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HAE Therapeutics in Asia-Pacific Markets to 2023 - Growth Driven by Ongoing Shift towards Plasma-Derivative Products and Expected Launch of Premium Therapies

Publisher Name :
Date: 31-Mar-2018
No. of pages: 92

Hereditary Angioedema (HAE) is a rare genetic disorder caused by a lack of a sufficient amount of a protein called C1 esterase inhibitor (C1-INH), which plays a role in immune system function, blood clotting, and bleeding. With insufficient C1-INH a peptide called bradykinin may be overproduced, which causes fluids to build up in body tissues (edema).

The onset of HAE may occur at any age but is most common during childhood or adolescence. The rarity of the disease, limited treatment options other than not-very-effective generic drugs, and the relatively high annual cost of therapy (ACoT) have made HAE treatment a highly lucrative market.

The approval of revolutionary plasma derivatives – including Cinryze and Berinert – for the treatment of HAE patients that are refractory to generic drugs triggered unparalleled growth in the market. Though the treatment options are limited to acute and prophylaxis patients, the HAE treatment patient pool is expected to increase over forecast period in APAC countries assessed in this report.

However, competition is intensifying as governments in Australia, South Korea and Japan are looking for more affordable treatment options due to the very high treatment costs. In 2018, two re-formulations of existing plasma derivative products will be launched – one each in Japan and Australia. Despite the superior efficacy of recently marketed therapies over traditional generic therapies, there are still unmet needs in the therapeutic landscape.

These include improved safety; the need to create products with more convenient and less invasive drug-delivery methods; and improved access to treatment. The difficulty of managing HAE is also compounded by poor patient adherence, a particular problem in countries where large segments of the population have limited access to healthcare. Most significantly, the HAE market lacks curative treatments, as current therapies aim to alleviate symptoms and reduce disease progression.

This major unmet need is not expected to be addressed directly by any of the pipeline agents. Additionally, any pipeline candidates that will be successfully approved and launched will compete for the same patient populations – although late-stage drugs are likely to address the need for a more convenient route of administration, as the pipeline contains potential kallikrein inhibitors that can be administered orally. Products with novel mechanisms of action and safer and more efficacious profiles will be welcome additions to the market.

Scope


  • The current Asia-Pacific HAE market contains premium therapies such as Berinert, Cinryze, Ruconest and Firazyr.

  • What are the competitive advantages of the existing premium therapies?

  • There are 24 active pipeline molecules, and the late-stage investigational drug candidates feature improved administration routes compared with currently marketed products.

  • Which classes of re-formulation drugs are most prominent in the pipeline?

  • What is the potential for pipeline products to address unmet needs in the HAE market?

  • Analysis of clinical trials since 2006 identified that the failure rates of HAE molecules were highest in Phase III, at 14.3%.

  • How do failure rates vary by stage of development, molecule type, and molecular target?

  • How do other factors, such as average trial duration and trial size, influence the costs and risks associated with product development?

  • Over the 2016–2023 forecast period, the Asia-Pacific HAE therapeutics market is expected to grow at a CAGR of 9.6%, from $54.5m to over $103.5m.

  • Which markets make the most significant contribution to the current market size?

  • What are the epidemiology trends in these markets?

  • Will new market entrants lead to substantial changes to annual therapy costs?

  • How will different treatment usage patterns impact growth in the five assessed Asia-Pacific markets?

  • The rising HAE prevalence population and the uptake of newer therapies will lead to significant market growth over the forecast period.

  • Will the launch of emerging pipeline molecules threaten the commercial success of existing drugs?

  • Licensing deals are the most common form of strategic alliance in the HAE therapeutics market, with deal values ranging from $2.7m to over $83m.

  • How do deal frequency and value compare between target families and molecule types?

  • What were the terms and conditions of key licensing deals?


Reasons to buy


  • Understand the clinical context of HAE by considering epidemiology, symptoms, etiology and pathophysiology, diagnosis, prognosis, treatment guidelines and options, and orphan drug policies.

  • Identify the therapeutic strategies, products, and companies that dominate the current marketed products landscape and recognize gaps and areas of unmet need.

  • Identify key pipeline trends in terms of molecule type, administration route, molecular target, and novelty.

  • Consider market opportunities and potential risks by examining trends in HAE clinical trial size, duration, and failure rate by stage of development, molecule type, and molecular target.

