Cystic Fibrosis Therapeutics：市場分析及び予測（～2017）...市場調査レポートについてご紹介

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 R&D 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