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OpportunityAnalyzer: Cystic Fibrosis - Opportunity Analysis and Forecasts to 2025

OpportunityAnalyzer: Cystic Fibrosis - Opportunity Analysis and Forecasts to 2025

Summary

Cystic fibrosis (CF), which has been given a the diagnostic code E84 from The International Classification of Diseases, Tenth Edition (ICD-10), is an autosomal recessive disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which codes for the CFTR protein. The CFTR protein is mainly located at the apical membrane (the surface of the plasma membrane that faces inward to the lumen) of polarized epithelial tissues, but expression of the CFTR gene in the body is widespread. CFTR protein has been found in tissues such as cardiac myocytes, smooth muscle, erythrocytes, and immune cells (Lubamba et al., 2012).

Global (7MM) sales for the cystic fibrosis (CF) market are forecast by GlobalData to grow from $2.1B in 2015 to $7.6B in 2025. The majority of sales are expected to come from the US, which will represent 68% of the market by 2025. The sales growth during the 2015-2025 forecast periods will be primarily driven by the launches of Vertex’s cystic fibrosis transmembrane conductance regulator (CFTR) modulator ivacaftor/tezacaftor. The CFTR modulator drug class is expected to grow from 43.3% of total CF drug sales in 2015 to 81.2% of total CF drug sales in 2025. Vertex Pharmaceuticals will continue to dominate the CF drug space as it has two marketed CFTR modulators and is expected to launch third CFTR modulator during the forecast period, fulfilling some of the major unmet needs of the CF population.

Major drivers of the growth of the CF market over the forecast period will include -

  • The introduction of the high-priced, disease-modifying drug Orkambi (ivacaftor + lumacaftor) in 2015 followed by the introduction of novel, high-priced, disease-modifying CFTR modulators during the forecast period.
  • The continued growth of the number of CF patients due to the overall population growth and continuously increasing life expectancy of CF patients.
R&D strategies in the CF space have focused on symptomatic relief and have targeted organ-specific sequelae of the underlying disease. This approach has mainly resulted in the development of new formulations of antibiotics that can be administered via inhalation for the management of chest infections, and therapies that enhance airway mucociliary clearance. However, over the last decade, advanced technologies have yielded a shift in research development towards therapies that target the underlying cause of the disease. These therapies aim to enhance CFTR function by either restoring wild-type CFTR protein expression or modulating the function of mutant CFTR proteins in cells (Mayer-Hamblett et al., 2016).

The report OpportunityAnalyzer: Cystic Fibrosis - Opportunity Analysis and Forecasts to 2025 provides overview of cystic fibrosis, including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and treatment guidelines.

In particular, this report enable you the following -
  • Annualized CF therapeutics market revenue, annual cost of therapy and treatment usage pattern data from 2015 and forecast for ten years to 2025.
  • Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping and implications for the CF therapeutics market.
  • Pipeline analysis: comprehensive data split across different phases, emerging novel trends under development, and detailed analysis of late-stage pipeline drugs.
  • Analysis of the current and future market competition in the global CF therapeutics market. Insightful review of the key industry drivers, restraints and challenges. Each trend is independently researched to provide qualitative analysis of its implications.
Companies mentioned in this report: AbbVie, Chiesis USA, Forest Laboratories, Gilead, Insmed Therapeutics, Johnson & Johnson, Novartis, Pharmaxis, Raptor Pharmaceuticals, Roche, Vertex Pharmaceuticals.

Scope
  • Overview of cystic fibrosis, including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and treatment guidelines.
  • Annualized CF therapeutics market revenue, annual cost of therapy and treatment usage pattern data from 2015 and forecast for ten years to 2025.
  • Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping and implications for the CF therapeutics market.
  • Pipeline analysis: comprehensive data split across different phases, emerging novel trends under development, and detailed analysis of late-stage pipeline drugs.
  • Analysis of the current and future market competition in the global CF therapeutics market. Insightful review of the key industry drivers, restraints and challenges. Each trend is independently researched to provide qualitative analysis of its implications.
Reasons to buy

The report will enable you to -
  • Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline. Additionally a list of acquisition targets included in the pipeline product company list.
  • Develop business strategies by understanding the trends shaping and driving the global CF therapeutics market.
  • Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the global CF therapeutics market in future.
  • Formulate effective sales and marketing strategies by understanding the competitive landscape and by analysing the performance of various competitors.
  • Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.
  • Track drug sales in the global CF therapeutics market from 2015-2025.
  • Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments and strategic partnerships.


