Hemophilia A and B Therapeutics in Asia-Pacific Markets to 2022 - Growth Driven by Increasing Treatment-Receiving Pool, Launch of Long-Acting Replacement Therapies and Non-factor Therapy

GBI Research
126 Pages - GBI10584
$4,995.00

Summary

Hemophilia is a rare disease affecting more than 400,000 people worldwide. Hemophilia A is four times as common as hemophilia B, and more than half of patients with hemophilia A have a severe form of hemophilia. Males have a much higher chance of developing hemophilia because they only receive one copy of the X chromosome from their mother.

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 of hemophilia A or B often require lifelong treatment to prevent and control bleeding episodes, and this therefore represents a lucrative market opportunity.

The current marketed products landscape comprises replacement factor therapies, such as recombinant therapies and new long-acting recombinant products. The current pipeline shows strong promise, as it shows 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 in patients with hemophilia.

Though late-stage pipeline for treatment of hemophilia seems promising, the main market restraint is likely to be low penetration of new long-acting recombinant therapies in India and China, owing to their high expected prices.

Scope

The Hemophilia A and B Asia-Pacific market will be valued at $699.1m and $112.9m respectively in 2022, growing from $444.9m and $76.3m in 2015, at compound annual growth rates of 6.7% and 5.8%.
 - How will non-factor therapies contribute to the growth?
 - What will be the impact of the new long-acting recombinant factor VIII and recombinant factor IX products in the hemophilia A and B market sales?
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 for hemophilia A and B?
 - Will the current first-in-class targets have a 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 total 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

This report will allow you to -
- 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 current 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 approaches to treating hemophilia A and B.
- 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.
- Predict 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 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.

