Table of Content

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. DelveInsight Capabilities
22. Disclaimer
23. About DelveInsight



List of Figures


Figure 1 Coagulation Cascade and the Effects of Recombinant Activated Factor Vll (rFVlla)
Figure 2 Inheritance Pattern of Hemophilia A
Figure 3 Established and Proposed Risk Factors for Inhibitor Development
Figure 4 Screening Tests for Hemophilia A
Figure 5 Total Prevalence of Hemophilia A in the 7MM (2017–2030)
Figure 6 Diagnosed and Treated Prevalent Population of Hemophilia A in the 7MM (2017–2030)
Figure 7 Total Prevalence of Hemophilia A in the 7MM (2017–2030)
Figure 8 Prevalence of Hemophilia A with or without Inhibitors in the 7MM (2017–2030)
Figure 9 Total Prevalent Population of Hemophilia A in the United States (2017–2030)
Figure 10 Diagnosed and Treated Prevalent Population of Hemophilia A in the US (2017–2030)
Figure 11 Severity- Specific Prevalence of Hemophilia A in the US (2017–2030)
Figure 12 Prevalence of Hemophilia A with Inhibitors and Without Inhibitors in the United States (2017–2030)
Figure 13 Total Prevalence of Hemophilia A in Germany (2017–2030)
Figure 14 Diagnosed and treated Prevalent Population of Hemophilia A in Germany (2017–2030)
Figure 15 Severity- Specific Prevalence of Hemophilia A in Germany (2017–2030)
Figure 16 Prevalence of Hemophilia A with or Non-Inhibitors in Germany (2017–2030)
Figure 17 Total Prevalence of Hemophilia A in France (2017–2030)
Figure 18 Diagnosed and Treated Prevalent Population of Hemophilia A in France (2017–2030)
Figure 19 Severity-Specific Prevalence of Hemophilia A in France (2017–2030)
Figure 20 Diagnosed Prevalence of Hemophilia A with or Non-Inhibitors in France (2017–2030)
Figure 21 Total Prevalence of Hemophilia A and B in Italy (2017–2030)
Figure 22 Diagnosed and Treated Prevalent Population of Hemophilia A in Italy (2017–2030)
Figure 23 Severity- Specific Prevalence of Hemophilia A in Italy (2017–2030)
Figure 24 Prevalence of Hemophilia A with or Non-Inhibitors in Italy (2017–2030)
Figure 25 Total Prevalence of Hemophilia A and B in Spain (2017–2030)
Figure 26 Diagnosed Prevalent Population of Hemophilia A in Spain (2017–2030)
Figure 27 Severity- Specific Prevalence of Hemophilia A in Spain (2017–2030)
Figure 28 Prevalence of Hemophilia A with or Non-Inhibitors in Spain (2017–2030)
Figure 29 Total Prevalence of Hemophilia A and B in the United Kingdom (2017–2030)
Figure 30 Diagnosed and Treated Prevalent Population of Hemophilia A in the United Kingdom (2017–2030)
Figure 31 Severity- Specific Prevalence of Hemophilia A in the United Kingdom (2017–2030)
Figure 32 Prevalence of Hemophilia A with or Non-Inhibitors in the United Kingdom (2017–2030)
Figure 33 Total Prevalent Population of Hemophilia A in Japan (2017–2030)
