OpportunityAnalyzer: Cystic Fibrosis Therapeutics - Opportunity Analysis and Forecasts to 2017



Summary



Cystic Fibrosis (CF) is an autosomal recessive disease characterized by the secretion of thick, sticky mucus which clogs the lungs and leads to life-threatening lung infections; and obstructs pancreatic enzyme secretions that are essential for the body to break down and absorb nutrients. CF patients have a limited number of available treatment options and significant unmet needs still exist. Opportunities are significant for new therapies that will improve symptoms, change the course of this disease, as well as increase therapy options. The introduction of the first disease modifying therapy, Kalydeco (ivacaftor), by Vertex Pharmaceuticals, paved the way for a new class of therapies known as cystic fibrosis transmembrane conductance regulator (CFTR) modulators. During our forecast period, new CFTR modulators will enter the CF market and rapid growth is forecast.

Highlights

Key Questions Answered

- What are the pressing unmet needs and remaining opportunities in CF?
- What are the R&D strategies drugmakers are pursuing in the CF therapeutics space?
- Will CFTR modulators affect the management of CF?
- How promising are these agents; and how will they shape the CF market over the coming five years?
- How will key product patent expiries in 2014 impact the CF therapeutics market?
- What unmet needs will new pipeline agents fulfil?
- What is the clinical and commercial positioning of pipeline products?

Key Findings

- Rapid growth in the CF market is expected from 2012 to 2017.
- Emerging market players are employing diverse R&D strategies to gain entry in the CF market.
- A curative therapy is the most pressing unmet need in CF.
- Significant opportunities exist for new disease modifying drugs.



Scope


- Overview of CF, including epidemiology, etiology, pathophysiology, symptoms and current treatment options.
- Annualized CF therapeutics market revenue, annual cost of therapies and forecasts for five years to 2017.
- Key topics covered include strategic product assessment, market characterization, unmet needs, R&D strategies, clinical trial design and implications for the CF therapeutics market.
- Pipeline analysis: comprehensive data split across different phases, emerging trends and mechanisms of action under development, including inhaled antibiotics, CFTR modulators and pancreatic enzyme products.
- Analysis of the current and future market competition in the US and five major EU CF therapeutics market. Clinical and commercial benchmarking of promising pipeline products versus standard of care treatments and competitive assessment of all therapies. Insightful review of the key industry drivers, restraints and challenges.



Key Benefits

- Identify the unmet needs and remaining opportunities in the CF therapeutics market.
- Develop business strategies by understanding the trends shaping and driving the US and five major EU CF therapeutics market.
- Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.
- Assess the clinical and commercial viability of promising pipeline products.
- Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies.
- Formulate effective sales and marketing strategies by understanding the competitive landscape and by analyzing the performance of various emerging therapies.
- Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments and strategic partnerships.
- Drive revenues by understanding the key trends, innovative products and technologies, market and segments likely to impact the US and five major EU CF therapeutics market in future.

