Cystic fibrosis therapeutics in major developed market share, size, growth, trend 2019

Cystic Fibrosis Therapeutics in Major Developed Market Share, Size, Growth, Trend 2019: Radiant Insights, Inc Summary GBI Research, a leading business intelligence provider, has released its latest research report "Cystic Fibrosis Therapeutics in Major Developed Markets to 2019 - CFTR Modulators Initiate Drive towards Personalized Treatment and Market Growth". The value of the Cystic Fibrosis (CF) market is expected to increase significantly in value over the forecast period across the leading eight developed nations, from $695.6 million in 2012 to almost $4.5 billion in 2019. This equates to a Compound Annual Growth Rate (CAGR) of 30.4%. Novel treatments with disease-modifying mechanisms of action are the primary factor driving the growth of the value of the market. The positive impact of new market entrants will offset the effects of key patent losses during the forecast period.

View Full Report with TOC @ http://www.radiantinsights.com/research/cystic-fibrosistherapeutics-in-major-developed-markets-to-2019-cftr-modulators-initiate-drive-towardspersonalized-treatment-and-market-growth Cystic Fibrosis Transmembrane conductance Regulators (CFTR) modulators are the first type of CF therapy to treat the root cause of the disease rather than the symptoms, and as a result, they offer patients improved relief and quality of life. Kalydeco (ivacaftor), which has been marketed since 2012, was the first CFTR modulator to reach the market, and despite only treating a small proportion of the CF population, it had a large impact on the value of the market. This effect will be repeated on a larger scale when lumacaftor and Ataluren (PTC124) enter the market, as these treatments can be used for a much larger proportion of the CF population. Vertex Pharmaceuticals Inc. are the developers of Kalydeco, lumacaftor and other early phase CFTR modulators, and are therefore expected to play a key role in the development of the future CF market.

The pricing of CFTR modulators will be premium due to the clinical benefits and disease-modifying effects that they represent. Symptomatic therapies will be used in conjunction with CFTR modulators, and will still be a necessary part of the CF treatment plan. The antibiotics segment in particular is expected to become overcrowded and competitive over the forecast period, due to cheap, generic tobramycin products and novel market entrants competing for market share.

Scope The report assesses the current CF market, and predicts market trends to 2019, analyzing key drivers and barriers. The areas covered include - A disease introduction, which defines the disease, including symptoms, diagnosis and treatment.

- An analysis of the CF marketed landscape, including a comparison of the efficacy and safety of the most prominent brands, as well as the unmet needs of CF treatment.

- A detailed analysis of the CF pipeline, detailing, among other parameters, drug distribution by phase, molecule type and mechanism of action. The CF clinical trial landscape is then analyzed, with a particular emphasis on failure rates across different trial phases as well as the trends in clinical trial size and duration. This section also includes profiles and single product forecasts for the most promising pipeline drugs.

- An epidemiological forecast of the major CF markets. The projected values include total and treated populations.

- An in-depth forecasting model for the CF market, which considers the current marketed therapies, in addition to the potential entry of new products into the market. The model consists of a projected outcome, with high and low variance results, depending on the potential performance of pipeline therapies.

- Analysis of strategic consolidations within the CF indication, including co-development and licensing agreements.

- An overview of the drivers and barriers for the CF market is also included.

View Full Report with TOC @ http://www.radiantinsights.com/research/cystic-fibrosistherapeutics-in-major-developed-markets-to-2019-cftr-modulators-initiate-drive-towardspersonalized-treatment-and-market-growth Reasons to buy The report enables clients to develop a strong understanding of CF as a disease and a market. The current therapeutic needs and future market trends contained in this report will highlight market opportunities and the Scope of competition between CF therapeutics. Primarily, the report will allow clients to - Understand the CF pipeline and the clinical needs it is addressing. In particular, the importance and benefits of CFTR modulators are highlighted and compared with symptomatic treatments.

- Develop their knowledge of key products that may enter the market before 2019. Detailed profiles of these products are provided, with a focus on their clinical trial performance, as well as how they can be incorporated into the CF treatment plan and what competition they face.

- Assess the risk associated with CF clinical trials compared with both industry and therapy area averages.

In addition, in order to understand the trends in both trial size and duration according to mechanism of action or molecule type.

- Identify patterns of growth in the CF market over the forecast period and understand the underlying causes, as well as observing the contributions made by each major market to this growth.

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 View Full Report with TOC @ http://www.radiantinsights.com/research/cystic-fibrosistherapeutics-in-major-developed-markets-to-2019-cftr-modulators-initiate-drive-towardspersonalized-treatment-and-market-growth 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 View Full Report with TOC @ http://www.radiantinsights.com/research/cystic-fibrosistherapeutics-in-major-developed-markets-to-2019-cftr-modulators-initiate-drive-towardspersonalized-treatment-and-market-growth 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 View Full Report with TOC @ http://www.radiantinsights.com/research/cystic-fibrosistherapeutics-in-major-developed-markets-to-2019-cftr-modulators-initiate-drive-towardspersonalized-treatment-and-market-growth 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 View Full Report with TOC @ http://www.radiantinsights.com/research/cystic-fibrosistherapeutics-in-major-developed-markets-to-2019-cftr-modulators-initiate-drive-towardspersonalized-treatment-and-market-growth 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 View Full Report with TOC @ http://www.radiantinsights.com/research/cystic-fibrosistherapeutics-in-major-developed-markets-to-2019-cftr-modulators-initiate-drive-towardspersonalized-treatment-and-market-growth 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 About Radiant Insights

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