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Hemophilia A - Market Insights, Epidemiology, and Market Forecast - 2030

  • ID: 4911959
  • Drug Pipelines
  • February 2020
  • Region: Global
  • 239 pages
  • DelveInsight
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FEATURED COMPANIES

  • Alnylam Pharmaceuticals
  • Bayer
  • Catalyst Biosciences
  • LFB Biotechnologies
  • Opko Biologics
  • Roche
  • MORE
‘Hemophilia A - 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 Hemophilia A in the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.

Geography Covered
  • The United States
  • EU5 (Germany, France, Italy, Spain, and the United Kingdom)
  • Japan
Study Period: 2017-2030

Hemophilia A - Disease Understanding and Treatment Algorithm

Hemophilia A is an X chromosome-linked genetic disorder caused by the mutations in the genes for factor VIII (FVIII). This clotting factor is a part of the intrinsic pathway of blood coagulation. Without enough factor VIII, the blood cannot clot properly to control bleeding. It is mainly a defect in the gene on chromosome X, females have two copies of the X chromosome. So, if the factor VIII gene on one chromosome does not work, the gene on the other chromosome can do the job of making enough factor VIII. On the other hand, males have only one X chromosome; if the factor VIII gene is missing on the male X chromosome, he will have hemophilia A that is why the most people with Hemophilia A are male.

The symptoms of Hemophilia A can vary greatly from one person to another, it ranges from mild to moderate to severe. The age of onset and frequency of bleeding episodes depend upon the amount of factor VIII protein and overall clotting ability of the blood. In most individuals, regardless of severity, bleeding episodes tend to be more frequent in childhood and adolescence than in adulthood.

Additionally, the diagnosis of Hemophilia A depends on the identification of characteristic symptoms, a detailed patient history, a thorough clinical evaluation, and a variety of specialized laboratory tests. The identification of a hemizygous F8 pathogenic variant on molecular genetic testing in a male proband confirms the diagnosis.

The treatment of Hemophilia A is mainly focused on the prophylaxis as there is no cure for hemophilia A. Treatment consists of replacing the missing clotting protein (factor VIII) and preventing the complications associated with the disorder. Replacement of this protein may be obtained through recombinant factor VIII, which is artificially created in a lab. Several recombinant forms of factor VII are also approved for the treatment of hemophilia A. In some cases, subjects with hemophilia A may develop ‘inhibitors' against the replacement factor VIII. Inhibitors are antibodies, which are specialized proteins created by the body's immune system to combat foreign or invading substances such as toxins or bacteria. The immune system may recognize replacement factor VIII as ‘foreign' and create these antibodies (inhibitors), which target and destroy the replacement factor. For inhibitors treatment is mainly dependent on bypassing agents and immne tolerance therapy (ITI).

Hemophilia A Epidemiology

The disease epidemiology covered in the report provides historical as well as forecasted epidemiology [segmented by Total Prevalence of Hemophilia A, Diagnosed and Treated Prevalent Population of Hemophilia A, Severity-Specific Prevalence of Hemophilia A, and Prevalence of Hemophilia A with Inhibitors and Without Inhibitors] scenario of Hemophilia A in the 7MM Countries covering United States, EU5 countries (Germany, France, Italy, Spain, and United Kingdom), and Japan from 2017 to 2030.

The research analysts have assessed that the total prevalent population of Hemophilia A in the 7MM was 42,458 in 2017. In addition to this, it was accessed that the total diagnosed and treated prevalent population of Hemophilia A in the 7MM was assessed to be 38,212 in 2017.

Hemophilia A Drug Chapters

Hemophilia A can be characterized by immediate or delayed bleeding or prolonged oozing after injuries, tooth extractions, or surgery or renewed bleeding after initial bleeding has stopped. Prolonged or delayed bleeding or wound hematoma formation after surgery is common. After circumcision, males with hemophilia A of any severity may have prolonged oozing, or they may heal normally without treatment. In severe hemophilia A, spontaneous joint bleeding is the most frequent symptom.