Figure 34 Diagnosed and Treated Patient Population of Hemophilia A in Japan (2017–2030)
Figure 35 Severity- Specific Prevalence of Hemophilia A in Japan (2017–2030)
Figure 36 Prevalence of Hemophilia A with Inhibitors and Without Inhibitors in Japan (2017–2030)
Figure 37 Treatment Algorithm for the patient with Hemophilia A
Figure 38 Management Algorithm for the patient with Inhibitors
Figure 39 Improvements in Hemophilia Therapy
Figure 40 Unmet Needs of Hemophilia A
Figure 41 Market Size of Hemophilia A in the 7MM in USD Million (2017–2030)
Figure 42 Market Size of Hemophilia A by therapies (Inhibitors)in the 7MM, in USD Million (2017–2030)
Figure 43 Market Size of Hemophilia A by therapies (Non-Inhibitors) in the 7MM, in USD Million (2017–2030)
Figure 44 Market Size of Hemophilia A in the US, USD Millions (2017–2030)
Figure 45 Market Size of Hemophilia A by therapies (Inhibitors) in the United States, in USD Million (2017–2030)
Figure 46 Market Size of Hemophilia A by therapies (Non-Inhibitors) the United States, in USD Million (2017–2030)
Figure 47 Market Size of Hemophilia A in Germany, USD Millions (2017–2030)
Figure 48 Market Size of Hemophilia A by therapies (Inhibitors) in Germany, in USD Million (2017–2030)
Figure 49 Market Size of Hemophilia A by therapies (Non-Inhibitors) in Germany, in USD Million (2017–2030)
Figure 50 Market Size of Hemophilia A in France, USD Millions (2017–2030)
Figure 51 Market Size of Hemophilia A by therapies (Inhibitors) in France, in USD Million (2017–2030)
Figure 52 Market Size of Hemophilia A by therapies (Non-Inhibitors) in France, in USD Million (2017–2030)
Figure 53 Market Size of Hemophilia A in Italy, USD Millions (2017–2030)
Figure 54 Market Size of Hemophilia A by therapies (Inhibitors) in Italy, in USD Million (2017–2030)
Figure 55 Market Size of Hemophilia A by therapies (Non-Inhibitors) in Italy, in USD Million (2017–2030)
Figure 56 Market Size of Hemophilia A in Spain, USD Millions (2017–2030)
Figure 57 Market Size of Hemophilia A by therapies (Inhibitors) in Spain, in USD Million (2017–2030)
Figure 58 Market Size of Hemophilia A by therapies (Non-Inhibitors) in Spain, in USD Million (2017–2030)
Figure 59 Market Size of Hemophilia A in the UK, USD Millions (2017–2030)
Figure 60 Market Size of Hemophilia A by therapies (Inhibitors) in the UK, in USD Million (2017–2030)
Figure 61 Market Size of Hemophilia A by therapies (Non-Inhibitors) in the UK, in USD Million (2017–2030)
Figure 62 Market Size of Hemophilia A in Japan, USD Millions (2017–2030)
Figure 63 Market Size of Hemophilia A by therapies (Inhibitors) in Japan, in USD Million (2017–2030)
Figure 64 Market Size of Hemophilia A by therapies (Non-Inhibitors) in Japan, in USD Million (2017–2030)
Figure 65 Market Drivers of Hemophilia A
Figure 66 Market Barriers of Hemophilia A


