1. Cystic Fibrosis Therapeutics in Major Developed Markets $4.5 billion in 2019:
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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.
TABLE OF CONTENT
1 Table of Contents
1.1 List of Tables
1.2 List of Figures
2 Introduction
2.1 Disease Introduction
2.2 Symptoms
2.3 Etiology
2.4 Pathophysiology
2.5 Diagnosis
2.6 Prognosis
2.7 Assessment of Disease Severity

2. 2.8 Treatment
3 Marketed Products
3.1 Overview
3.2 Product Profiles
3.2.1 Pulmozyme (dornase alfa) – Genentech
3.2.2 Bronchitol (mannitol) – Pharmaxis
3.2.3 TOBI (tobramycin) – Novartis
3.2.4 TOBI Podhaler (tobramycin) – Novartis
3.2.5 Bramitob/Bethkis (tobramycin) – Chiesi Farmaceutici
3.2.6 Colobreathe (colistimethate sodium) – Forest Laboratories
3.2.7 Cayston (aztreonam) – Gilead Sciences
3.2.8 Kalydeco (ivacaftor) – Vertex Pharmaceuticals
3.2.9 Pancreatic Enzyme Replacement Therapies
3.2.10 Other Successful Products
3.3 Heat Map of Marketed Products
3.4 Conclusion
4 Developmental Pipeline
4.1 Overview
4.2 Mechanisms of Action in the Pipeline
4.3 Clinical Trials
4.3.1 Failure Rate
4.3.2 Clinical Trial Duration
4.3.3 Clinical Trial Size
4.4 Promising Pipeline Molecules
4.4.1 Lumacaftor (VX809) – Vertex Pharmaceuticals

3. 4.4.2 Ataluren (PTC124) – PTC Therapeutics
4.4.3 Aeroquin (levofloxacin) – Aptalis Pharma
4.4.4 Arikace (amikacin liposomal) – Insmed
4.4.5 Other Promising Molecules
4.5 Heat Map of Pipeline Products
4.6 Conclusion
5 Market Forecast to 2019
5.1 Geographical Markets
5.1.1 Global Markets
5.1.2 North America
5.1.3 Top Five EU Countries
5.2 Drivers and Barriers
5.2.1 Drivers
5.2.2 Barriers
6 Strategic Consolidations
6.1 Major Co-Development Deals
6.2 Major Licensing Deals
7 Appendix
7.1 References
7.1.1 Marketed Product Heat Map References
7.1.2 Pipeline Product Heat Map References
7.2 Market Definition
7.3 Abbreviations
7.4 Methodology
7.5 Coverage

4. 7.6 Secondary Research
7.7 Therapeutic Landscape
7.8 Epidemiology-Based Forecasting
7.9 Market Size by Geography
7.10 Geographical Landscape
7.11 Pipeline Analysis
7.12 Competitive Landscape
7.12.1 Expert Panel Validation
7.13 Contact Us
7.14 Disclaimer
List of Tables
Table 1: Cystic Fibrosis Market, Global, Treatment Plan
Table 2: Cystic Fibrosis Market, Global, Pipeline, Preclinical
Table 3: Cystic Fibrosis Market, Global, Pipeline, Phase I
Table 4: Cystic Fibrosis Market, Global, Pipeline, Phase II
Table 5: Cystic Fibrosis Market, Global, Pipeline, Phase III
Table 6: Cystic Fibrosis Market, Global, Pipeline, Pre-Registration
Table 7: Cystic Fibrosis Market, Global, Pipeline, Undisclosed
Table 8: Cystic Fibrosis Market, Global, Market Forecast, 2012-2019
Table 9: Cystic Fibrosis Market, US, Market Forecast, 2012-2019
Table 10: Cystic Fibrosis Market, Canada, Market Forecast, 2012-2019
Table 11: Cystic Fibrosis Market, UK, Market Forecast, 2012-2019
Table 12: Cystic Fibrosis Market, France, Market Forecast, 2012-2019
Table 13: Cystic Fibrosis Market, Germany, Market Forecast, 2012-2019
Table 14: Cystic Fibrosis Market, Italy, Market Forecast, 2012-2019

5. Table 15: Cystic Fibrosis Market, Spain, Market Forecast, 2012-2019
List of Figures
Figure 1: Cystic Fibrosis Market, Global, Marketed, Heat Map
Figure 2: Cystic Fibrosis Market, Global, Pipeline Summary
Figure 3: Cystic Fibrosis Market, Global, Pipeline Mechanisms of Action
Figure 4: Cystic Fibrosis Market, Global, Clinical Trial Failure Rate
Figure 5: Cystic Fibrosis Market, Global, Pipeline Trial Duration
Figure 6: Cystic Fibrosis Market, Global, Pipeline, Trial Duration by Mechanism of Action
Figure 7: Cystic Fibrosis Market, Global, Pipeline Trial Size
Figure 8: Cystic Fibrosis Market, Global, Pipeline, Trial Size by Mechanism of Action
Figure 9: Cystic Fibrosis Market, Global, Pipeline, Promising Pipeline Products, Lumacaftor Forecast,
2015-2019
Figure 10: Cystic Fibrosis Market, Global, Pipeline, Promising Pipeline Products, Ataluren Forecast,
2016-2019
Figure 11: Cystic Fibrosis Market, Global, Pipeline Promising Pipeline Products, Aeroquin Forecast,
2014-2019
Figure 12: Cystic Fibrosis Market, Global, Pipeline, Promising Pipeline Products, Arikace Forecast,
2015-2019
Figure 13: Cystic Fibrosis Market, Global, Pipeline, Heat Map
Figure 14: Cystic Fibrosis Market, Global, Treatment Patterns, 2012–2019
Figure 15: Cystic Fibrosis Market, Global, Market Size, 2012–2019
Figure 16: Cystic Fibrosis Market, North America, Treatment Patterns, 2012–2019
Figure 17: Cystic Fibrosis Market, North America, Annual Cost of Treatment, 2012–2019
Figure 18: Cystic Fibrosis Market, North America, Market Size, 2012–2019
Figure 19: Cystic Fibrosis Market, Top Five EU Countries, Treatment Patterns, 2012–2019

6. Figure 20: Cystic Fibrosis Market, Top Five EU Countries, Annual Cost of Treatment, 2012–2019
Figure 21: Cystic Fibrosis Market, Top Five EU Countries, Market Size, 2012–2019
Figure 22: Cystic Fibrosis Market, Global, Major Co-developments by Region and Value, 2006–2013
Figure 23: Cystic Fibrosis Market, Global, Major Co-development Deals by Year, 2006–2013
Figure 24: Cystic Fibrosis Market, Global, Co-development Deals by Phase, 2006–2013
Figure 25: Cystic Fibrosis Market, Global, Licensing Deals by Region, 2006–2013
Figure 26: Cystic Fibrosis Market, Global, Licensing Deals by Year, 2006–2013
Figure 27: Cystic Fibrosis Market, Global, Licensing Deals by Phase, 2006–2013
Figure 28: Forecasting Model for Therapeutic Areas
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