thalassemia (NTDT) cases was higher than that of transfusion-dependent thalassemia (TDT).

Alpha Thalassemia Recent Developments

• In January 2025, Agios Pharmaceuticals (AGIO) announced that the FDA has accepted its supplemental new drug application (sNDA) for label expansion of Pyrukynd, its only marketed drug, for the treatment of thalassemia.

Alpha Thalassemia Drugs Chapters

The drug chapter segment of the alpha thalassemia market outlook report encloses a detailed analysis of the late-stage (Phase III ) and mid-stage (Phase II/III and Phase II) Alpha Thalassemia pipeline drugs. The current key Alpha Thalassemia Companies include Agios Pharmaceuticals (Mitapivat), Novo Nordisk (Etavopivat), and Bristol-Myers Squibb (Luspatercept). The drug chapter also helps understand the Alpha thalassemia clinical trials details, pharmacological action, agreements and collaborations, approval, and patent details, and the latest Alpha thalassemia 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. The company expects to release the results of the ENERGIZE-T trial in the second quarter of 2024. Additionally, they are aiming for regulatory approval of the drug in 2025 and are strategizing to initiate clinical trials for pediatric thalassemia.

In January 2024, Agios Pharmaceuticals announced that the global Phase III ENERGIZE study of mitapivat in adults with non-transfusion dependent (NTD) alpha-or beta-thalassemia achieved its primary endpoint of hemoglobin response.

• Etavopivat: Novo Nordisk

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 III trial for patients with thalassemia TD/NTD. Novo Nordisk and Forma Therapeutics entered into a definitive agreement under which Novo Nordisk acquired Forma Therapeutics in September 2022.

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. Currently, the drug is being investigated in a Phase II clinical trial in patients with thalassemia.

Alpha Thalassemia Drugs Markets Insights

Pyruvate kinase is a key enzyme in glycolysis, the sole source of adenosine triphosphate, which is essential for all energy-dependent activities of red blood cells. Activating Pyruvate kinase shows great potential for treating a broad range of hemolytic anemias beyond Pyruvate kinase deficiency because it also enhances the activity of wild-type Pyruvate kinase. Motivated by observations of sickle-cell complications in sickle-trait individuals with concomitant Pyruvate kinase deficiency, activating endogenous Pyruvate kinase offers a novel and promising approach for treating patients with sickle-cell disease.

TMPRSS6 is a serine protease highly expressed in the liver. Its role in iron regulation was first reported in 2008 when mutations in TMPRSS6 were shown to be the cause of iron-refractory Iiron Deficiency Anemia (IRIDA) in humans and mouse models. TMPRSS6 functions as a negative regulator of the expression of the systemic iron-regulatory hormone hepcidin. Over the last decade and a half, a growing understanding of TMPRSS6 biology and mechanism of action has enabled the development of new therapeutic approaches for patients with diseases of erythropoiesis and iron homeostasis.

Alpha thalassemia Market Outlook

Most of the attention in the field of thalassemia is given to the research and development of drugs for patients with (B)-thalassemia even though (a)-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 a-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.

Some treatments include blood transfusions, iron chelation therapy to manage iron overload, and bone marrow transplantation in severe cases. In addition, the pipeline holds in-utero hematopoietic stem cell transplantation for a-thalassemia major (ATM). The HSCT is in development by the University of California, San Francisco, in collaboration with the California Institute for Regenerative Medicine (CIRM). The treatment is based on the fact that the only current treatment to allow the fetus to be born is to perform in-utero transfusions (IUT) of RBCs to treat the anemia and avoid the complications of hydrops and fetal demise. Often, affected pregnancies undergo elective termination after diagnosis. Cases with the prenatal diagnosis of ATM who receive IUT and survive to birth will ultimately require lifelong monthly blood transfusions or bone marrow transplants if a suitable donor is identified. The Phase I clinical trial is aimed to demonstrate the safety, feasibility, and efficacy of performing in-utero stem cell transplantation on fetuses affected with ATM.