The treatment of hemophilia A is based upon the severity of disease if the disease is mild and moderate then on-demand (episodic) treatment will be given, and if the disease is severe, then continuous prophylaxis treatment is given. Some individuals with severe hemophilia A may receive periodic factor VIII infusions at regular intervals to prevent bleeding episodes and associated complications, such as joint damage. This is referred to as prophylactic therapy.

The treatment landscape of Hemophilia A comprises of recombinant or plasma derived Factor VIII (long and short acting) as well as antibody for non-inhibitors patients and for inhibitors Hemophilia A patients the treatment is mainly comprises of by passing agents and ITI.

The US Food and Drug Administration (FDA) has approved several recombinant forms of factor VIII for the treatment of hemophilia A, including HelixateFS (CSL Behring); Recombinate (Baxter); Kogenate FS (Bayer HealthCare); Advate (Baxter); ReFacto (Pfizer); Eloctate (Biogen-Idec), and Xyntha (Pfizer). Human plasma-derived preparations include Monarc-M (Baxter), Monoclate-P (CSL Behring), Hemofil M (Baxter), Koate-DVI (Kedrion), Nuwiq (Octapharma), Adynovate (Baxalta), and Jivi (Bayer). Individuals with mild or moderate hemophilia A may be treated with replacement therapy as needed (i.e., to treat specific bleeding episodes). This is referred to as ‘on-demand' therapy.

Another breakthrough treatment for both Inhibitors and non-inhibitors patients is Hemlibra (Roche) which was approved by US FDA in 2017. It is a bispecific monoclonal antibody for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients ages newborn and older with hemophilia A.

Hemophilia A Market Outlook

The market size of Hemophilia A in seven major markets (7MM) is estimated to be USD 6,266 million in 2017. The United States accounts for the highest market size of Hemophilia A, i.e., USD 3,178 million, in comparison to the other major markets i.e., EU5 countries (Germany, France, Italy, Spain, and the United Kingdom), and Japan. Among the EU5 countries, the United Kingdom had the highest market size in 2017, while Spain had the lowest market size of Hemophilia A in 2017.

Hemophilia A Drugs Uptake

With the launch of emerging therapies like Valoctocogene Roxaparvovec (BMN 270, a product of Biomarin Pharmaceutical), which is a gene therapy based on the use of adeno-associated virus (AAV) vectors, Concizumab (NN7415, a product of Novo Nordisk), Fitusiran (Sanofi Genzyme/ Alnylam Pharmaceuticals), BIVV001 (Sanofi), Marstacimab (Pfizer), SPK-8011 (Spark Therapeutics) and others, the market of Hemophilia A is anticipated to change during the forecast period (2019-2030).

Products that are anticipated to be launched during the forecast period are in late clinical stages of development, while others are in ongoing late clinical development stages. Some of the above-mentioned drug candidates have shown very promising results and it has been anticipated by analysts that completion of clinical development and launch of these products in the market, might increase the market share of these companies, and besides this, patients of Hemophilia A will have better management practices.

According to our assessment, potential emerging candidates shall launch in the upcoming years of the forecast period [2019-2030], and with their anticipated launch, the market size of Hemophilia A will also experience significant growth.

Hemophilia A Report Insights
  • Total Prevalence of Hemophilia A
  • Diagnosed and Treated Prevalent Population of Hemophilia A
  • Severity-Specific Prevalence of Hemophilia A
  • Prevalence of Hemophilia A with Inhibitors and without Inhibitors
  • Therapeutic Approaches
  • Pipeline Analysis
  • Market Size and Trends
  • Market Opportunities
  • Impact of upcoming Therapies
Hemophilia A Report Key Strengths
  • 12-Year Plus Forecast
  • 7MM Coverage
  • Epidemiology Segmentation
  • Drugs Uptake
  • Highly Analyzed Market
  • Key Cross Competition
Hemophilia A Report Assessment
  • Current Treatment Practices
  • Unmet Needs
  • Detailed Pipeline Product Profiles
  • Market Attractiveness
  • Market Drivers and Barriers
Key Benefits
  • This report will help to develop Business Strategies by understanding the trends shaping and driving Hemophilia A market
  • Organize sales and marketing efforts by identifying the best opportunities for Hemophilia A market
  • To understand the future market competition in the Hemophilia A market.
Note: Product cover images may vary from those shown
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FEATURED COMPANIES

