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Hemophilia A and B Therapeutics in Asia-Pacific Markets to 2023 - Growth Driven by Increasing Treatment-Receiving Pool, Launch of Long-Acting Replacement Therapies and Non-factor Therapy

Report Code : gbi0023
Published Date : 01 November, 2017 | No of Pages: 133

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Hemophilia A and B Therapeutics in Asia-Pacific Markets to 2023 - Growth Driven by Increasing Treatment-Receiving Pool, Launch of Long-Acting Replacement Therapies and Non-factor Therapy

Summary

Hemophilia is a rare disease affecting more than 400,000 people worldwide. Hemophilia A is four to six times as common as hemophilia B, and more than half of hemophilia A patients have a severe form of hemophilia. The mainstay of treatment for hemophilia is to replace the missing factor VIII or factor IX, produced using plasma-derived or recombinant methods. Some patients develop inhibitors to factor VIII or factor IX treatment and need a bypassing agent, such as factor VIIa, to resolve bleeds. Patients with hemophilia A or B often require lifelong treatment to prevent and control bleeding episodes, and they therefore represent a strong market opportunity. The current marketed products landscape comprises replacement factor therapies, including recombinant therapies and new long-acting recombinant products. The pipeline shows strong promise, and highlights a gradual shift from short-acting recombinant factor VIII or factor IX to long-acting recombinant factor VIII, and from factor replacement therapies to non-factor therapies. Nevertheless, significant unmet need remains for products that can avoid the development of inhibitors. Although the late-stage pipeline for hemophilia treatment seems promising, the main market restraint is likely to be low penetration of new long-acting recombinant therapies - particularly in India and China - owing to their high expected prices.

Scope

The Asia-Pacific hemophilia A market will be valued at $1,499.2m in 2023, growing from $838.9m in 2016, at a compound annual growth rate of 8.6%.
- How will the market value of hemophilia B compare?
- How will non-factor therapies contribute to growth?
- What will be the impact of the new long-acting recombinant factor VIII and recombinant factor IX products?
The pipeline contains a range of molecule types and molecular targets, including both those that are well established in hemophilia A and B, and novel targeted therapies.
- Which molecular targets appear most frequently in the pipeline?
- Will the pipeline address unmet needs such as less frequently administered therapies and risk of inhibitor development for hemophilia A and B patients?
- What are the most promising first-in-class targets? 
- Will the current first-in-class targets have broader therapeutic potential across the Asia-Pacific markets? 
Various drivers and barriers will influence the market over the forecast period.
- What are the barriers that will limit the uptake of premium-priced recombinant factor VIII and recombinant factor IX therapies in the assessed countries?
- Which factors are most likely to drive the market in these countries?
Co-development deals are the most common form of strategic alliance in hemophilia A and B, with deal values ranging from under $10m to over $280m.
- How do deal frequency and value compare between target families and molecule types?
- What were the terms and conditions of key co-development deals?

Reasons to buy

- Understand the current clinical and commercial landscape by considering disease pathogenesis, diagnosis, prognosis, and the treatment options available at each stage of diagnosis, including a clinical comparison of marketed therapies.
- Visualize the composition of the hemophilia A and B market in terms of the dominant therapies for each patient subset, along with their clinical and commercial standing. Unmet needs are highlighted to allow a competitive understanding of gaps in the market.
- Analyze the hemophilia A and B pipeline and stratify pipeline therapies by stage of development, molecule type and molecular target. There are promising signs in the pipeline that the industry is seeking novel treatment approaches.
- Understand the potential of late-stage therapies, with extensive profiles of products that could enter the market over the forecast period, highlighting clinical performance, potential commercial positioning, and how they will compete with other therapies.
- Anticipate levels of hemophilia A and B market growth in the five Asia-Pacific markets, with epidemiological and annual cost of therapy forecasts across India, China, Australia, South Korea and Japan, as well as coverage of the individual contributions of promising late-stage molecules to market growth.
- Identify commercial opportunities in the hemophilia A and B deals landscape by analyzing trends in licensing and co-development deals.