Key Findings

• The total Alpha thalassemia Market Size in the US was estimated to be more than USD 70 million in 2023, which is expected to grow during the forecast period (2024-2034).
• Among the EU4 and the UK, Italy accounted for the largest Alpha Thalassemia market share with a USD 30 million Alpha Thalassemia market size in 2023, which is expected to grow during the forecast period (2024-2034).
• According to an analysis, mitapivat 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. The drug's potential is attributed to the strong safety and efficacy demonstrated by the drug during clinical development.

Alpha thalassemia Drugs Uptake

This section focuses on the uptake rate of potential Alpha thalassemia drugs expected to be launched in the market during 2024-2034. The Alpha Thalassemia treatment market landscape has experienced a transformation with the uptake of novel drugs. These innovative therapies are redefining standards of care. Furthermore, the increased uptake of these transformative drugs is a testament to the unwavering dedication of hematologists and other professionals and the entire healthcare community in their tireless pursuit of advancing healthcare. This momentous shift in treatment paradigms is a testament to the power of research, collaboration, and human resilience. AG-348 (Mitapivat) is an allosteric activator of red blood cell pyruvate kinase that increases enzymatic activity, protein stability, and ATP levels. It is expected that the drug will enter the market by 2025.

Alpha thalassemia Pipeline Development Activities

The Alpha thalassemia drugs market report provides insights into therapeutic candidates in Phase III, Phase II/III, and Phase II. It also analyzes Alpha thalassemia companies involved in developing targeted therapeutics. Alpha thalassemia companies like Agios Pharmaceuticals, Novo Nordisk, Bristol-Myers Squibb, and others actively engage in late and mid-stage research and development efforts for alpha thalassemia. The pipeline alpha thalassemia possesses few potential drugs. However, there is a positive outlook for the therapeutics market, with expectations of growth during the forecast period (2024-2034).

Pipeline Development Activities

The Alpha thalassemia pipeline segment covers information on collaborations, acquisitions and mergers, licensing, and patent details for Alpha thalassemia emerging therapy.

KOL Views

To keep up with current Alpha thalassemia drugs market trends, we take KOLs and SMEs' opinions working in the domain through primary research to fill the data gaps and validate our secondary research. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the Alpha thalassemia unmet needs. Our in-house conference/congress intelligence experts gather information from various websites of congresses and analyze the data to map the KOLs attending the congresses

Qualitative Analysis

We perform Qualitative and Alpha Thalassemia drugs market Intelligence analysis using various approaches, such as SWOT analysis and Analyst views. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided. These pointers are based on the Analyst's discretion and assessment of the patient burden, cost analysis, and existing and evolving Alpha thalassemia treatment landscape.

Alpha Thalassemia Drugs Market Access and Reimbursement

Reimbursement of rare disease therapies can be limited due to lack of supporting policies and funding, challenges of high prices, lack of specific approaches to evaluating rare disease drugs given limited evidence, and payers' concerns about budget impact. The high cost of rare disease drugs usually has a limited impact on the budget due to the small number of eligible patients being prescribed the drug. From a patient perspective, health insurance and payer coverage guidelines surrounding rare disease treatments restrict broad access to these treatments, leaving only a small number of patients who can bypass insurance and pay for products on their own. Insured patients in the United States are typically granted access to rare disease drugs. This is due to several factors, including about half of rare diseases affecting children, which makes management difficult without coverage after approval.