  • Recognize the late-stage pipeline molecules that have demonstrated strong therapeutic potential in HAE by examining clinical trial data and multi-scenario product forecast projections.

  • Compare treatment usage patterns, annual therapy costs, and market growth projections for India, China, Australia, Japan and South Korea.

  • Discover trends in licensing and co-development deals concerning HAE products and identify the major strategic consolidations that have shaped the commercial landscape.

HAE Therapeutics in Asia-Pacific Markets to 2023 - Growth Driven by Ongoing Shift towards Plasma-Derivative Products and Expected Launch of Premium Therapies

Table of Contents

1 Table of Contents 4
1.1 List of Tables 6
1.2 List of Figures 6

2 Introduction 8
2.1 Disease Introduction 8
2.2 Epidemiology 8
2.3 Symptoms 8
2.4 Etiology and Pathophysiology 9
2.5 Diagnosis 9
2.6 Prognosis 11
2.7 Treatment Algorithm and Treatment Options 11
2.7.1 On-demand Treatment (Acute Attacks) 13
2.7.2 Short-term Prophylaxis 13
2.7.3 Long-term Prophylaxis 14
2.7.4 Special Considerations for Children and Women 14
2.8 Expert involvement and Checkups 16
2.9 Orphan Drug Policies in APAC Countries 16
2.9.1 China 16
2.9.2 India 17
2.9.3 Japan 18
2.9.4 South Korea 19
2.9.5 Australia 20

3 Marketed Products 21
3.1 Overview 21
3.1.1 Berinert (C1 esterase inhibitor-human) – CSL 21
3.1.2 Cinryze (C1 esterase inhibitor (human)) – Shire 24
3.1.3 Ruconest (Conestat alfa (Ruconest/Rhucin)) – Pharming Group NV 25
3.1.4 Firazyr (Icatibant acetate) – Shire 27
3.1.5 Kalbitor (Ecallantide) – Shire 28
3.1.6 Neurabol (Stanozolol) 29
3.1.7 Danazol 30
3.1.8 Cyklokapron (tranexamic acid) – Pfizer 31
3.2 Comparative Efficacy and Safety of Marketed Products 31

4 Pipeline Analysis 33
4.1 Overview 33
4.2 Pipeline by Stage of Development, Molecule Type, Route of Administration and Program Type 34
4.3 Pipeline by Molecular Target 35
4.4 Promising Pipeline Candidates 36
4.4.1 SHP-616/Cinryze (C1 esterase inhibitor-human) – Shire 36
4.4.2 CSL-830/Berinert (C1 esterase inhibitor (human)) – CSL Behring 38
4.4.3 DX-2930 (Lanadelumab) – Shire 39
4.4.4 BCX 7353 – BioCryst 40
4.5 Comparative Efficacy and Safety of Pipeline Products 42
4.6 Product Competitiveness Framework 42

5 Clinical Trial Analysis 43
5.1 Failure Rate 43
5.1.1 Overall Failure Rate 43
5.2 Clinical Trial Size 43
5.2.1 Patient Enrollment per Product by Molecule Type and Stage of Development 43
5.2.2 Patient Enrollment per Product by Molecular Target and Stage of Development 44
5.2.3 Patient Enrollment per Trial by Molecule Type and Stage of Development 45
5.2.4 Patient Enrollment per Trial by Molecular Target and Stage of Development 46
5.3 Clinical Trial Duration 47
5.3.1 Trial Duration by Molecule Type and Stage of Development 47
5.3.2 Trial Duration by Molecular Target and Stage of Development 48
5.4 Summary of Clinical Trial Metrics 49

6 Multi-scenario Forecast 51
6.1 Geographical Markets 51
6.2 Asia-Pacific Market 52
6.2.1 Treatment Usage Patterns 52
6.2.2 Market Size 53
6.3 India 54
6.3.1 Treatment Usage Patterns 54
6.3.2 Annual Cost of Therapy 55
6.3.3 Market Size 56
6.4 China 57
6.4.1 Treatment Usage Patterns 57
6.4.2 Annual Cost of Therapy 58
6.4.3 Market Size 59
6.5 Australia 60
6.5.1 Treatment Usage Patterns 60
6.5.2 Annual Cost of Therapy 61
6.5.3 Market Size 62
6.6 South Korea 63
6.6.1 Treatment Usage Patterns 63
6.6.2 Annual Cost of Therapy 64
6.6.3 Market Size 65
6.7 Japan 66
6.7.1 Treatment Usage Patterns 66
6.7.2 Annual Cost of Therapy 67
6.7.3 Market Size 68