Table Of Contents

1 Table of Contents
1 Table of Contents 6
1.1 List of Tables 10
1.2 List of Figures 11
2 Introduction 12
2.1 Catalyst 12
2.2 Related Reports 12
2.3 Upcoming Related Reports 12
3 Disease Overview 13
3.1 Etiology and Pathophysiology 13
3.1.1 Etiology 13
3.1.2 Pathophysiology 14
3.1.3 Prognosis 16
3.1.4 Quality of Life 16
3.2 Symptoms 17
4 Epidemiology 18
4.1 Risk Factors and Co-morbidities 18
4.2 Global and Historical Trends 19
4.2.1 US 19
4.2.2 France 19
4.2.3 Germany 20
4.2.4 Italy 20
4.2.5 Spain 21
4.2.6 UK 21
4.3 Forecast Methodology 22
4.3.1 Sources Used 22
4.3.2 Forecast Assumptions and Methods 24
4.3.3 Sources Not Used 27
4.4 Epidemiology Forecast 27
4.4.1 Total Prevalent Cases of Cystic Fibrosis 27
4.4.2 Total Prevalent Cases of Cystic Fibrosis Segmented by Age 29
4.4.3 Total Prevalent Cases of Cystic Fibrosis Segmented by Sex 30
4.4.4 Total Prevalent Cases of Cystic Fibrosis by Mutation Type 31
4.5 Discussion 33
4.5.1 Conclusion on Epidemiology Trends 33
4.5.2 Limitations of the Analysis 33
4.5.3 Strengths of the Analysis 34
5 Current Treatment Options 35
5.1 Overview 35
5.2 Product Profiles - Major Brands, Inhaled Antibiotics 37
5.2.1 TOBI (tobramycin) 37
5.2.2 TOBI Podhaler (tobramycin inhalation powder) 40
5.2.3 Bramitob (tobramycin) 44
5.2.4 Colistimethate Sodium (nebulized; numerous generic names) 47
5.2.5 Colobreathe (colistimethate sodium dry powder) 49
5.2.6 Cayston (aztreonam) 52
5.3 Product Profiles - Major Brands, Mucolytics 56
5.3.1 Pulmozyme (dornase alfa) 56
5.3.2 Bronchitol (mannitol) 59
5.4 Product Profiles - Major Brands, CFTR Modulators 63
5.4.1 Kalydeco (ivacaftor, VX-770) 63
5.5 Product Profiles - Major Brands, Other Therapies 67
5.5.1 Pancreatic Enzyme Replacement Therapies (PERTs) 67
6 Unmet Needs Assessment and Oppportunity Analysis 69
6.1 Overview 69
6.2 Unmet Needs Analysis 70
6.2.1 The Development of Curative Therapies 70
6.2.2 Improving Treatment of CF-related Lung Infections 71
6.2.3 Improving Airway Clearance with Mucolytic Agents 72
6.2.4 Improving Treatment Compliance 72
6.2.5 The Development of Safe Anti-inflammatory Therapies 73
6.3 Opportunity Analysis 74
6.3.1 Therapies that Target CFTR Protein Function 74
6.3.2 New Classes and Formulations of Inhaled Antibiotics 75
6.3.3 Novel Mucolytic Agents 75
6.3.4 Novel Anti-inflammatory Agents 76
7 RandD Strategies 77
7.1 Overview 77
7.1.1 Reformulation Strategies 77
7.1.2 Personalized Treatment Approach 77
7.1.3 Diverse Proof-of-Concept Research 78
7.1.4 Licensing and Alliances 78
7.2 Clinical Trial Design 79
8 Pipeline Assessment 81
8.1 Overview 81
8.2 Promising Drugs in Clinical Development 81
8.3 Promising Drugs in Clinical Development, Inhaled Antibiotics 82
8.3.1 Aeroquin (levofloxacin, MP-376) 82
8.3.2 Arikace (amikacin) 85
8.4 Promising Drugs in Clinical Development, CFTR Modulators 89
8.4.1 Lumacaftor (VX-809)/Kalydeco (VX-770) 89
8.4.2 Ataluren (PTC124) 93
8.5 Promising Drugs in Clinical Development, PERTs 97
8.5.1 Liprotamase (LY3031642) 97
8.6 Innovative Early-stage Approaches 100
9 Pipeline Valuation Analysis 103
9.1 Clinical Benchmark of Key Pipeline Drugs 103
9.2 Commercial Benchmark of Key Pipeline Drugs 105
9.3 Competitive Assessment 107
9.4 Top-Line Five-Year Forecast 110
9.4.1 US 111
9.4.2 5EU 112
10 Appendix 114
10.1 Bibliography 114
10.2 Abbreviations 127
10.3 Methodology 129
10.4 Forecasting Methodology 129
10.4.1 Diagnosed CF Patients 129
10.4.2 Percent Drug-treated Patients 129
10.4.3 Drugs Included in Each Therapeutic Class 129
10.4.4 Launch and Patent Expiry Dates 130
10.4.5 General Pricing Assumptions 130
10.4.6 Drug Assumptions 131
10.4.7 Generic Erosion 132
10.4.8 Pricing of Pipeline Agents 132
10.5 Physicians and Specialists Included in this Study 133
10.6 About the Authors 134
10.6.1 Analysts 134
10.6.2 Epidemiologist 135
10.6.3 Global Head of Healthcare 136
10.7 About GlobalData 137
10.8 Contact Us 137
10.9 Disclaimer 137