  • Alnylam Pharmaceuticals
  • Bayer
  • Catalyst Biosciences
  • LFB Biotechnologies
  • Opko Biologics
  • Roche
  • MORE
1. Key Insights

2. Executive Summary

3. Hemophilia A: Market Overview at a Glance
3.1. Total Market Share (%) Distribution of Hemophilia A in 2017
3.2. Total Market Share (%) Distribution of Hemophilia A in 2030

4. Hemophilia A: Disease Background and Overview
4.1. Introduction
4.1.1. Sign and Symptoms of Hemophilia A
4.1.2. Inheritance Pattern of Hemophilia A
4.1.3. Molecular Pathogenesis of Hemophilia A
4.1.4. Pathophysiology of Hemophilia A
4.1.5. Risk Factors of Hemophilia A
4.2. Diagnosis of Hemophilia A
4.2.1. Establishing the Diagnosis
4.2.2. Molecular Genetic Testing
4.2.3. Screening Tests
4.2.4. Clotting Factor Tests
4.2.5. Inhibitor Testing

5. Epidemiology and Patient Population
5.1. Epidemiology Key Findings
5.2. Assumptions and Rationale: 7MM
5.3. Epidemiology Scenario: 7MM
5.3.1. Total Prevalence of Hemophilia A in the 7MM
5.3.2. Diagnosed and Treated Prevalent Population of Hemophilia A in the 7MM
5.3.3. Severity- Specific Prevalence of Hemophilia A in the 7MM
5.3.4. Prevalence of Hemophilia A with or without Inhibitors the 7MM

6. United States Epidemiology
6.1. Total Prevalence of Hemophilia A in the United States
6.2. Diagnosed and Treated Prevalent Population of Hemophilia A in the United States
6.3. Severity- Specific Prevalence of Hemophilia A in the United States
6.4. Prevalence of Hemophilia A with Inhibitors and Without Inhibitors in the United States

7. EU-5 Country-wise Epidemiology
7.1. Germany Epidemiology
7.1.1. Total Prevalence of Hemophilia A in Germany
7.1.2. Diagnosed and Treated Prevalent Population of Hemophilia A in Germany
7.1.3. Severity- Specific Prevalence of Hemophilia A in Germany
7.1.4. Prevalence of Hemophilia A with or without Inhibitors in Germany
7.2. France Epidemiology
7.2.1. Total Prevalence of Hemophilia A in France
7.2.2. Diagnosed and Treated Prevalent Population of Hemophilia A in France
7.2.3. Severity- Specific Prevalence of Hemophilia A in France
7.2.4. Prevalence of Hemophilia A with or without Inhibitors in France
7.3. Italy Epidemiology
7.3.1. Total Prevalence of Hemophilia A and B in Italy
7.3.2. Diagnosed and Treated Prevalent Population of Hemophilia A in Italy
7.3.3. Severity-Specific Prevalence of Hemophilia A in Italy
7.3.4. Prevalence of Hemophilia A with or without Inhibitors in Italy
7.4. Spain Epidemiology
7.4.1. Total Prevalence of Hemophilia A in Spain
7.4.2. Diagnosed Prevalent Population of Hemophilia A in Spain
7.4.3. Severity- Specific Prevalence of Hemophilia A in Spain
7.4.4. Prevalence of Hemophilia A with Inhibitors or Without-Inhibitors in Spain
7.5. United Kingdom Epidemiology
7.5.1. Total Prevalence of Hemophilia A in the United Kingdom
7.5.2. Diagnosed and Treated Prevalent Population of Hemophilia A in the United Kingdom
7.5.3. Severity- Specific Prevalence of Hemophilia A in the United Kingdom
7.5.4. Prevalence of Hemophilia A with Inhibitors or Non-Inhibitors in the United Kingdom