1 Table of Contents
1 Table of Contents 4
1.1 List of Tables 7
1.2 List of Figures 8
2 Introduction 10
2.1 Disease Introduction 10
2.2 Epidemiology 11
2.3 Symptoms 11
2.4 Etiology and Pathophysiology 12
2.4.1 Hemophilia A 12
2.4.2 Hemophilia B 13
2.5 Diagnosis 13
2.6 Diagnosis of Inhibitors 13
2.7 Disease stages 14
2.8 Prognosis 14
2.9 Treatment Options 14
2.9.1 Management of Bleeding with Factor Replacement Therapy 15
2.9.2 Antifibrinolytic Medication 17
2.9.3 Treatment of Inhibitors 17
2.9.4 Gene Therapy 17
2.9.5 Hemophilia A 18
2.9.6 Hemophilia B 18
3 Marketed Products 20
3.1 Overview 20
3.2 Marketed Products - Hemophilia A 20
3.2.1 Advate (octacog alfa) - Shire 20
3.2.2 Adynovate (rurioctocog alfa pegol) - Shire 22
3.2.3 Xyntha/ReFacto AF (moroctocog alfa) - Pfizer 23
3.2.4 Kogenate FS (octacog alfa) - Bayer 24
3.2.5 Kovaltry (octacog alfa) - Bayer 25
3.2.6 Eloctate (efmoroctocog alfa) - Bioverativ 27
3.2.7 Nuwiq (simoctocog alfa) - Octapharma 29
3.2.8 NovoEight (turoctocog alfa) - Novo Nordisk 30
3.2.9 GreenGene F (beroctocog alfa) - Green Cross 31
3.2.10 Obizur (susoctocog alfa) - Shire 32
3.2.11 Afstyla (lonoctocog alfa) - CSL Behring 33
3.3 Marketed Products - Hemophilia B 34
3.3.1 Alprolix (eftrenonacog alfa) - Bioverativ 34
3.3.2 BeneFix (nonacog alfa) - Pfizer 36
3.3.3 Rixubis (nonacog gamma) - Shire 37
3.3.4 Idelvion (albutrepenonacog alfa) - CSL Behring 38
3.4 Marketed Products - Hemophilia A and B 40
3.4.1 Feiba NF (anti-inhibitor coagulant complex) - Baxter 40
3.4.2 Byclot (freeze-dried-activated human blood coagulation factor VII concentrate containing factor X) - Kaketsuken 40
3.5 Comparative Efficacy and Safety of Marketed Products 41
4 Pipeline Analysis 44
4.1 Overview 44
4.2 Pipeline by Stage of Development, Molecule Type, Route of Administration and Program Type 44
4.3 Pipeline by Molecular Target 45
4.4 Promising Pipeline Candidates - Hemophilia A 47
4.4.1 Emicizumab (ACE-910/RG6013) - Roche and Chugai Pharma 47
4.4.2 N8-GP (turoctocog alfa pegol) - Novo Nordisk 50
4.4.3 BAY94-9027 (damoctocog alfa pegol) - Bayer 52
4.5 Promising Pipeline Candidates -Hemophilia B 54
4.5.1 N9-GP/NN-7999 (nonacog beta pegol) - Novo Nordisk 54
4.5.2 Ixinity (trenonacog alfa) - Aptevo Therapeutics 56
4.6 Promising Pipeline Candidates - Hemophilia A and B 57
4.6.1 CSL-689 (recombinant activated VII-FP) - CSL Behring 57
4.7 Comparative Efficacy and Safety of Pipeline Products 59
4.8 Product Competitiveness Framework 62
5 Clinical Trial Analysis 65
5.1 Failure Rate 65
5.1.1 Overall Failure Rate 65
5.1.2 Failure Rate by Phase and Molecule Type 67
5.1.3 Failure Rate by Phase and Molecular Target 67
5.2 Clinical Trial Size 68
5.2.1 Patient Enrollment per Product by Molecule Type and Stage of Development 68
5.2.2 Patient Enrollment per Product by Molecular Target and Stage of Development 69
5.2.3 Patient Enrollment per Trial by Molecule Type, Molecular Target and Stage of Development 70
5.2.4 Patient Enrollment per Trial by Molecular Target and Stage of Development 71
5.3 Clinical Trial Duration 72
5.3.1 Clinical Trial Duration by Molecule Type 72
5.3.2 Clinical Trial Duration by Molecular Target 73
5.4 Competitive Clinical Trials Metrics Analysis 74
6 Multi-scenario Forecast 76
6.1 Geographical Markets 76
6.2 Asia-Pacific Market 76
6.3 India 79
6.3.1 Treatment Usage Patterns 79
6.3.2 Annual Cost of Therapy 80
6.3.3 Market Size 82
6.4 China 83
6.4.1 Treatment Usage Patterns 83
