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OpportunityAnalyzer: Cystic Fibrosis - Opportunity Analysis and Forecasts to 2018 - Event-Driven Update

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 in 2012, paved the way for a new class of therapies known as cystic fibrosis transmembrane conductance regulator (CFTR) modulators. On June 24, 2014, Vertex announced positive Phase III clinical trial data for lumacaftor in combination with Kalydeco in patients with CF who have two copies of the F508del mutation. This novel combination therapy of CFTR modulators has the potential to treat the underlying cause of the disease for approximately 50% of all CF patients and drive rapid growth in the CF market.

Highlights

Key Questions Answered

- What are the pressing unmet needs and remaining opportunities in the CF market?
- What R&D strategies are drug developers pursuing in the CF therapeutics space?
- What is the significance of CFTR modulators in the CF market?
- What is the clinical and commercial positioning of key pipeline products in the CF market?
- What are the innovative early-stage treatment approaches in the CF market?

Key Findings

- Rapid growth in the CF market is expected from 2012 to 2018.
- 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 novel 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 2018.
- 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.

Reasons to buy

- 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

OpportunityAnalyzer: Cystic Fibrosis - Opportunity Analysis and Forecasts to 2018 - Event-Driven Update
1 Table of Contents

2 Introduction 14
2.1 Catalyst 14
2.2 Related Reports 15
2.3 Upcoming Related Reports 15
3 Disease Overview 16
3.1 Etiology and Pathophysiology 16
3.1.1 Etiology 16
3.1.2 Pathophysiology 17
3.1.3 Prognosis 19
3.1.4 Quality of Life 20
3.2 Symptoms 20
4 Epidemiology 22
4.1 Risk Factors and Co-morbidities 22
4.2 Global and Historical Trends 23
4.2.1 US 23
4.2.2 France 24
4.2.3 Germany 24
4.2.4 Italy 25
4.2.5 Spain 25
4.2.6 UK 26
4.3 Forecast Methodology 26
4.3.1 Sources Used 26
4.3.2 Forecast Assumptions and Methods 29
4.3.3 Sources Not Used 32
4.4 Epidemiology Forecast 32
4.4.1 Total Prevalent Cases of Cystic Fibrosis 32
4.4.2 Total Prevalent Cases of Cystic Fibrosis Segmented by Age 34
4.4.3 Total Prevalent Cases of Cystic Fibrosis Segmented by Sex 35
4.4.4 Total Prevalent Cases of Cystic Fibrosis by Mutation Type 35
4.5 Discussion 37
4.5.1 Conclusion on Epidemiology Trends 37
4.5.2 Limitations of the Analysis 37
4.5.3 Strengths of the Analysis 38
5 Current Treatment Options 39
5.1 Overview 39
5.2 Product Profiles - Major Brands, Inhaled Antibiotics 41
5.2.1 TOBI (tobramycin) 41
5.2.2 TOBI Podhaler (tobramycin inhalation powder) 44
5.2.3 Bramitob (tobramycin) 50
5.2.4 Colistimethate Sodium (nebulized; numerous generic names) 53
5.2.5 Colobreathe (colistimethate sodium dry powder) 56
5.2.6 Cayston (aztreonam) 59
5.3 Product Profiles - Major Brands, Mucolytics 63
5.3.1 Pulmozyme (dornase alfa) 63
5.3.2 Bronchitol (mannitol) 66
5.4 Product Profiles - Major Brands, CFTR Modulators 71
5.4.1 Kalydeco (ivacaftor, VX-770) 71
5.5 Product Profiles - Major Brands, Other Therapies 77
5.5.1 Pancreatic Enzyme Replacement Therapies (PERTs) 77
6 Unmet Needs Assessment and Oppportunity Analysis 80
6.1 Overview 80
6.2 Unmet Needs Analysis 81
6.2.1 The Development of Curative Therapies 81
6.2.2 Improving Treatment of CF-related Lung Infections 82
6.2.3 Improving Airway Clearance with Mucolytic Agents 83
6.2.4 Improving Treatment Compliance 84
6.2.5 The Development of Safe Anti-inflammatory Therapies 85
6.3 Opportunity Analysis 86
6.3.1 Therapies that Target CFTR Protein Function 86
6.3.2 New Classes and Formulations of Inhaled Antibiotics 86
6.3.3 Novel Mucolytic Agents 87
6.3.4 Novel Anti-inflammatory Agents 88
7 RandD Strategies 90
7.1 Overview 90
7.1.1 Reformulation Strategies 90
7.1.2 Personalized Treatment Approach 90
7.1.3 Diverse Proof-of-Concept Research 91
7.1.4 Licensing and Alliances 92
7.2 Clinical Trial Design 92
8 Pipeline Assessment 95
8.1 Overview 95
8.2 Promising Drugs in Clinical Development 95
8.3 Promising Drugs in Clinical Development, Inhaled Antibiotics 96
8.3.1 Arikayce (amikacin) 96
8.4 Promising Drugs in Clinical Development, CFTR Modulators 101
8.4.1 Lumacaftor (VX-809)/Kalydeco (VX-770) 101
8.4.2 Ataluren (PTC124) 105
8.5 Innovative Early-Stage Approaches 109
9 Pipeline Valuation Analysis 113
9.1 Clinical Benchmark of Key Pipeline Drugs 113
9.2 Commercial Benchmark of Key Pipeline Drugs 114
9.3 Competitive Assessment 116
9.4 Top-Line Six-Year Forecast 117
9.4.1 US 119
9.4.2 5EU 120
10 Appendix 122
10.1 Bibliography 122
10.2 Abbreviations 137
10.3 Methodology 140
10.4 Forecasting Methodology 140
10.4.1 Diagnosed CF Patients 140
10.4.2 Percent Drug-treated Patients 140
10.4.3 Drugs Included in Each Therapeutic Class 141
10.4.4 Launch and Patent Expiry Dates 141
10.4.5 General Pricing Assumptions 142
10.4.6 Drug Assumptions 142
10.4.7 Generic Erosion 144
10.4.8 Pricing of Pipeline Agents 144
10.5 Physicians and Specialists Included in this Study 145
10.6 About the Authors 146
10.6.1 Analyst 146
10.6.2 Reviewer 146
10.6.3 Epidemiologist 147
10.6.4 Global Head of Healthcare 147
10.7 About GlobalData 148
10.8 Disclaimer 148