8. Japan Epidemiology
8.1. Total Prevalence of Hemophilia A in Japan
8.2. Diagnosed and Treated Prevalent Population of Hemophilia A in Japan
8.3. Severity- Specific Prevalence of Hemophilia A in Japan
8.4. Prevalence of Hemophilia A with Inhibitors and Without Inhibitors in Japan

9. Treatment Algorithm, Current Treatment, and Medical Practices
9.1. Treatment and Management of Hemophilia A
9.1.1. Algorithm for treatment of Hemophilia A
9.2. Principles of care of Hemophilia A
9.3. Clotting Factors Concentrates
9.4. Prevention of Primary Manifestations
9.5. Prevention of Secondary Complications
9.6. Agents/Circumstances to Avoid
9.7. Inhibitors
9.8. Pregnancy Management

10. Unmet Needs

11. Marketed Products
11.1. List of Marketed Products in the 7MM
11.2. Esperoct (N8-GP; Turoctocog alfa pegol): Novo Nordisk
11.2.1. Product Description
11.2.2. Regulatory Milestones
11.2.3. Other Developmental Activities
11.2.4. Pivotal Clinical Trials
11.3. Jivi (formerly BAY94-9027): Bayer
11.3.1. Drug Description
11.3.2. Regulatory Milestones
11.3.3. Other Developmental Activities
11.3.4. Pivotal Clinical Trial
11.3.5. Summary of Pivotal Clinical Trial
11.4. Hemlibra (Emicizumab-kxwh): Chugai/ Genentech/Roche
11.4.1. Product Description
11.4.2. Regulatory Milestones
11.4.3. Other Developmental Activities
11.4.4. Pivotal Clinical Trials
11.5. Wilate: Octapharma
11.5.1. Product Description
11.5.2. Regulatory Milestones
11.5.3. Pivotal Clinical Trials
11.6. Adynovate (Adynovi; BAX 855): Takeda
11.6.1. Product Description
11.6.2. Regulatory Milestones
11.6.3. Other Developmental Activities
11.6.4. Pivotal Clinical Trials
11.7. Eloctate [Antihemophilic Factor (Recombinant), Fc Fusion Protein; Elocta (efmoroctocog alfa)]: Sanofi/Sobi
11.7.1. Product Description
11.7.2. Regulatory Milestones
11.7.3. Other Developmental Activities
11.7.4. Pivotal Clinical Trials
11.8. Afstyla (Lonoctocog alfa): CSL Behring
11.8.1. Product Description
11.8.2. Regulatory Milestones
11.8.3. Other Developmental Activities
11.8.4. Pivotal Clinical Trials
11.9. Kovaltry (BAY 81-8973): Bayer
11.9.1. Product Description
11.9.2. Regulatory Milestones
11.9.3. Other Developmental Activities
11.9.4. Pivotal Clinical Trials
11.10. Nuwiq (simoctocog alfa): Octapharma
11.10.1. Product Description
11.10.2. Regulatory Milestones
11.10.3. Pivotal Clinical Trials
11.11. NovoEight (Turoctocog alfa): Novo Nordisk
11.11.1. Product Description
11.11.2. Regulatory Milestones
11.11.3. Other Developmental Activities
11.11.4. Pivotal Clinical Trials
11.12. Obizur: Takeda
11.12.1. Product Description
11.12.2. Regulatory Milestones
11.12.3. Other Developmental Activities
11.12.4. Pivotal Clinical Trials
11.13. Kogenate FS (octocog alfa): Bayer
11.13.1. Product Description
11.13.2. Regulatory Milestones
11.13.3. Pivotal Clinical Trials
11.14. Xyntha (ReFacto AF): Pfizer
11.14.1. Product Description
11.14.2. Regulatory Milestones
11.14.3. Other Developmental Activities
11.14.4. Pivotal Clinical Trials
11.15. Feiba: Takeda
11.15.1. Product Description
11.15.2. Regulatory Milestones
11.15.3. Other Developmental Activities
11.15.4. Pivotal Clinical Trials