7 Market Dynamics (Drivers and Barriers) of HAE Market 70
7.1 Drivers 70
7.1.1 Increasing Awareness 70
7.1.2 Orphan Drug Assistance in APAC Countries 70
7.1.3 Promising Late-Stage Pipeline Products and Treatment Advancements 70
7.1.4 Advancements in Diagnosis 71
7.2 Barriers 71
7.2.1 HAE – An Under-researched Disease 71
7.2.2 No Curative Treatment Therapy 71
7.2.3 Delayed/Low Diagnosis and Treatment Rates 71
7.2.4 Steep Prices of HAE Therapeutics to Slow Down Market Growth 71

8 Deals and Strategic Consolidations 72
8.1 Licensing Deals 72
8.1.1 Deals by Region and Value 72
8.1.2 Number of Disclosed and Undisclosed Deals by Year, Aggregate Deal Value 73
8.1.3 Deal Value by Stage of Development, Molecule Type, and Molecular Target 74
8.1.4 Key Licensing Deals 76
8.2 Co-development Deals 78
8.2.2 Number of Disclosed and Undisclosed Deals by Year, Aggregate Deal Value 78
8.2.3 Deal Value by Stage of Development, Molecule Type, and Molecular Target 79
8.2.4 Key Co-development Deals 79

9 Appendix 81
9.1 All Pipeline Drugs by Stage of Development 81
9.2 Market Forecasts to 2023 82
9.2.1 Asia-Pacific 82
9.2.2 India 82
9.2.3 China 83
9.2.4 Australia 83
9.2.5 South Korea 84
9.2.6 Japan 84
9.3 Bibliography 85
9.4 Abbreviations 87
9.5 Research Methodology 88
9.5.1 Secondary Research 89
9.5.2 Marketed Product Profiles 89
9.5.3 Late-Stage Pipeline Candidates 89
9.5.4 Comparative Efficacy and Safety Heat Map for Marketed and Pipeline Products 89
9.5.5 Product Competitiveness Framework 90
9.5.6 Pipeline Analysis 90
9.5.7 Forecasting Model 91
9.5.8 Deals Data Analysis 91
9.6 Contact Us 92
9.7 Disclaimer 92

List of Tables

Table 1: Complement Levels in HAE-Acquired Angioedema and ACE Inhibitor-Induced Angioedema 10
Table 2: ASCIA Position Paper Recommendations for Management of HAE in Sub-populations, Australia 15
Table 3: HAE Therapeutics Market, Procedures Recommended for Long-term Care of HAE Patients 16
Table 4: HAE Therapeutics Market, Global, Co-development Deals Value, 2007–2017 78
Table 5: HAE Therapeutics Market, Global, Co-development Deals Value, 2007–2017 80
Table 6: HAE Therapeutics Market, Global, All Pipeline Products, Discovery, 2017 81
Table 7: HAE Therapeutics Market, Asia-Pacific, Market Forecast, 2016–2023 82
Table 8: HAE Therapeutics Market, India, Market Forecast, 2016–2023 82
Table 9: HAE Therapeutics Market, China, Market Forecast, 2016–2023 83
Table 10: HAE Therapeutics Market, Australia, Market Forecast, 2016–2023 83
Table 11: HAE Therapeutics Market, South Korea, Market Forecast, 2016–2023 84
Table 12: HAE Therapeutics Market, Japan, Market Forecast, 2016–2023 84