6.4.2 Annual Cost of Therapy 84
6.4.3 Market Size 86
6.5 Australia 87
6.5.1 Treatment Usage Patterns 87
6.5.2 Annual Cost of Therapy 88
6.5.3 Market Size 89
6.6 South Korea 90
6.6.1 Treatment Usage Patterns 90
6.6.2 Annual Cost of Therapy 91
6.6.3 Market Size 92
6.7 Japan 94
6.7.1 Treatment Usage Patterns 94
6.7.2 Annual Cost of Therapy 95
6.7.3 Market Size 96
7 Drivers and Barriers 98
7.1 Drivers 98
7.1.1 Increasing Awareness 98
7.1.2 Promising Late-Stage Pipeline Products with Potential to Change Treatment Paradigm 98
7.1.3 Continued Uptake of Recently Approved Therapies 98
7.1.4 Increasing Usage of Prophylactic Therapies 99
7.2 Barriers 99
7.2.1 Low Diagnosis and Low Treatment Rate 99
7.2.2 Low Access to Factor Replacement Therapies 99
7.2.3 Limitation of Premium Pricing 99
8 Deals and Strategic Consolidations 100
8.1 Licensing Deals 100
8.1.1 Deals by Region and Value 100
8.1.2 Number of Disclosed and Undisclosed Deals by Year, Aggregate Deal Value 101
8.1.3 Deal Value by Stage of Development, Molecule Type, and Molecular Target 101
8.1.4 Key Licensing Deals 104
8.2 Co-development Deals 106
8.2.1 Deals by Region and Value 106
8.2.2 Number of Disclosed and Undisclosed Deals by Year, Aggregate Deal Value 107
8.2.3 Deal Value by Stage of Development, Molecule Type, and Molecular Target 108
8.2.4 Key Co-development Deals 110
9 Appendix 112
9.1 All Pipeline Drugs by Stage of Development 112
9.1.1 Discovery 112
9.1.2 Preclinical 112
9.1.3 IND/CTA-filed 113
9.1.4 Phase I 114
9.1.5 Phase II 114
9.1.6 Phase III 115
9.1.7 Pre-registration 115
9.2 Market Forecasts to 2023 115
9.2.1 Asia-Pacific 115
9.2.2 India 116
9.2.3 China 117
9.2.4 Australia 117
9.2.5 South Korea 118
9.2.6 Japan 119
9.3 Bibliography 119
9.4 Abbreviations 129
9.5 Research Methodology 129
9.5.1 Secondary Research 129
9.5.2 Marketed Product Profiles 130
9.5.3 Late-Stage Pipeline Candidates 130
9.5.4 Comparative Efficacy and Safety Heatmap for Marketed and Pipeline Products 130
9.5.5 Product Competitiveness Framework 130
9.5.6 Pipeline Analysis 130
9.5.7 Forecasting Model 131
9.5.8 Deals Data Analysis 132
9.6 Contact Us 133
9.7 Disclaimer 1331.1 List of Tables
Table 1: Hemophilia Therapeutics Market, APAC, Frequency of Bleeding at Various Sites of Body 12
Table 2: Diagnosis and Classification of Hemophilia Based on Clotting Factor Level and Bleeding Severity 14
Table 3: Hemophilia Therapeutics, APAC, Management of Bleeding Episodes 16
Table 4: Hemophilia A and B Therapeutics Market, Global, Licensing Deals Valued Above $10m, 2006-2017 105
Table 5: Hemophilia A and B Therapeutics Market, Global, Co-development Deals with Disclosed Deal Value, 2006-2017 111
Table 6: Hemophilia A and B Therapeutics Market, Global, All Pipeline Products, Discovery, 2017 112
Table 7: Hemophilia A and B Therapeutics Market, Global, All Pipeline Products, Preclinical, 2017 112
Table 8: Hemophilia A and B Therapeutics Market, Global, All Pipeline Products, IND/CTA-filed, 2017 113
Table 9: Hemophilia A and B Therapeutics Market, Global, All Pipeline Products, Phase I, 2017 114
Table 10: Hemophilia A and B Therapeutics Market, Global, All Pipeline Products, Phase II, 2017 114
Table 11: Hemophilia A and B Therapeutics Market, Global, All Pipeline Products, Phase III, 2017 115
Table 12: Hemophilia A and B Therapeutics Market, Global, All Pipeline Products, Pre-registration, 2017 115
Table 13: Hemophilia A Therapeutics Market, Asia-Pacific, Market Forecast, 2016-2023 115