1.1 List of Tables

Table 1: Classes of the CFTR Gene Mutations in Cystic Fibrosis 17
Table 2: Symptoms of Cystic Fibrosis 21
Table 3: Six Major Markets, Sources of Cystic Fibrosis Prevalence Data 27
Table 4: Six Major Markets, Sources of Cystic Fibrosis-Causing Mutation Data 27
Table 5: Six Major Markets, Total Prevalent Cases of Cystic Fibrosis, Ages 0-35 Years, Men and Women, Selected Years, 2012-2022 33
Table 6: Six Major Markets, Total Prevalent Cases of Cystic Fibrosis, Ages 0-35 Years, By Sex, 2012, N (%) 35
Table 7: Six Major Markets, Cystic Fibrosis Prevalent Cases Segmented by Mutation Type, Ages 0-35 Years, Men and Women, 2012 36
Table 8: Leading Treatments for Cystic Fibrosis, 2014 40
Table 9: Product Profile - TOBI 42
Table 10: Product Profile - TOBI Podhaler 46
Table 11: Product Profile - Bramitob 51
Table 12: Product Profile - Colistimethate Sodium 54
Table 13: Product Profile - Colobreathe 57
Table 14: Product Profile - Cayston 60
Table 15: Product Profile - Pulmozyme 64
Table 16: Product Profile - Bronchitol 67
Table 17: Product Profile - Kalydeco 73
Table 18: Overall Unmet Needs - Current Level of Attainment 81
Table 19: Cystic Fibrosis - Late-Stage Pipeline, 2014 95
Table 20: Product Profile - Arikayce 97
Table 21: Product Profile - Lumacaftor/Kalydeco 102
Table 22: Product Profile - Ataluren 106
Table 23: Early-Stage Pipeline Products in Cystic Fibrosis, 2014 112
Table 24: Clinical Benchmark of Key Pipeline Drugs - Inhaled Antibiotics 113
Table 25: Clinical Benchmark of Key Pipeline Drugs - CFTR Modulators 114
Table 26: Commercial Benchmark of Key Pipeline Drugs - Inhaled Antibiotics 115
Table 27: Commercial Benchmark of Key Pipeline Drugs - CFTR Modulators 115
Table 28: Top-Line Sales Forecasts ($m) for Cystic Fibrosis, 2012-2018 118
Table 29: Key Events Impacting Sales for Cystic Fibrosis, 2012-2018 121
Table 30: Cystic Fibrosis Market - Drivers and Barriers, 2012-2018 121
Table 31: Key Launch Dates 141
Table 32: Key Patent Expiries 141

1.2 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 33
Figure 2: Six Major Markets, Total Prevalent Cases of Cystic Fibrosis, By Age, Men and Women, 2012 34
Figure 3: Six Major Markets, Cystic Fibrosis Prevalent Cases Segmented by Mutation Type, Ages 0-35 Years, Men and Women, 2012 36
Figure 4: Competitive Assessment of Late-Stage Pipeline Inhaled Antibiotics in Cystic Fibrosis, 2012-2018 116
Figure 5: Competitive Assessment of Late-Stage Pipeline CFTR Modulators in Cystic Fibrosis, 2012-2018 117
Figure 6: Sales for Cystic Fibrosis by Region, 2012-2018 118

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