Alpha Thalassemia Therapeutics Market Report Scope

• The Alpha Thalassemia Therapeutics Market Report covers a segment of key events, an executive summary, and a descriptive overview, explaining its causes, signs, symptoms, pathogenesis, and currently used therapies.
• Comprehensive insight into the epidemiology segments and forecasts, disease progression, and Alpha Thalassemia treatment market guidelines has been provided.
• Additionally, an all-inclusive account of the Alpha Thalassemia emerging therapies and the elaborative profiles of late-stage and prominent therapies will impact the current Alpha Thalassemia treatment market landscape.
• A detailed review of the alpha thalassemia therapeutics market, historical and forecasted Alpha Thalassemia market size, Alpha Thalassemia market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach.
• The Alpha Thalassemia therapeutics market report provides an edge while developing business strategies by understanding trends through SWOT analysis and expert insights/KOL views, patient journey, and treatment preferences that help shape and drive Alpha thalassemia.

Alpha thalassemia Therapeutics Market Report Insights

• Patient-based Alpha Thalassemia Market Forecasting
• Therapeutic Approaches
• Alpha thalassemia Pipeline Drugs Analysis
• Alpha thalassemia Market Size
• Alpha Thalassemia Market Trends
• Existing and Future Alpha Thalassemia Therapeutics Market Opportunity

Alpha thalassemia Therapeutics Market Report Key Strengths

• 11 Years Alpha Thalassemia Market Forecast
• The 7MM Coverage
• Alpha thalassemia Epidemiology Segmentation
• Key Cross Competition
• Alpha Thalassemia Drugs Uptake
• Key Alpha Thalassemia Market Forecast Assumptions

Alpha Thalassemia Treatment Market Report Assessment

• Current Alpha Thalassemia Treatment Market Practices
• Alpha Thalassemia Unmet Needs
• Alpha Thalassemia Pipeline Product Profiles
• Alpha Thalassemia Drugs Market Attractiveness
• Qualitative Analysis (SWOT and Analyst Views)

FAQs:

• What was the Alpha thalassemia market size, the Alpha Thalassemia market size by therapies, Alpha Thalassemia market share (%) distribution in 2023, and what would it look like by 2034? What are the contributing factors for this growth?
• What can be the future treatment paradigm for Alpha thalassemia?
• What are the disease risks, burdens, and unmet needs of alpha thalassemia? What will be the growth opportunities across the 7MM concerning the patient population with alpha thalassemia?
• What are the current options for the treatment of Alpha thalassemia? What are the current guidelines for treating Alpha thalassemia in the 7MM?
• What will be the impact of Mitapivat's expected approval by 2025?
• What is the patient share in Alpha thalassemia?

Reasons to Buy:

• The Alpha Thalassemia Therapeutics Market Report will help develop business strategies by understanding the latest trends and changing treatment dynamics driving alpha thalassemia treatment market.
• Insights on patient burden Alpha Thalassemia prevalence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years.
• Understand the existing Alpha Thalassemia therapeutics market opportunities in varying geographies and the growth potential over the coming years.
• Identifying strong upcoming Alpha Thalassemia Companies in the Alpha Thalassemia therapeutics market will help devise strategies to help get ahead of competitors.
• Detailed analysis ranking of class-wise potential emerging therapies under the analyst view section to provide visibility around leading classes.
• Highlights of access and reimbursement policies of current therapies, barriers to accessibility of expensive off-label therapies, and patient assistance programs.
• To understand Key Opinion Leaders' perspectives around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.
• Detailed insights on the Alpha Thalassemia unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.

Table of Contents

1. Key Insights

2. Report Introduction

3. Alpha Thalassemia Drugs Market Overview at a Glance

• 3.1. Market Share (%) Distribution of Alpha Thalassemia in 2020
• 3.2. Market Share (%) Distribution of Alpha Thalassemia in 2034