Table 14: Hemophilia B Therapeutics Market, Asia-Pacific, Market Forecast, 2016-2023 116
Table 15: Hemophilia A Therapeutics Market, India, Market Forecast, 2016-2023 116
Table 16: Hemophilia B Therapeutics Market, India, Market Forecast, 2016-2023 116
Table 17: Hemophilia A Therapeutics Market, China, Market Forecast, 2016-2023 117
Table 18: Hemophilia B Therapeutics Market, China, Market Forecast, 2016-2023 117
Table 19: Hemophilia A Therapeutics Market, Australia, Market Forecast, 2016-2023 117
Table 20: Hemophilia B Therapeutics Market, Australia, Market Forecast, 2016-2023 118
Table 21: Hemophilia A Therapeutics Market, South Korea, Market Forecast, 2016-2023 118
Table 22: Hemophilia B Therapeutics Market, South Korea, Market Forecast, 2016-2023 118
Table 23: Hemophilia A Therapeutics Market, Japan, Market Forecast, 2016-2023 119
Table 24: Hemophilia B Therapeutics Market, Japan, Market Forecast, 2016-2023 1191.2 List of Figures
Figure 1: Hemophilia A and B Therapeutics Market, Comparative Efficacy and Safety Heatmap for Marketed Products for Treatment of Hemophilia A 42
Figure 2: Hemophilia A and B Therapeutics Market, Comparative Efficacy and Safety Heatmap for Marketed Products for the Treatment of Hemophilia B 43
Figure 3: Pipeline for Hemophilia A and B Therapeutics, Global, 2017 45
Figure 4: Pipeline for Hemophilia A and B Therapeutics by Molecular Target, Global, 2017 47
Figure 5: Hemophilia A and B Therapeutics Market, APAC, Emicizumab Market ($m), 2018-2023 50
Figure 6: Hemophilia A and B Therapeutics Market, APAC, N8-GP Market ($m), 2020-2023 52
Figure 7: Hemophilia A and B Therapeutics Market, APAC, BAY94-9027 Market ($m), 2019-2023 54
Figure 8: Hemophilia A and B Therapeutics Market, APAC, N9-GP Market ($m), 2018-2023 56
Figure 9: Hemophilia A and B Therapeutics Market, APAC, CSL-689 Market ($m), 2021-2023 59
Figure 10: Hemophilia A and B Therapeutics Market, Comparative Efficacy and Safety Heatmap for Pipeline Products for Treatment of Hemophilia A 61
Figure 11: Hemophilia A and B Therapeutics Market, Comparative Efficacy and Safety Heatmap for Pipeline Products for Treatment of Hemophilia B 62
Figure 12: Hemophilia A and B Therapeutics Market, Competitor Matrix for Hemophilia A Marketed and Pipeline Products, 2017 63
Figure 13: Hemophilia A and B Therapeutics Market, Competitor Matrix for Hemophilia B Marketed and Pipeline Products, 2017 64
Figure 14: Hemophilia A and B Therapeutics, Global, Average Clinical Trial Failure Rate (%), 2006-2016 66
Figure 15: Hemophilia A and B Therapeutics, Global, Clinical Trial Failure Rates by Molecule Type (%), 2006-2016 67
Figure 16: Hemophilia A and B Therapeutics, Global, Clinical Trial Failure Rates by Molecular Target (%), 2006-2016 68
Figure 17: Hemophilia A and B Therapeutics, Global, Clinical Trial Size per Product by Molecule Type (participants), 2006-2016 69
Figure 18: Hemophilia A and B Therapeutics, Global, Clinical Trial Size per Product by Molecular Target (participants), 2006-2016 70
Figure 19: Hemophilia A and B Therapeutics, Global, Clinical Trial Size per Trial by Molecule Type (participants), 2006-2016 71
Figure 20: Hemophilia A and B Therapeutics, Global, Clinical Trial Size per Trial by Molecular Target (participants), 2006-2016 72
Figure 21: Hemophilia A and B Therapeutics, Global, Clinical Trial Duration by Molecule Type (months), 2006-2016 73
Figure 22: Hemophilia A and B Therapeutics, Global, Clinical Trial Duration by Molecular Target (months), 2006-2016 74
