RnRMarketResearch.com Offers “OpportunityAnalyzer: Cystic Fibrosis Therapeutics –Opportunity Analysis and Forecasts to 201...
It is estimated that approximately 10% of all CF patients are carriers of nonsense mutations.Get Report Details @ http://w...
Table of Content1 Table of Contents 61.1 List of Tables 101.2 List of Figures 112 Introduction 122.1 Catalyst 122.2 Relate...
5.5 Product Profiles - Major Brands, Other Therapies 675.5.1 Pancreatic Enzyme Replacement Therapies (PERTs) 676 Unmet Nee...
10.4.7 Generic Erosion 13210.4.8 Pricing of Pipeline Agents 13210.5 Physicians and Specialists Included in this Study 1331...
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Cystic Fibrosis Therapeutics Opportunity Analysis 2017 Opportunity Analyzer

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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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Cystic Fibrosis Therapeutics Opportunity Analysis 2017 Opportunity Analyzer

  1. 1. RnRMarketResearch.com Offers “OpportunityAnalyzer: Cystic Fibrosis Therapeutics –Opportunity Analysis and Forecasts to 2017” Report at US$ 5995 Single User License.The report got published in Apr 2013 & Contains 137 Pages.RnRMarketResearch.com adds “OpportunityAnalyzer: Cystic Fibrosis Therapeutics – OpportunityAnalysis and Forecasts to 2017” new report on its database.New Disease-Modifying, Personalized Cystic Fibrosis Treatment, Starting a Trend in Drug DevelopmentDisease-modifying treatments are becoming a reality for Cystic Fibrosis (CF) sufferers, potentiallytransforming this life-threatening condition into a manageable one, according to new analysis by researchand consulting firm GlobalData.The new report highlights Vertex Pharmaceuticals’ Kalydeco (ivacaftor), which received MarketingAuthorization Approvals (MAA) in the US and EU last year, and represents a milestone in the CFtherapeutics 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 organswith epithelial cell linings, including the respiratory tract. Over time, lung infections lead to airwaydestruction, respiratory failure and death. The first-in-class CFTR modulator Kalydeco, a CFTRpotentiator works to restore the function of the mutated CFTR protein in patients with the G551D gatingmutation 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 neededto 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 treatmentapproach to patients and could be highly profitable for its developers.Request a Sample Copy @ http://www.rnrmarketresearch.com/contacts/request-sample?rname=94492Vertex 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 F508delmutation in the CFTR gene. The CFTR corrector, lumacaftor increases trafficking of the defective CFTRprotein to the cell membrane, where it can exert its functions. The combination therapy is currently inPhase 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 Vertexhas also announced plans to explore the lumacaftor/Kalydeco combination therapy in patients that areheterozygous to the F508del mutation – a condition that is thought to apply to 75%–80% of all CFpatients. This means that the combination therapy could potentially be used by the vast majority of CFpatients in the future. Vertex’s goal to offer treatment to as many CF patients as possible is also backedby the development of a new generation of CFTR correctors, which are currently in early stages of clinicaldevelopment.In addition, PTC Therapeutics is collaborating with Genzyme to enter the upcoming CFTR modulatormarket, with its pipeline drug ataluren which is currently in Phase III development for patients withnonsense mutations in the CFTR gene. This drug is designed to allow the protein synthesis machinery toignore these nonsense mutations, potentially addressing the underlying cause of CF in this patient group.
  2. 2. It is estimated that approximately 10% of all CF patients are carriers of nonsense mutations.Get Report Details @ http://www.rnrmarketresearch.com/opportunityanalyzer-cystic-fibrosis-therapeutics-opportunity-analysis-and-forecasts-to-2017-market-report.htmlRegulatory authorities are supporting pharmaceutical development in this therapy area. Lumacaftor andKalydeco are the first two drugs to be granted Breakthrough Therapy Designation by the FDA, and bothdrugs now have orphan drug status in the US and EU, giving them further regulatory support and marketexclusivity for seven years in the US and 10 years in the EU. However, the high price of CFTRmodulators may prevent reimbursement by local health authorities and insurance companies, hinderingthe market success of these new therapies.GlobalData valued the CF therapeutics market across the US, France, Germany, Italy, Spain, and the UKat $1.2 billion in 2012, and is predicted to rise to $4.6 billion by 2017 at a rapid Compound Annual GrowthRate (CAGR) of 31.9%.This report provides an overview of CF, including epidemiology, etiology, pathophysiology, symptoms andcurrent treatment options. Key topics covered include strategic product assessment, marketcharacterization, unmet needs, R&D strategies, clinical trial design and implications for the CFtherapeutics 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 industryexperts.This report was built using data and information sourced from proprietary databases, primary andsecondary research, and in-house analysis conducted by GlobalData’s team of industry experts.Buy a Report Copy @ http://www.rnrmarketresearch.com/contacts/purchase?rname=94492Key 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 treatmentoptions.