Buy the report "OpportunityAnalyzer: Cystic Fibrosis Therapeutics – Opportunity Analysis and Forecasts to 2017" at US $5995 for a Single User PDF License from RnR Market Research Reports Library.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.

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Opportunity Analysis and Forecasts to 2017” Report at US$ 5995 Single User License.
The report got published in Apr 2013 & Contains 137 Pages.
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Analysis and Forecasts to 2017” new report on its database.
New Disease-Modifying, Personalized Cystic Fibrosis Treatment, Starting a Trend in Drug Development
Disease-modifying treatments are becoming a reality for Cystic Fibrosis (CF) sufferers, potentially
transforming this life-threatening condition into a manageable one, according to new analysis by research
and consulting firm GlobalData.
The new report highlights Vertex Pharmaceuticals’ Kalydeco (ivacaftor), which received Marketing
Authorization Approvals (MAA) in the US and EU last year, and represents a milestone in the CF
therapeutics market, as we begin to enter an era of personalized medicine.
CF is caused by a genetic mutation which leads to the production of thickened sticky secretions in organs
with epithelial cell linings, including the respiratory tract. Over time, lung infections lead to airway
destruction, respiratory failure and death. The first-in-class CFTR modulator Kalydeco, a CFTR
potentiator works to restore the function of the mutated CFTR protein in patients with the G551D gating
mutation of CF, allowing an improved flow of salt and fluids on the surface of the lungs.
Only around 4% of CF patients in the US have the G551D gating mutation, and more studies are needed
to help determine whether people with other CF gating mutations might also be eligible for Kalydeco. Still,
the drug’s approval has paved the way for a new class of therapies, which offer a personalized treatment
approach to patients and could be highly profitable for its developers.
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Vertex is now developing a combination therapy of potential first-in-class CFTR modulator lumacaftor
(VX-809) with Kalydeco (VX-770) for the treatment of CF patients who are homozygous for the F508del
mutation in the CFTR gene. The CFTR corrector, lumacaftor increases trafficking of the defective CFTR
protein to the cell membrane, where it can exert its functions. The combination therapy is currently in
Phase III stage of development, and is expected to gain approval in 2014.
It is estimated that around half of all CF patients are homozygous to the F508del mutation, and Vertex
has also announced plans to explore the lumacaftor/Kalydeco combination therapy in patients that are
heterozygous to the F508del mutation – a condition that is thought to apply to 75%–80% of all CF
patients. This means that the combination therapy could potentially be used by the vast majority of CF
patients in the future. Vertex’s goal to offer treatment to as many CF patients as possible is also backed
by the development of a new generation of CFTR correctors, which are currently in early stages of clinical
development.
In addition, PTC Therapeutics is collaborating with Genzyme to enter the upcoming CFTR modulator
market, with its pipeline drug ataluren which is currently in Phase III development for patients with
nonsense mutations in the CFTR gene. This drug is designed to allow the protein synthesis machinery to
ignore these nonsense mutations, potentially addressing the underlying cause of CF in this patient group.

2. It is estimated that approximately 10% of all CF patients are carriers of nonsense mutations.
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Regulatory authorities are supporting pharmaceutical development in this therapy area. Lumacaftor and
Kalydeco are the first two drugs to be granted Breakthrough Therapy Designation by the FDA, and both
drugs now have orphan drug status in the US and EU, giving them further regulatory support and market
exclusivity for seven years in the US and 10 years in the EU. However, the high price of CFTR
modulators may prevent reimbursement by local health authorities and insurance companies, hindering
the market success of these new therapies.
GlobalData valued the CF therapeutics market across the US, France, Germany, Italy, Spain, and the UK
at $1.2 billion in 2012, and is predicted to rise to $4.6 billion by 2017 at a rapid Compound Annual Growth
Rate (CAGR) of 31.9%.
This report provides an overview of CF, including epidemiology, etiology, pathophysiology, symptoms and
current treatment options. Key topics covered include strategic product assessment, market
characterization, unmet needs, R&D strategies, clinical trial design and implications for the CF
therapeutics market. This report was built using data and information sourced from proprietary databases,
primary and secondary research, and in-house analysis conducted by GlobalData’s team of industry
experts.
This report was built using data and information sourced from proprietary databases, primary and
secondary research, and in-house analysis conducted by GlobalData’s team of industry experts.
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Key Findings
- Rapid growth in the CF market is expected from 2012 to 2017.
- 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 new 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
2017.
- 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.
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3. Table of Content
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

4. 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

5. 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
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