Figure 23: Hemophilia A and B Therapeutics Market, Global, Comparison of Average Trial Metrics by Phase and Molecule Type 74
Figure 24: Hemophilia A and B Therapeutics Market, Global, Comparison of Average Trial Metrics by Phase and Molecular Target 75
Figure 25: Hemophilia A and B Therapeutics Market, APAC, Treatment Patterns (000’), 2016-2023 77
Figure 26: Hemophilia A and B Therapeutics Market, APAC, Market Size ($m), 2016-2023 78
Figure 27: Hemophilia A and B Therapeutics Market, India, Treatment Patterns (000’), 2016-2023 80
Figure 28: Hemophilia A and B Therapeutics Market, India, Annual Cost of Therapy ($), 2016-2023 81
Figure 29: Hemophilia A and B Therapeutics Market, India, Market Size ($m), 2016-2023 82
Figure 30: Hemophilia A and B Therapeutics Market, China, Treatment Patterns (000’), 2016-2023 84
Figure 31: Hemophilia A and B Therapeutics Market, China, Annual Cost of Therapy ($), 2016-2023 85
Figure 32: Hemophilia A and B Therapeutics Market, China, Market Size ($m), 2016-2023 86
Figure 33: Hemophilia A and B Therapeutics Market, Australia, Treatment Patterns (‘000), 2016-2023 87
Figure 34: Hemophilia A and B Therapeutics Market, Australia, Annual Cost of Therapy ($), 2016-2023 88
Figure 35: Hemophilia A and B Therapeutics Market, Australia, Market Size ($m), 2016-2023 90
Figure 36: Hemophilia A and B Therapeutics Market, South Korea, Treatment Patterns (‘000), 2016-2023 91
Figure 37: Hemophilia A and B Therapeutics Market, South Korea, Annual Cost of Therapy ($), 2016-2023 92
Figure 38: Hemophilia A and B Therapeutics Market, South Korea, Market Size ($m), 2016-2023 93
Figure 39: Hemophilia A and B Therapeutics Market, Japan, Treatment Patterns (‘000), 2016-2023 94
Figure 40: Hemophilia A and B Therapeutics Market, Japan, Annual Cost of Therapy ($), 2016-2023 95
Figure 41: Hemophilia A and B Therapeutics Market, Japan, Market Size ($m), 2016-2023 96
Figure 42: Hemophilia A and B Therapeutics Market, Global, Licensing Deals by Region and Value, 2006-2017 100
Figure 43: Hemophilia A and B Therapeutics Market, Global, Number of Disclosed and Undisclosed Licensing Deals by Year, Aggregate Deal Value and Aggregate Upfront Payment Value, 2006-2017 101
Figure 44: Hemophilia A and B Therapeutics Market, Global, Licensing Deals by Stage of Development, Deal Value and Upfront Payment Value, 2006-2017 102
Figure 45: Hemophilia A and B Therapeutics Market, Global, Licensing Deals by Molecule Type and Stage of Development, 2006-2017 103
Figure 46: Hemophilia A and B Therapeutics, Global, Licensing Deals by Molecular Targets and Aggregate Deal Value ($), 2006-2017 104
Figure 47: Hemophilia A and B Therapeutics Market, Global, Co-development Deals by Region and Deal Value, 2006-2017 106
Figure 48: Hemophilia A and B Therapeutics Market, Global, Number of Disclosed and Undisclosed Co-development Deals by Year, Aggregate Deal Value and Aggregate Upfront Payment Value, 2006-2017 107
Figure 49: Hemophilia A and B Therapeutics Market, Global, Co-development Deals by Stage of Development, Deal Value and Upfront Payment Value, 2006-2017 108
Figure 50: Hemophilia A and B Therapeutics Market, Global, Co-development Deals by Molecule type and Stage of Development, 2006-2017 109
Figure 51: Hemophilia A and B Therapeutics Market, Global, Co-development Deals by Molecular Target, 2006-2017 110
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