- Annualized CF therapeutics market revenue, annual cost of therapies and forecasts for five years to2017.- Key topics covered include strategic product assessment, market characterization, unmet needs, R&Dstrategies, clinical trial design and implications for the CF therapeutics market.- Pipeline analysis: comprehensive data split across different phases, emerging trends and mechanismsof action under development, including inhaled antibiotics, CFTR modulators and pancreatic enzymeproducts.- Analysis of the current and future market competition in the US and five major EU CF therapeuticsmarket. Clinical and commercial benchmarking of promising pipeline products versus standard of caretreatments and competitive assessment of all therapies. Insightful review of the key industry drivers,restraints and challenges.Inquire for Discount @ http://www.rnrmarketresearch.com/contacts/discount?rname=94492
  3. 3. Table of Content1 Table of Contents 61.1 List of Tables 101.2 List of Figures 112 Introduction 122.1 Catalyst 122.2 Related Reports 122.3 Upcoming Related Reports 123 Disease Overview 133.1 Etiology and Pathophysiology 133.1.1 Etiology 133.1.2 Pathophysiology 143.1.3 Prognosis 163.1.4 Quality of Life 163.2 Symptoms 174 Epidemiology 184.1 Risk Factors and Co-morbidities 184.2 Global and Historical Trends 194.2.1 US 194.2.2 France 194.2.3 Germany 204.2.4 Italy 204.2.5 Spain 214.2.6 UK 214.3 Forecast Methodology 224.3.1 Sources Used 224.3.2 Forecast Assumptions and Methods 244.3.3 Sources Not Used 274.4 Epidemiology Forecast 274.4.1 Total Prevalent Cases of Cystic Fibrosis 274.4.2 Total Prevalent Cases of Cystic Fibrosis Segmented by Age 294.4.3 Total Prevalent Cases of Cystic Fibrosis Segmented by Sex 304.4.4 Total Prevalent Cases of Cystic Fibrosis by Mutation Type 314.5 Discussion 334.5.1 Conclusion on Epidemiology Trends 334.5.2 Limitations of the Analysis 334.5.3 Strengths of the Analysis 345 Current Treatment Options 355.1 Overview 355.2 Product Profiles - Major Brands, Inhaled Antibiotics 375.2.1 TOBI (tobramycin) 375.2.2 TOBI Podhaler (tobramycin inhalation powder) 405.2.3 Bramitob (tobramycin) 445.2.4 Colistimethate Sodium (nebulized; numerous generic names) 475.2.5 Colobreathe (colistimethate sodium dry powder) 495.2.6 Cayston (aztreonam) 525.3 Product Profiles - Major Brands, Mucolytics 565.3.1 Pulmozyme (dornase alfa) 565.3.2 Bronchitol (mannitol) 595.4 Product Profiles - Major Brands, CFTR Modulators 635.4.1 Kalydeco (ivacaftor, VX-770) 63
  4. 4. 5.5 Product Profiles - Major Brands, Other Therapies 675.5.1 Pancreatic Enzyme Replacement Therapies (PERTs) 676 Unmet Needs Assessment and Oppportunity Analysis 696.1 Overview 696.2 Unmet Needs Analysis 706.2.1 The Development of Curative Therapies 706.2.2 Improving Treatment of CF-related Lung Infections 716.2.3 Improving Airway Clearance with Mucolytic Agents 726.2.4 Improving Treatment Compliance 726.2.5 The Development of Safe Anti-inflammatory Therapies 736.3 Opportunity Analysis 746.3.1 Therapies that Target CFTR Protein Function 746.3.2 New Classes and Formulations of Inhaled Antibiotics 756.3.3 Novel Mucolytic Agents 756.3.4 Novel Anti-inflammatory Agents 767 R&D Strategies 777.1 Overview 777.1.1 Reformulation Strategies 777.1.2 Personalized Treatment Approach 777.1.3 Diverse Proof-of-Concept Research 787.1.4 Licensing and Alliances 787.2 Clinical Trial Design 798 Pipeline Assessment 818.1 Overview 818.2 Promising Drugs in Clinical Development 818.3 Promising Drugs in Clinical Development, Inhaled Antibiotics 828.3.1 Aeroquin (levofloxacin, MP-376) 828.3.2 Arikace (amikacin) 858.4 Promising Drugs in Clinical Development, CFTR Modulators 898.4.1 Lumacaftor (VX-809)/Kalydeco (VX-770) 898.4.2 Ataluren (PTC124) 938.5 Promising Drugs in Clinical Development, PERTs 978.5.1 Liprotamase (LY3031642) 978.6 Innovative Early-stage Approaches 1009 Pipeline Valuation Analysis 1039.1 Clinical Benchmark of Key Pipeline Drugs 1039.2 Commercial Benchmark of Key Pipeline Drugs 1059.3 Competitive Assessment 1079.4 Top-Line Five-Year Forecast 1109.4.1 US 1119.4.2 5EU 11210 Appendix 11410.1 Bibliography 11410.2 Abbreviations 12710.3 Methodology 12910.4 Forecasting Methodology 12910.4.1 Diagnosed CF Patients 12910.4.2 Percent Drug-treated Patients 12910.4.3 Drugs Included in Each Therapeutic Class 12910.4.4 Launch and Patent Expiry Dates 13010.4.5 General Pricing Assumptions 13010.4.6 Drug Assumptions 131
  5. 5. 10.4.7 Generic Erosion 13210.4.8 Pricing of Pipeline Agents 13210.5 Physicians and Specialists Included in this Study 13310.6 About the Authors 13410.6.1 Analysts 13410.6.2 Epidemiologist 13510.6.3 Global Head of Healthcare 13610.7 About GlobalData 13710.8 Contact Us 13710.9 Disclaimer 137For more details contact Mr. Priyank Tiwari: sales@rnrmarketresearch.com / +18883915441Website: http://www.rnrmarketresearch.com

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