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1 Table of Contents
1 Table of Contents 5
1.1 List of Tables 8
1.2 List of Figures 9
2 Introduction 11
2.1 Disease Introduction 11
2.2 Epidemiology 12
2.3 Symptoms 12
2.4 Etiology and Pathophysiology 13
2.4.1 Hemophilia A 13
2.4.2 Hemophilia B 13
2.5 Diagnosis 14
2.6 Diagnosis of Inhibitors 14
2.7 Disease stages 15
2.8 Prognosis 15
2.9 Treatment Options 15
2.9.1 Management of Bleeding with Factor Replacement Therapy 16
2.9.2 Antifibrinolytic Medication 18
2.9.3 Treatment of Inhibitors 18
2.9.4 Gene Therapy 19
2.9.5 Hemophilia A 19
2.9.6 Hemophilia B 19
3 Marketed Products 21
3.1 Overview 21
3.2 Marketed Products - Hemophilia A 21
3.2.1 Advate (octacog alfa) - Baxalta 21
3.2.2 Adynovate (rurioctocog alfa pegol) - Baxalta 23
3.2.3 Xyntha/ReFacto AF (moroctocog alfa) - Pfizer 24
3.2.4 Kogenate FS (octacog alfa) - Bayer 25
3.2.5 Kovaltry (octacog alfa) - Bayer 26
3.2.6 Eloctate (efmoroctocog Alfa) - Biogen 28
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 32
3.3 Marketed Products - Hemophilia B 33
3.3.1 Alprolix (Eftrenonacog alfa) - Biogen 33
3.3.2 BeneFix (nonacog alfa) - Pfizer 34
3.3.3 Rixubis (nonacog gamma) - Baxter 36
3.3.4 Idelvion (albutrepenonacog alfa) - CSL Behring 36
3.4 Marketed Products - Hemophilia A and B 38
3.4.1 Feiba NF (anti-inhibitor coagulant complex) - Baxter 38
3.4.2 Byclot (freeze-dried-activated human blood coagulation factor VII concentrate containing factor X) - Kaketsuken 39
3.5 Comparative Efficacy and Safety of Marketed Products 40
4 Pipeline Analysis 42
4.1 Overview 42
4.2 Pipeline by Stage of Development, Molecule Type, Route of Administration and Program Type 42
4.3 Pipeline by Molecular Target 44
4.4 Promising Pipeline Candidates - Hemophilia A 45
4.4.1 BAX-801 (susoctocog alfa) - Baxalta 45
4.4.2 CSL-627/NBP-601 (Lonoctocog alfa) - CSL Behring 47
4.4.3 Emicizumab (ACE-910/RG6013) - Roche 49
4.4.4 N8-GP (turoctocog Alfa Pegol) - Novo Nordisk 51
4.4.5 BAY94-9027 (damoctocog alfa pegol) - Bayer 53
4.5 Promising Pipeline Candidates -Hemophilia B 55
4.5.1 N9-GP/NN-7999 (nonacog beta pegol) - Novo Nordisk 55
4.6 Promising Pipeline Candidates -Hemophilia A and B 57
4.6.1 BAX-817 (recombinant activated factor VII BI) - Baxalta 57
4.6.2 CSL-689 (recombinant activated VII-FP) - CSL Behring 58
4.7 Comparative Efficacy and Safety of Pipeline Products 59
4.8 Product Competitiveness Framework 60
5 Clinical Trial Analysis 63
5.1 Failure Rate 63
5.1.1 Overall Failure Rate 63
5.1.2 Failure Rate by Phase and Molecule Type 64
5.1.3 Failure Rate by Phase and Molecular Target 64
5.2 Clinical Trial Duration 65
5.2.1 Clinical Trial Duration by Molecule Type 65
5.2.2 Clinical Trial Duration by Molecular Target 66
5.3 Clinical Trial Size 67
5.3.1 Patient Enrollment per Product by Molecule Type, Molecular Target and Stage of Development 67
5.3.2 Patient Enrollment per Trial by Molecule Type, Molecular Target and Stage of Development 69
5.4 Competitive Clinical Trials Metrics Analysis 71
6 Multi-scenario Forecast 73
6.1 Geographical Markets 73
6.2 Asia-Pacific Market 73
6.3 India 76
6.3.1 Treatment Usage Patterns 76
6.3.2 Annual Cost of Therapy 77
6.3.3 Market Size 78
6.4 China 80
6.4.1 Treatment Usage Patterns 80
6.4.2 Annual Cost of Therapy 81
6.4.3 Market Size 82
6.5 Australia 83
6.5.1 Treatment Usage Patterns 83
6.5.2 Annual Cost of Therapy 84
6.5.3 Market Size 85
6.6 South Korea 87
6.6.1 Treatment Usage Patterns 87
6.6.2 Annual Cost of Therapy 88
6.6.3 Market Size 89
6.7 Japan 90
6.7.1 Treatment Usage Patterns 90
6.7.2 Annual Cost of Therapy 90
6.7.3 Market Size 91
7 Drivers and Barriers 93
7.1 Drivers 93
7.1.1 Increasing Awareness 93
7.1.2 Promising Late-Stage Pipeline Products with Potential to Change Treatment Paradigm 93
7.1.3 Continued Uptake of Recently Approved Therapies 93
7.1.4 Increasing Usage of Prophylactic Therapies 94
7.2 Barriers 94
7.2.1 Low Diagnosis and Low Treatment Rate 94
7.2.2 Low Access to Factor Replacement Therapies 94
7.2.3 Limitation of Premium Pricing 94
8 Deals and Strategic Consolidations 95
8.1 Licensing Deals 95
8.1.1 Deals by Region and Value 95
8.1.2 Number of Disclosed and Undisclosed Deals by Year, Aggregate Deal Value 95
8.1.3 Deal Value by Stage of Development, Molecule Type, and Molecular Target 96
8.1.4 Key Licensing Deals 99
8.2 Co-development Deals 100
8.2.1 Deals by Region and Value 100
8.2.2 Number of Disclosed and Undisclosed Deals by Year, Aggregate Deal Value 101
8.2.3 Deal Value by Stage of Development, Molecule Type, and Molecular Target 101
8.2.4 Key Co-development Deals 104
9 Appendix 106
9.1 All Pipeline Drugs by Stage of Development 106
9.1.1 Discovery 106
9.1.2 Preclinical 106
9.1.3 Phase I 108
9.1.4 Phase II 108
9.1.5 Phase III 109
9.1.6 Pre-registration 109
9.2 Market Forecasts to 2021 110
9.2.1 Asia-Pacific 110
9.2.2 India 110
9.2.3 China 111
9.2.4 Australia 112
9.2.5 South Korea 112
9.2.6 Japan 113
9.3 Bibliography 114
9.4 Abbreviations 122
9.5 Research Methodology 122
9.5.1 Secondary Research 122
9.5.2 Marketed Product Profiles 123
9.5.3 Late-Stage Pipeline Candidates 123
9.5.4 Comparative Efficacy and Safety Heatmap for Marketed and Pipeline Products 123
9.5.5 Product Competitiveness Framework 123
9.5.6 Pipeline Analysis 123
9.5.7 Forecasting Model 124
9.5.8 Deals Data Analysis 125
9.6 Contact Us 126
9.7 Disclaimer 126

