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Cystic Fibrosis Therapeutics in Major Developed Markets to 2019 - CFTR Modulators Initiate Drive towards Personalized Treatment and Market Growth

Feb 6, 2014 | 76 Pages | GBI Research | Pharmaceuticals & Healthcare

The cystic fibrosis market had a market value of more than USD 695 million in 2012 and is anticipated to rise drastically over the future period in the major 8 developed countries and will reach more than USD 4.0 billion by 2019. Thus associates to compound annual growth rate of more than 30%. 

Original treatment with disease-modifying mechanism of action is the main feature increasing the growth of the market in terms of value. The constructive effect of the market’s new applicant will counteract the effects of major patent failures throughout the estimated period.

The first ever cystic fibrosis therapy was CFTR (Cystic Fibrosis Transmembrane conductance Regulator) for treating the root reasons of the illness instead of the symptom. This resulted into the Patients’ enhanced help and value of life. Among CFTR modulator, Kalydeco was the first one to reach the market. This was in the market since 2012. It could treat only a tiny part of CF people, yet the market value had a major effect of it. 

 With the market entry of Ataluren & lumacaftor, the effect is going to be repetitive on a huge extent, because of the ability of it being used on a bigger number of the CF sufferers. Llumacaftor, Kalydeco, and early previous phase CFTR modulator are developed by Vertex Pharmaceuticals Inc. These are hence anticipated to be performing a major role in developing the forth coming market.

The rates of the Cystic Fibrosis Transmembrane conductance Regulators are expected to be high owing to the effects that are represented on the disease modification and benefits for the clinics. Suggestive therapies are going to be used in CFTR modulator combination, and yet it will be an essential part for the plan of the CF treatment. Particularly, the antibiotic sector is anticipated to turn out to be ready for action and filled to capacity in the future period. This will be because of the fresh market applicants challenging for the share of the market, low in price, and products of standard tobramycin.
 Table of Contents
Table of Contents 4
1.1 List of Tables 5
1.2 List of Figures 6
2 Introduction 7
2.1 Disease Introduction 7
2.2 Symptoms 8
2.3 Etiology 8
2.4 Pathophysiology 9
2.5 Diagnosis 9
2.6 Prognosis 10
2.7 Assessment of Disease Severity 10
2.8 Treatment 10
3 Marketed Products 12
3.1 Overview 12
3.2 Product Profiles 13
3.2.1 Pulmozyme (dornase alfa) - Genentech 13
3.2.2 Bronchitol (mannitol) - Pharmaxis 14
3.2.3 TOBI (tobramycin) - Novartis 15
3.2.4 TOBI Podhaler (tobramycin) - Novartis 16
3.2.5 Bramitob/Bethkis (tobramycin) - Chiesi Farmaceutici 17
3.2.6 Colobreathe (colistimethate sodium) - Forest Laboratories 17
3.2.7 Cayston (aztreonam) - Gilead Sciences 18
3.2.8 Kalydeco (ivacaftor) - Vertex Pharmaceuticals 19
3.2.9 Pancreatic Enzyme Replacement Therapies 20
3.2.10 Other Successful Products 21
3.3 Heat Map of Marketed Products 21
3.4 Conclusion 23
4 Developmental Pipeline 25
4.1 Overview 25
4.2 Mechanisms of Action in the Pipeline 27
4.3 Clinical Trials 30
4.3.1 Failure Rate 30
4.3.2 Clinical Trial Duration 31
4.3.3 Clinical Trial Size 32
4.4 Promising Pipeline Molecules 35
4.4.1 Lumacaftor (VX809) - Vertex Pharmaceuticals 35
4.4.2 Ataluren (PTC124) - PTC Therapeutics 37
4.4.3 Aeroquin (levofloxacin) - Aptalis Pharma 38
4.4.4 Arikace (amikacin liposomal) - Insmed 40
4.4.5 Other Promising Molecules 41
4.5 Heat Map of Pipeline Products 42
4.6 Conclusion 44
5 Market Forecast to 2019 45
5.1 Geographical Markets 45
5.1.1 Global Markets 46
5.1.2 North America 48
5.1.3 Top Five EU Countries 51
5.2 Drivers and Barriers 55
5.2.1 Drivers 55
5.2.2 Barriers 56
6 Strategic Consolidations 57
6.1 Major Co-Development Deals 57
6.2 Major Licensing Deals 60
7 Appendix 63
7.1 References 68
7.1.1 Marketed Product Heat Map References 70
7.1.2 Pipeline Product Heat Map References 71
7.2 Market Definition 71
7.3 Abbreviations 72
7.4 Methodology 72
7.5 Coverage 72
7.6 Secondary Research 73
7.7 Therapeutic Landscape 73
7.8 Epidemiology-Based Forecasting 74
7.9 Market Size by Geography 75
7.10 Geographical Landscape 75
7.11 Pipeline Analysis 76
7.12 Competitive Landscape 76
7.12.1 Expert Panel Validation 76
7.13 Contact Us 76
7.14 Disclaimer 76
List of Tables
Table 1: Cystic Fibrosis Market, Global, Treatment Plan 11
Table 2: Cystic Fibrosis Market, Global, Pipeline, Preclinical 63
Table 3: Cystic Fibrosis Market, Global, Pipeline, Phase I 64
Table 4: Cystic Fibrosis Market, Global, Pipeline, Phase II 64
Table 5: Cystic Fibrosis Market, Global, Pipeline, Phase III 64
Table 6: Cystic Fibrosis Market, Global, Pipeline, Pre-Registration 65
Table 7: Cystic Fibrosis Market, Global, Pipeline, Undisclosed 65
Table 8: Cystic Fibrosis Market, Global, Market Forecast, 2012-2019 65
Table 9: Cystic Fibrosis Market, US, Market Forecast, 2012-2019 65
Table 10: Cystic Fibrosis Market, Canada, Market Forecast, 2012-2019 66
Table 11: Cystic Fibrosis Market, UK, Market Forecast, 2012-2019 66
Table 12: Cystic Fibrosis Market, France, Market Forecast, 2012-2019 66
Table 13: Cystic Fibrosis Market, Germany, Market Forecast, 2012-2019 67
Table 14: Cystic Fibrosis Market, Italy, Market Forecast, 2012-2019 67
Table 15: Cystic Fibrosis Market, Spain, Market Forecast, 2012-2019 67 