List of Tables


Table 1 Summary of Hemophilia A Market, Epidemiology, and Key Events (2017–2030)
Table 2 Approximate Frequency of Bleeding at Different Sites
Table 3 Sites of Bleeding In Hemophilia
Table 4 Levels of Factors in Hemophilia A
Table 5 Summary of sources explored for the epidemiology of hemophilia A
Table 6 Total Prevalence of HemophiliaA in the 7MM (2017–2030)
Table 7 Diagnosed and Treated Prevalent Population of Hemophilia A in the 7MM (2017–2030)
Table 8 Severity- Specific Prevalence of Hemophilia A in the 7MM (2017–2030)
Table 9 Prevalence of Hemophilia A with or without Inhibitors the 7MM (2017–2030)
Table 10 Total Prevalence of Hemophilia A in the United States (2017–2030)
Table 11 Diagnosed and Treated Prevalent Population of Hemophilia A in the US (2017–2030)
Table 12 Severity- Specific Prevalence of Hemophilia A in the US (2017–2030)
Table 13 Prevalence of Hemophilia A with Inhibitors and Without Inhibitors in the United States (2017–2030)
Table 14 Total Prevalence of Hemophilia A in Germany (2017–2030)
Table 15 Diagnosed and Treated Prevalent Population of Hemophilia A in Germany (2017–2030)
Table 16 Severity- Specific Prevalence of Hemophilia A in Germany (2017–2030)
Table 17 Diagnosed Prevalence of Hemophilia A with or Non-Inhibitors in Germany (2017–2030)
Table 18 Total Prevalence of Hemophilia A in France (2017–2030)
Table 19 Diagnosed and Treated Prevalent Population of Hemophilia A in France (2017–2030)
Table 20 Severity- Specific Prevalence of Hemophilia A in France (2017–2030)
Table 21 Diagnosed Prevalence of Hemophilia A with or Non-Inhibitors in France (2017–2030)
Table 22 Total Prevalence of Hemophilia A in Italy (2017–2030)
Table 23 Diagnosed and Treated Prevalent Population of Hemophilia A in Italy (2017–2030)
Table 24 Severity-Specific Diagnosed Prevalence of Hemophilia A in Italy (2017–2030)
Table 25 Prevalence of Hemophilia A with or Non-Inhibitors in Italy (2017–2030)
Table 26 Total Prevalence of Hemophilia A in the Spain (2017–2030)
Table 27 Diagnosed and Treated Prevalent Population of Hemophilia A in Spain (2017–2030)
Table 28 Severity-Specific Prevalence of Hemophilia A in Spain (2017–2030)
Table 29 Diagnosed Prevalence of Hemophilia A with or Non-Inhibitors in Spain (2017–2030)
Table 30 Total Prevalence of Hemophilia A and B in the United Kingdom (2017–2030)
Table 31 Diagnosed and Treated Prevalent Population of Hemophilia A in the United Kingdom (2017–2030)
Table 32 Severity-Specific Prevalence of Hemophilia A in the United Kingdom (2017–2030)
Table 33 Prevalence of Hemophilia A with or Non-Inhibitors in the United Kingdom (2017–2030)
Table 34 Total Prevalence of Hemophilia A in Japan (2017–2030)
Table 35 Diagnosed and Treated Prevalent Population of Hemophilia A in Japan (2017–2030)
Table 36 Severity- Specific Prevalence of Hemophilia A in Japan (2017–2030)
Table 37 Prevalence of Hemophilia A with Inhibitors and Without Inhibitors in Japan (2017–2030)
Table 38 Jivi, Clinical Trial Description, 2019
Table 39 Emerging Drugs key-Cross
Table 40 Valoctocogene Roxaparvovec (BMN 270), Clinical Trial Description, 2019
Table 41 LR769 (Eptacogum Beta Activated), Clinical Trial Description, 2019
Table 42 Concizumab, Clinical Trial Description, 2019
Table 43 Fitusiran, Clinical Trial Description, 2019
Table 44 BIVV001, Clinical Trial Description, 2019
Table 45 Marstacimab (PF-06741086), Clinical Trial Description, 2019
Table 46 Marzeptacog alfa (Activated), Clinical Trial Description, 2019
Table 47 OPK88005, Clinical Trial Description, 2019
Table 48 SPK-8011 , Clinical Trial Description, 2019
Table 49 Market Size of Hemophilia A in the 7MM in USD Million (2017–2030)
Table 50 The US Market Size of Hemophilia A in USD Million (2017–2030)
Table 51 Market Size of Hemophilia A by therapies (Inhibitors), in the United States, in USD Million (2017–2030)
Table 52 Market Size of Hemophilia A by therapies (Non-Inhibitors) in the United States, in USD Million (2017–2030)
Table 53 Germany Market Size of Hemophilia A in USD Million (2017–2030)
Table 54 Market Size of Hemophilia A by therapies (Inhibitors) in Germany, in USD Million (2017–2030)
Table 55 Market Size of Hemophilia A by therapies (Non-Inhibitors) in France, in USD Million (2017–2030)
Table 56 Market Size in USD Million (2017–2030)
Table 57 Market Size of Hemophilia A by therapies (Inhibitors) in France, in USD Million (2017–2030)
Table 58 Market Size of Hemophilia A by therapies (Non-Inhibitors) in France, in USD Million (2017–2030)
Table 59 Market Size in USD Million (2017–2030)
Table 60 Market Size of Hemophilia A by therapies (Inhibitors) in Italy, in USD Million (2017–2030)
Table 61 Market Size of Hemophilia A by therapies (Non-Inhibitors) in Italy, in USD Million (2017–2030)
Table 62 Market Size in USD Million (2017–2030)
Table 63 Market Size of Hemophilia A by therapies (Inhibitors) in Spain, in USD Million (2017–2030)
Table 64 Market Size of Hemophilia A by therapies (Non-Inhibitors) in the Spain, in USD Million (2017–2030)
Table 65 Market Size in USD Million (2017–2030)
Table 66 Market Size of Hemophilia A by therapies (Inhibitors) in the UK, in USD Million (2017–2030)
Table 67 Market Size of Hemophilia A by therapies (Non-Inhibitors) in the UK, in USD Million (2017–2030)
Table 68 Japan Market Size of Hemophilia A in USD Million (2017–2030)
Table 69 Market Size of Hemophilia A by therapies (Inhibitors) in Japan, in USD Million (2017–2030)
Table 70 Market Size of Hemophilia A by therapies (Non-Inhibitors) in Japan, in USD Million (2017–2030)