1.1 List of Tables
Table 1: Hemophilia Therapeutics Market, APAC, Frequency of Bleeding at Various Sites of Body 13
Table 2: Diagnosis and Classification of Hemophilia Based on Clotting Factor Level and Bleeding Severity 15
Table 3: Hemophilia Therapeutics, APAC, Management of Bleeding Episodes 17
Table 4: Hemophilia A and B Therapeutics Market, Global, All Pipeline Products, Discovery, 2016 106
Table 5: Hemophilia A and B Therapeutics Market, Global, All Pipeline Products, Preclinical, 2016 106
Table 6: Hemophilia A and B Therapeutics Market, Global, All Pipeline Products, Phase I, 2016 108
Table 7: Hemophilia A and B Therapeutics Market, Global, All Pipeline Products, Phase II, 2016 108
Table 8: Hemophilia A and B Therapeutics Market, Global, All Pipeline Products, Phase III, 2016 109
Table 9: Hemophilia A and B Therapeutics Market, Global, All Pipeline Products, Pre-registration, 2016 109
Table 10: Hemophilia A Therapeutics Market, Asia-Pacific, Market Forecast, 2015-2022 110
Table 11: Hemophilia B Therapeutics Market, Asia-Pacific, Market Forecast, 2015-2022 110
Table 12: Hemophilia A Therapeutics Market, India, Market Forecast, 2015-2022 110
Table 13: Hemophilia B Therapeutics Market, India, Market Forecast, 2015-2022 111
Table 14: Hemophilia A Therapeutics Market, China, Market Forecast, 2015-2022 111
Table 15: Hemophilia B Therapeutics Market, China, Market Forecast, 2015-2022 111
Table 16: Hemophilia A Therapeutics Market, Australia, Market Forecast, 2015-2022 112
Table 17: Hemophilia B Therapeutics Market, Australia, Market Forecast, 2015-2022 112
Table 18: Hemophilia A Therapeutics Market, South Korea, Market Forecast, 2015-2022 112
Table 19: Hemophilia B Therapeutics Market, South Korea, Market Forecast, 2015-2022 113
Table 20: Hemophilia A Therapeutics Market, Japan, Market Forecast, 2015-2022 113
Table 21: Hemophilia B Therapeutics Market, Japan, Market Forecast, 2015-2022 113