List of Figures
Figure 1: Cystic Fibrosis Market, Global, Marketed, Heat Map 22
Figure 2: Cystic Fibrosis Market, Global, Pipeline Summary 26
Figure 3: Cystic Fibrosis Market, Global, Pipeline Mechanisms of Action 28
Figure 4: Cystic Fibrosis Market, Global, Clinical Trial Failure Rate 30
Figure 5: Cystic Fibrosis Market, Global, Pipeline Trial Duration 31
Figure 6: Cystic Fibrosis Market, Global, Pipeline, Trial Duration by Mechanism of Action 32
Figure 7: Cystic Fibrosis Market, Global, Pipeline Trial Size 33
Figure 8: Cystic Fibrosis Market, Global, Pipeline, Trial Size by Mechanism of Action 34
Figure 9: Cystic Fibrosis Market, Global, Pipeline, Promising Pipeline Products, Lumacaftor Forecast, 2015-2019 36
Figure 10: Cystic Fibrosis Market, Global, Pipeline, Promising Pipeline Products, Ataluren Forecast, 2016-2019 38
Figure 11: Cystic Fibrosis Market, Global, Pipeline Promising Pipeline Products, Aeroquin Forecast, 2014-2019 39
Figure 12: Cystic Fibrosis Market, Global, Pipeline, Promising Pipeline Products, Arikace Forecast, 2015-2019 41
Figure 13: Cystic Fibrosis Market, Global, Pipeline, Heat Map 43
Figure 14: Cystic Fibrosis Market, Global, Treatment Patterns, 2012-2019 46
Figure 15: Cystic Fibrosis Market, Global, Market Size, 2012-2019 47
Figure 16: Cystic Fibrosis Market, North America, Treatment Patterns, 2012-2019 48
Figure 17: Cystic Fibrosis Market, North America, Annual Cost of Treatment, 2012-2019 49
Figure 18: Cystic Fibrosis Market, North America, Market Size, 2012-2019 50
Figure 19: Cystic Fibrosis Market, Top Five EU Countries, Treatment Patterns, 2012-2019 51
Figure 20: Cystic Fibrosis Market, Top Five EU Countries, Annual Cost of Treatment, 2012-2019 53
Figure 21: Cystic Fibrosis Market, Top Five EU Countries, Market Size, 2012-2019 54
Figure 22: Cystic Fibrosis Market, Global, Major Co-developments by Region and Value, 2006-2013 57
Figure 23: Cystic Fibrosis Market, Global, Major Co-development Deals by Year, 2006-2013 58
Figure 24: Cystic Fibrosis Market, Global, Co-development Deals by Phase, 2006-2013 58
Figure 25: Cystic Fibrosis Market, Global, Licensing Deals by Region, 2006-2013 60
Figure 26: Cystic Fibrosis Market, Global, Licensing Deals by Year, 2006-2013 60
Figure 27: Cystic Fibrosis Market, Global, Licensing Deals by Phase, 2006-2013 61
Figure 28: Forecasting Model for Therapeutic Areas 75

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