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

  • ID: 4530574
  • Report
  • Region: Asia Pacific
  • 92 Pages
  • GBI Research
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Hereditary angioedema (HAE) is a disease whose symptoms are recurrent episodes of angioedema, without urticaria or pruritus, which most often affect the skin or mucosal tissues of the upper respiratory and gastrointestinal tracts.

Although the swelling is self-limited, laryngeal involvement may cause fatal asphyxiation. HAE is a rare genetic disorder caused by a deficiency in the amount of C1 esterase inhibitor (C1-INH), which is a protein that a plays role in immune system function, blood clotting, and bleeding. Without a sufficient amount of C1-INH, a peptide called bradykinin may be overproduced cause fluids to build up in body tissues (edema).

HAE’s symptoms include recurrent attacks of swelling, which commonly affect the hands, feet, arms, legs, face, abdomen, tongue, genitals and larynx (throat). Attacks involving the abdomen are often associated with severe pain, vomiting and diarrhea. Attacks of the larynx can lead to airway obstruction and be life threatening. Swelling can occur in multiple locations. The severity of the attacks gets worse with age.

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.

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

  • 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.

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.
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1 Table of Contents

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

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

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

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

6 Multi-scenario Forecast
6.1 Geographical Markets
6.2 Asia-Pacific Market
6.2.1 Treatment Usage Patterns
6.2.2 Market Size
6.3 India
6.3.1 Treatment Usage Patterns
6.3.2 Annual Cost of Therapy
6.3.3 Market Size
6.4 China
6.4.1 Treatment Usage Patterns
6.4.2 Annual Cost of Therapy
6.4.3 Market Size
6.5 Australia
6.5.1 Treatment Usage Patterns
6.5.2 Annual Cost of Therapy
6.5.3 Market Size
6.6 South Korea
6.6.1 Treatment Usage Patterns
6.6.2 Annual Cost of Therapy
6.6.3 Market Size
6.7 Japan
6.7.1 Treatment Usage Patterns
6.7.2 Annual Cost of Therapy
6.7.3 Market Size

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

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

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

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

List of Figures
Figure 1: HAE Therapeutics Market, Diagnosis Algorithm of HAE
Figure 2: ASCIA Clinical Management Algorithm for an Acute Attack of HAE, Australia
Figure 3: HAE Therapeutics Market, Comparative Efficacy and Safety Heatmap for Marketed Products
Figure 4: Hereditary Angioedema Therapeutics Market, Global, Pipeline, 2017
Figure 5: Hereditary angioedema Therapeutics Market, Global, Pipeline by Molecular Target, 2017
Figure 6: HAE Therapeutics Market, APAC, SHP-616 Forecast ($m), 2018–2023
Figure 7: HAE Therapeutics Market, APAC, CSL-830 Forecast ($m), 2018–2023
Figure 8: HAE Therapeutics Market, Comparative Efficacy and Safety Heatmap for Pipeline Products
Figure 9: Hereditary angioedema Therapeutics, Global, Clinical Trial Size per Product by Molecule Type (participants), 2007–2017
Figure 10: Hereditary angioedema Therapeutics, Global, Clinical Trial Size per Product by Molecular Target (participants), 2007–2017
Figure 11: Hereditary angioedema Therapeutics, Global, Clinical Trial Size per Trial by Molecule Type (participants), 2007–2017
Figure 12: Hereditary angioedema Therapeutics, Global, Clinical Trial Size per Trial by Molecular Target (participants), 2007–2017
Figure 13: Hereditary angioedema Therapeutics, Global, Clinical Trial Duration by Molecule Type (months), 2007–2017
Figure 14: Hereditary angioedema Therapeutics, Global, Clinical Trial Duration by Molecular Target (months), 2007–2017
Figure 15: Hereditary angioedema Therapeutics Market, Global, Comparison of Average Trial Metrics by Phase and Molecule Type
Figure 16: Hereditary angioedema Therapeutics Market, Global, Comparison of Average Trial Metrics by Phase and Molecular Target
Figure 17: HAE Therapeutics Market, APAC, Treatment Patterns, 2016–2023
Figure 18: HAE Therapeutics Market, APAC, Market Size ($m), 2016–2023
Figure 19: HAE Therapeutics Market, India, Treatment Patterns, 2016–2023
Figure 20: HAE Therapeutics Market, India, Annual Cost of Therapy ($), 2016–2023
Figure 21: HAE Therapeutics Market, India, Market Size ($m), 2016–2023
Figure 22: HAE Therapeutics Market, China, Treatment Patterns, 2016–2023
Figure 23: HAE Therapeutics Market, China, Annual Cost of Therapy ($), 2016–2023
Figure 24: HAE Therapeutics Market, China, Market Size ($m), 2016–2023
Figure 25: HAE Therapeutics Market, Australia, Treatment Usage Patterns 2016–2023
Figure 26: HAE Therapeutics Market, Australia, Annual Cost of Therapy ($), 2016–2023
Figure 27: HAE Therapeutics Market, Australia, Market Size ($m), 2016–2023
Figure 28: HAE Therapeutics Market, South Korea, Treatment Usage Patterns, 2016–2023
Figure 29: HAE Therapeutics Market, South Korea, Annual Cost of Therapy ($), 2016–2023
Figure 30: HAE Therapeutics Market, South Korea, Market Size ($m), 2016–2023
Figure 31: HAE Therapeutics Market, Japan, Treatment Usage Patterns, 2016–2023
Figure 32: HAE Therapeutics Market, Japan, Annual Cost of Therapy ($), 2016–2023
Figure 33: HAE Therapeutics Market, Japan, Market Size ($m), 2016–2023
Figure 34: HAE Therapeutics Market, Global, Licensing Deals by Region and Value, 2007–2017
Figure 35: HAE Therapeutics Market, Global, Number of Disclosed and Undisclosed Licensing Deals by Year, Aggregate Deal Value and Aggregate Upfront Payment Value, 2007–2017
Figure 36: HAE Therapeutics Market, Global, Licensing Deals by Stage of Development, Deal Value and Upfront Payment Value, 2007–2017
Figure 37: HAE Therapeutics Market, Global, Licensing Deals by Molecule Type and Stage of Development, 2007–2017
Figure 38: HAE Therapeutics, Global, Licensing Deals by Molecular Targets and Aggregate Deal Value ($), 2007–2017
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
Figure 40: HAE Therapeutics Market, Global, Co-development Deals by Stage of Development, Deal Value and Upfront Payment Value, 2007–2017

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