List of Tables

Table 1: Classes of the CFTR Gene Mutations in Cystic Fibrosis 14
Table 2: Symptoms of Cystic Fibrosis 17
Table 3: Six Major Markets, Sources of Cystic Fibrosis Prevalence Data 22
Table 4: Six Major Markets, Sources of Cystic Fibrosis-Causing Mutation Data 22
Table 5: Six Major Markets, Total Prevalent Cases of Cystic Fibrosis, Ages 0-35 Years, Men and Women, Selected Years, 2012-2022 28
Table 6: Six Major Markets, Total Prevalent Cases of Cystic Fibrosis, Ages 0-35 Years, By Sex, 2012, N (%) 30
Table 7: Six Major Markets, Cystic Fibrosis Prevalent Cases Segmented by Mutation Type, Ages 0-35 Years, Men and Women, 2012 31
Table 8: Leading Treatments for Cystic Fibrosis, 2013 36
Table 9: Product Profile - TOBI 38
Table 10: Product Profile - TOBI Podhaler 41
Table 11: Product Profile - Bramitob 45
Table 12: Product Profile - Colistimethate Sodium 47
Table 13: Product Profile - Colobreathe 50
Table 14: Product Profile - Cayston 53
Table 15: Product Profile - Pulmozyme 57
Table 16: Product Profile - Bronchitol 59
Table 17: Product Profile - Kalydeco 64
Table 18: Overall Unmet Needs - Current Level of Attainment 69
Table 19: Cystic Fibrosis - Late-Stage Pipeline, 2013 81
Table 20: Product Profile - Aeroquin 83
Table 21: Product Profile - Arikace 86
Table 22: Product Profile - Lumacaftor/Kalydeco 90
Table 23: Product Profile - Ataluren 94
Table 24: Product Profile - Liprotamase 98
Table 25: Early-Stage Pipeline Products in Cystic Fibrosis, 2013 102
Table 26: Clinical Benchmark of Key Pipeline Drugs - Inhaled Antibiotics 103
Table 27: Clinical Benchmark of Key Pipeline Drugs - CFTR Modulators 104
Table 28: Clinical Benchmark of Key Pipeline Drugs - PEPs 104
Table 29: Commercial Benchmark of Key Pipeline Drugs - Inhaled Antibiotics 105
Table 30: Commercial Benchmark of Key Pipeline Drugs - CFTR Modulators 106
Table 31: Commercial Benchmark of Key Pipeline Drugs - PEPs 106
Table 32: Top-Line Sales Forecasts ($) for Cystic Fibrosis, 2012-2017 110
Table 33: Key Events Impacting Sales for Cystic Fibrosis, 2012-2017 113
Table 34: Cystic Fibrosis Market - Drivers and Barriers, 2012-2017 113
Table 35: Key Launch Dates 130
Table 36: Key Patent Expiries 130

List of Figures

Figure 1: Six Major Markets, Total Prevalent Cases of Cystic Fibrosis, Ages 0-35 Years, Men and Women, Selected Years, 2012-2022 28
Figure 2: Six Major Markets, Total Prevalent Cases of Cystic Fibrosis, By Age, Men and Women, 2012 29
Figure 3: Six Major Markets, Cystic Fibrosis Prevalent Cases Segmented by Mutation Type, Ages 0-35 Years, Men and Women, 2012 32
Figure 4: Competitive Assessment of Late-Stage Pipeline Inhaled Antibiotics in Cystic Fibrosis, 2012-2017 107
Figure 5: Competitive Assessment of Late-Stage Pipeline CFTR Modulators in Cystic Fibrosis, 2012-2017 108
Figure 6: Competitive Assessment of Late-Stage Pipeline PEPs in Cystic Fibrosis, 2012-2017 109
Figure 7: Sales for Cystic Fibrosis by Region, 2012-2017 111

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