12. Emerging Therapies
12.1. Key Cross
12.2. Valoctocogene Roxaparvovec (BMN 270): BioMarin Pharmaceutical
12.2.1. Product Description
12.2.2. Other Developmental Activities
12.2.3. Clinical Development
12.2.4. Safety and Efficacy
12.3. LR769: HEMA Biologics/LFB Biotechnologies
12.3.1. Product Description
12.3.2. Other Developmental Activities
12.3.3. Clinical Development
12.3.4. Safety and Efficacy
12.4. Concizumab (NN7415): Novo Nordisk
12.4.1. Product Description
12.4.2. Other Developmental Activities
12.4.3. Clinical Development
12.4.4. Safety and Efficacy
12.5. Fitusiran (ALN-AT3, SAR-439774): Sanofi Genzyme/ Alnylam Pharmaceuticals
12.5.1. Product Description
12.5.2. Other Developmental Activities
12.5.3. Clinical Development
12.5.4. Safety and Efficacy
12.6. BIVV001 (rFVIIIFc-VWF-XTEN): Sanofi
12.6.1. Product Description
12.6.2. Other Developmental Activities
12.6.3. Clinical Development
12.6.4. Safety and Efficacy
12.7. Marstacimab (PF-06741086): Pfizer
12.7.1. Product Description
12.7.2. Other Developmental Activities
12.7.3. Clinical Development
12.7.4. Safety and Efficacy
12.8. Marzeptacog alfa (Activated): Catalyst Biosciences
12.8.1. Product Description
12.8.2. Other Developmental Activities
12.8.3. Clinical Development
12.8.4. Safety and Efficacy
12.9. OPK88005: OPKO Biologics
12.9.1. Product Description
12.9.2. Other Developmental Activities
12.9.3. Clinical Development
12.10. SPK-8011: Spark Therapeutics
12.10.1. Product Description
12.10.2. Other Developmental Activities
12.10.3. Clinical Development
12.10.4. Safety and Efficacy

13. Hemophilia A: Seven Major Market Analysis
13.1. Key Findings
13.2. Market Size of Hemophilia A in 7MM
13.3. Market Size of Hemophilia A by Therapies in the 7MM

14. 7MM: Market Outlook

15. United States: Market Size
15.1. Total Market size of Hemophilia A in the United States
15.2. Market Size of Hemophilia A by Therapies in the US

16. EU-5 countries: Market Size and Outlook
16.1. Germany Market Size
16.1.1. Total Market size of Hemophilia A in Germany
16.1.2. Market Size of Hemophilia A by therapies in Germany
16.2. France Market Size
16.2.1. Total Market size of Hemophilia A in France
16.2.2. Market Size of Hemophilia A by therapies in France
16.3. Italy Market Size
16.3.1. Total Market size of Hemophilia A in Italy
16.3.2. Market Size of Hemophilia A by therapies in Italy
16.4. Spain Market Size
16.4.1. Total Market size of Hemophilia A in Spain
16.4.2. Market Size of Hemophilia A by therapies in Spain
16.5. United Kingdom Market Size
16.5.1. Total Market size of Hemophilia A in the United Kingdom
16.5.2. Market Size of Hemophilia A by therapies in the UK

17. Japan Market Outlook
17.1. Japan Market Size
17.1.1. Total Market size of Hemophilia A in Japan
17.1.2. Market Size of Hemophilia A by therapies in Japan

18. Market Drivers

19. Market Barriers

20. Appendix
20.1. Report Methodology

21. Publisher Capabilities

22. Disclaimer

23. About the Publisher
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  • Novo Nordisk
  • Shire
  • Sanofi
  • Bayer
  • Roche
  • Pfizer
  • Biomarin Pharmaceutical
  • Alnylam Pharmaceuticals
  • HEMA Biologics
  • LFB Biotechnologies
  • Catalyst Biosciences
  • Opko Biologics
  • Spark Therapeutics
  • Sangamo Therapeutics
  • ApcinteX Ltd
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