4. Executive Summary of Alpha Thalassemia

5. Key Events

6. Epidemiology and Market Forecast Methodology

7. Disease Background and Overview

• 7.1. Signs and Symptoms
• 7.2. Causes
• 7.3. Clinical Manifestations
• 7.4. Pathophysiology
• 7.5. Diagnosis
  • 7.5.1. Hematology
  • 7.5.2. Alpha/beta-globin chain synthesis
  • 7.5.3. Molecular analysis
• 7.6. Differential Diagnosis and Genetic Counseling
• 7.7 Treatment and Management of Alpha (a)-thalassemia
• 7.8 Guidelines for the Management of Transfusion Dependent Thalassemia (TDT) (Thalassemia International Federation)
• 7.9. Guidelines for the Management of Non-transfusion-dependent Thalassemia (NTDT)

7.10.Centers for Disease Control and Prevention (CDC)

8 Alpha Thalassemia Epidemiology and Patient Population

• 8.1 Key Findings
• 8.2 Total Diagnosed Prevalent Population of Alpha Thalassemia in the 7MM
• 8.3 Assumptions and Rationale
• 8.4 The United States
  • 8.4.1 Total Diagnosed Prevalent Population of Alpha Thalassemia in the United States
  • 8.4.2 Diagnosed Prevalent Cases of Alpha Thalassemia based on Transfusion Requirement in the United States
  • 8.4.3 Treatment Eligible Cases of Alpha Thalassemia in the United States (2018-2030) in the United States
• 8.5 EU4 and the UK
  • 8.5.1 Total Diagnosed Prevalent Population of Alpha Thalassemia in EU4 and the UK
  • 8.5.2 Diagnosed Prevalent Cases of Alpha Thalassemia based on Transfusion Requirement in EU4 and the UK
  • 8.5.3 Treatment-eligible Cases of Alpha Thalassemia in EU4 and the UK
• 8.6 Japan
  • 8.6.1 Total Diagnosed Prevalent Population of Alpha Thalassemia in Japan
  • 8.6.2 Diagnosed Prevalent Cases of Alpha Thalassemia Based on Transfusion Requirement in Japan
  • 8.6.3 Treatment eligible Cases of Alpha Thalassemia in Japan

9 Patient Journey

10 Alpha Thalassemia Emerging Therapies

• 10.1 Key Competitors
• 10.2 Mitapivat: Agios Pharmaceuticals
  • 10.2.1 Drug description
  • 10.2.2 Other development activities
  • 10.2.3 Clinical development
  • 10.2.4 Safety and Efficacy
• 10.3 . Etavopivat: Novo Nordisk
  • 10.3.1 Drug description
  • 10.3.2 Clinical development
  • 10.3.3 Safety and Efficacy

11 Alpha Thalassemia: Seven Major Market Analysis

• 11.1 Key Findings
• 11.2 Total Market Size of Alpha Thalassemia in the 7MM
• 11.3 Market Size of Alpha Thalassemia by Therapies in the 7MM
• 11.4 Market Outlook
• 11.5 Conjoint Analysis
• 11.6 Key Market Forecast Assumptions
• 11.7 United States Market Size
  • 11.7.1 Total Market Size of Alpha Thalassemia in the United States
  • 11.7.2 Market Size of Alpha Thalassemia by Therapies in the United States
• 11.8 E4 and the UK Market Size
  • 11.8.1 Total Market size of Alpha Thalassemia in EU4 and the UK
  • 11.8.2 Market Size of Alpha Thalassemia by Therapies in EU4 and the UK
• 11.9 Japan
  • 11.9.1 Total Market size of Alpha Thalassemia in Japan
  • 11.9.2 Market Size of Alpha Thalassemia by Therapies in Japan

12 Unmet Needs

13 SWOT Analysis

14 Reimbursement and Alpha Thalassemia Drugs Market Access

• 14.1 Reimbursement of Rare Diseases Therapies
• 14.2 United States Reimbursement Policies
• 14.3 Europe Reimbursement Policies
• 14.4 Japan reimbursement policies
• 14.5 Co-pay Program

15 Appendix

• 15.1 Bibliography
• 15.2 Report Methodology

16 DelveInsight Capabilities

17 Disclaimer

18 About DelveInsight