1.2 List of Figures
Figure 1: Hemophilia A and B Therapeutics Market, Comparative Efficacy and Safety Heatmap for Marketed Products for Treatment of Hemophilia A 40
Figure 2: Hemophilia A and B Therapeutics Market, Comparative Efficacy and Safety Heatmap for Marketed Products for the Treatment of Hemophilia B 41
Figure 3: Pipeline for Hemophilia A and B Therapeutics, Global, 2016 43
Figure 4: Pipeline for Hemophilia A and B Therapeutics by Molecular Target, Global, 2016 45
Figure 5: Hemophilia A and B Therapeutics Market, APAC, BAX-801 Market ($m), 2017-2022 47
Figure 6: Hemophilia A and B Therapeutics Market, APAC, CSL-627 Market ($m), 2016-2022 49
Figure 7: Hemophilia A and B Therapeutics Market, APAC, Emicizumab Market ($m), 2019-2022 51
Figure 8: Hemophilia A and B Therapeutics Market, APAC, N8-GP Market ($m), 2020-2022 53
Figure 9: Hemophilia A and B Therapeutics Market, APAC, BAY94-9027 Market ($m), 2020-2022 54
Figure 10: Hemophilia A and B Therapeutics Market, APAC, N9-GP Market ($m), 2018-2022 56
Figure 11: Hemophilia A and B Therapeutics Market, APAC, BAX-817 Market ($m), 2017-2022 58
Figure 12: Hemophilia A and B Therapeutics Market, APAC, CSL-689 Market ($m), 2018-2022 59
Figure 13: Hemophilia A and B Therapeutics Market, Comparative Efficacy and Safety Heatmap for Pipeline Products for Treatment of Hemophilia A 60
Figure 14: Hemophilia A and B Therapeutics Market, Competitor Matrix for Hemophilia A Marketed and Pipeline Products, 2016 61
Figure 15: Hemophilia A and B Therapeutics Market, Competitor Matrix for Hemophilia B Marketed and Pipeline Products, 2016 62
Figure 16: Hemophilia A and B Therapeutics, Global, Average Clinical Trial Failure Rate (%), 2006-2016 63
Figure 17: Hemophilia A and B Therapeutics, Global, Clinical Trial Failure Rates by Molecule Type (%), 2006-2016 64
Figure 18: Hemophilia A and B Therapeutics, Global, Clinical Trial Failure Rates by Molecular Target (%), 2006-2016 65
Figure 19: Hemophilia A and B Therapeutics, Global, Clinical Trial Duration by Molecule Type (months), 2006-2016 66
Figure 20: Hemophilia A and B Therapeutics, Global, Clinical Trial Duration by Molecular Target (months), 2006-2016 67
Figure 21: Hemophilia A and B Therapeutics, Global, Clinical Trial Size per Product by Molecule Type (participants), 2006-2016 68
Figure 22: Hemophilia A and B Therapeutics, Global, Clinical Trial Size per Product by Molecular Target (participants), 2006-2016 69
Figure 23: Hemophilia A and B Therapeutics, Global, Clinical Trial Size per Trial by Molecule Type (participants), 2006-2016 70
Figure 24: Hemophilia A and B Therapeutics, Global, Clinical Trial Size per Trial by Molecular Target (participants), 2006-2016 71
Figure 25: Hemophilia A and B Therapeutics Market, Global, Comparison of Average Trial Metrics by Phase and Molecule Type 71
Figure 26: Hemophilia A and B Therapeutics Market, Global, Comparison of Average Trial Metrics by Phase and Molecular Target 72
Figure 27: Hemophilia A and B Therapeutics Market, APAC, Treatment Patterns (000’), 2015-2022 74
Figure 28: Hemophilia A and B Therapeutics Market, APAC, Market Size ($m), 2015-2022 75
Figure 29: Hemophilia A and B Therapeutics Market, India, Treatment Patterns (000’), 2015-2022 77
Figure 30: Hemophilia A and B Therapeutics Market, India, Annual Cost of Therapy ($), 2015-2022 78
Figure 31: Hemophilia A and B Therapeutics Market, India, Market Size ($m), 2015-2022 79
Figure 32: Hemophilia A and B Therapeutics Market, China, Treatment Patterns (million), 2015-2022 81
Figure 33: Hemophilia A and B Therapeutics Market, China, Annual Cost of Therapy ($), 2015-2022 82
Figure 34: Hemophilia A and B Therapeutics Market, China, Market Size ($m), 2015-2022 83
Figure 35: Hemophilia A and B Therapeutics Market, Australia, Treatment Patterns (‘000), 2015-2022 84
Figure 36: Hemophilia A and B Therapeutics Market, Australia, Annual Cost of Therapy ($), 2015-2022 85
Figure 37: Hemophilia A and B Therapeutics Market, Australia, Market Size ($m), 2015-2022 86
Figure 38: Hemophilia A and B Therapeutics Market, South Korea, Treatment Patterns (‘000), 2015-2022 87
Figure 39: Hemophilia A and B Therapeutics Market, South Korea, Annual Cost of Therapy ($), 2015-2022 88
Figure 40: Hemophilia A and B Therapeutics Market, South Korea, Market Size ($m), 2015-2022 89
Figure 41: Hemophilia A and B Therapeutics Market, Japan, Treatment Patterns (‘000), 2015-2022 90
Figure 42: Hemophilia A and B Therapeutics Market, Japan, Annual Cost of Therapy ($), 2015-2022 91
Figure 43: Hemophilia A and B Therapeutics Market, Japan, Market Size ($m), 2015-2022 92
Figure 44: Hemophilia A and B Therapeutics Market, Global, Licensing Deals by Region and Value, 2006-2015 95
Figure 45: Hemophilia A and B Therapeutics Market, Global, Number of Disclosed and Undisclosed Licensing Deals by Year, Aggregate Deal Value and Aggregate Upfront Payment Value, 2006-2015 96
Figure 46: Hemophilia A and B Therapeutics Market, Global, Licensing Deals by Stage of Development, Deal Value and Upfront Payment Value, 2006-2015 97
Figure 47: Hemophilia A and B Therapeutics Market, Global, Licensing Deals by Molecule Type and Stage of Development, 2006-2015 98
Figure 48: Hemophilia A and B Therapeutics, Global, Licensing Deals by Molecular Targets and Aggregate Deal Value ($), 2006-2015 99
Figure 49: Hemophilia A and B Therapeutics Market, Global, Co-development Deals by Region and Deal Value, 2006-2015 100
Figure 50: 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, 2006-2015 101
Figure 51: Hemophilia A and B Therapeutics Market, Global, Co-development Deals by Stage of Development, Deal Value and Upfront Payment Value, 2006-2015 102
Figure 52: Hemophilia A and B Therapeutics Market, Global, Co-development Deals by Molecule type and Stage of Development, 2006-2015 103
Figure 53: Hemophilia A and B Therapeutics Market, Global, Co-development Deals by Molecular Target, 2006-2015 104

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