List of Figures

Figure 1: HAE Therapeutics Market, Diagnosis Algorithm of HAE 11
Figure 2: ASCIA Clinical Management Algorithm for an Acute Attack of HAE, Australia 12
Figure 3: HAE Therapeutics Market, Comparative Efficacy and Safety Heatmap for Marketed Products 32
Figure 4: Hereditary Angioedema Therapeutics Market, Global, Pipeline, 2017 35
Figure 5: Hereditary angioedema Therapeutics Market, Global, Pipeline by Molecular Target, 2017 36
Figure 6: HAE Therapeutics Market, APAC, SHP-616 Forecast ($m), 2018–2023 38
Figure 7: HAE Therapeutics Market, APAC, CSL-830 Forecast ($m), 2018–2023 39
Figure 8: HAE Therapeutics Market, Comparative Efficacy and Safety Heatmap for Pipeline Products 42
Figure 9: Hereditary angioedema Therapeutics, Global, Clinical Trial Size per Product by Molecule Type (participants), 2007–2017 44
Figure 10: Hereditary angioedema Therapeutics, Global, Clinical Trial Size per Product by Molecular Target (participants), 2007–2017 45
Figure 11: Hereditary angioedema Therapeutics, Global, Clinical Trial Size per Trial by Molecule Type (participants), 2007–2017 46
Figure 12: Hereditary angioedema Therapeutics, Global, Clinical Trial Size per Trial by Molecular Target (participants), 2007–2017 47
Figure 13: Hereditary angioedema Therapeutics, Global, Clinical Trial Duration by Molecule Type (months), 2007–2017 48
Figure 14: Hereditary angioedema Therapeutics, Global, Clinical Trial Duration by Molecular Target (months), 2007–2017 49
Figure 15: Hereditary angioedema Therapeutics Market, Global, Comparison of Average Trial Metrics by Phase and Molecule Type 50
Figure 16: Hereditary angioedema Therapeutics Market, Global, Comparison of Average Trial Metrics by Phase and Molecular Target 50
Figure 17: HAE Therapeutics Market, APAC, Treatment Patterns, 2016–2023 52
Figure 18: HAE Therapeutics Market, APAC, Market Size ($m), 2016–2023 53
Figure 19: HAE Therapeutics Market, India, Treatment Patterns, 2016–2023 54
Figure 20: HAE Therapeutics Market, India, Annual Cost of Therapy ($), 2016–2023 56
Figure 21: HAE Therapeutics Market, India, Market Size ($m), 2016–2023 57
Figure 22: HAE Therapeutics Market, China, Treatment Patterns, 2016–2023 58
Figure 23: HAE Therapeutics Market, China, Annual Cost of Therapy ($), 2016–2023 59
Figure 24: HAE Therapeutics Market, China, Market Size ($m), 2016–2023 60
Figure 25: HAE Therapeutics Market, Australia, Treatment Usage Patterns 2016–2023 61
Figure 26: HAE Therapeutics Market, Australia, Annual Cost of Therapy ($), 2016–2023 62
Figure 27: HAE Therapeutics Market, Australia, Market Size ($m), 2016–2023 63
Figure 28: HAE Therapeutics Market, South Korea, Treatment Usage Patterns, 2016–2023 64
Figure 29: HAE Therapeutics Market, South Korea, Annual Cost of Therapy ($), 2016–2023 65
Figure 30: HAE Therapeutics Market, South Korea, Market Size ($m), 2016–2023 66
Figure 31: HAE Therapeutics Market, Japan, Treatment Usage Patterns, 2016–2023 67
Figure 32: HAE Therapeutics Market, Japan, Annual Cost of Therapy ($), 2016–2023 68
Figure 33: HAE Therapeutics Market, Japan, Market Size ($m), 2016–2023 69
Figure 34: HAE Therapeutics Market, Global, Licensing Deals by Region and Value, 2007–2017 72
Figure 35: HAE Therapeutics Market, Global, Number of Disclosed and Undisclosed Licensing Deals by Year, Aggregate Deal Value and Aggregate Upfront Payment Value, 2007–2017 73
Figure 36: HAE Therapeutics Market, Global, Licensing Deals by Stage of Development, Deal Value and Upfront Payment Value, 2007–2017 74
Figure 37: HAE Therapeutics Market, Global, Licensing Deals by Molecule Type and Stage of Development, 2007–2017 75
Figure 38: HAE Therapeutics, Global, Licensing Deals by Molecular Targets and Aggregate Deal Value ($), 2007–2017 76
Figure 39: HAE Therapeutics Market, Global, Number of Disclosed and Undisclosed Co-development Deals by Year, Aggregate Deal Value and Aggregate Upfront Payment Value, 2007–2017 78
Figure 40: HAE Therapeutics Market, Global, Co-development Deals by Stage of Development, Deal Value and Upfront Payment Value, 2007–2017 79

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