1 Table of Contents
1.1 List of Tables
1.2 List of Figures
2 Cystic Fibrosis: Executive Summary
2.1 CF Transmembrane Conductance Regulator Modulators Expected to Drive Substantial Growth in the Cystic Fibrosis Market from 2015-2025
2.2 Licensing Activity and the Formation of New Partnerships Will Steer Small Companies to Continue Research of Novel Compounds
2.3 Marketed and Pipeline CF Transmembrane Conductance Regulator Modulators Will Provide Appropriate Treatment for the Majority of the CF Population, but the High Unmet Needs Will Remain
2.4 Opportunities Will Remain for the Development of New CF Transmembrane Conductance Regulator Modulators
2.5 What Do Physicians Think?
3 Introduction
3.1 Catalyst
3.2 Related Reports
3.3 Upcoming Related Reports
4 Disease Overview
4.1 Etiology and Pathophysiology
4.1.1 Etiology
4.1.2 Pathophysiology
4.2 Classification
4.3 Symptoms
4.4 Prognosis
4.5 Quality of Life
5 Epidemiology
5.1 Disease Background
5.2 Risk Factors and Comorbidities
5.3 Global Trends
5.3.1 Diagnosed Incidence and Diagnosed Prevalence
5.4 Forecast Methodology
5.4.1 Sources Used
5.4.2 Forecast Assumptions and Methods
5.4.3 Sources Not Used
5.5 Epidemiological Forecast for CF (2015-2025)
5.5.1 Diagnosed Incident Cases, Based on Registry Data
5.5.2 Diagnosed Prevalent Cases, Based on Registry Data
5.5.3 CF Mortality, Based on Registry Data
5.5.4 Diagnosed Incident Cases, Adjusted for Underestimation
5.5.5 Diagnosed Prevalent Cases, Adjusted for Underestimation
5.5.6 CF Mortality, Adjusted for Underestimation
5.6 Discussion
5.6.1 Epidemiological Forecast Insight
5.6.2 Limitations of the Analysis
5.6.3 Strengths of the Analysis
6 Current Treatment Options
6.1 Overview
6.2 Diagnosis and Treatment
6.2.1 Diagnosis
6.2.2 Treatment Guidelines and Leading Prescribed Drugs
6.3 Clinical Practice
6.4 Product Profiles - Major Brands, Inhaled Antibiotics
6.4.1 TOBI (tobramycin)
6.4.2 TOBI Podhaler (tobramycin)
6.4.3 Quinsair (levofloxacin)
6.4.4 Cayston (aztreonam)
6.4.5 Bramitob/Bethkis (tobramycin)
6.4.6 Colobreathe (colistimethate sodium)
6.5 Product Profiles - Major Brands, Mucolytics
6.5.1 Pulmozyme (dornase alfa)
6.5.2 Bronchitol (mannitol)
6.6 Product Profiles - Major Brands, CF Transmembrane Conductance Regulator Modulators
6.6.1 Kalydeco (ivacaftor)
6.6.2 Orkambi (ivacaftor + lumacaftor)
6.7 Product Profiles - Major Brands, Other Therapies
6.7.1 Pancreatic Enzyme Replacement Therapies
7 Unmet Needs Assessment and Opportunity Analysis
7.1 Overview
7.2 Development of More Effective or Curative Therapies
7.2.1 Unmet Need
7.2.2 Gap Analysis
7.2.3 Opportunity
7.3 Development of Chronic Lung Infection Therapies
7.3.1 Unmet Need
7.3.2 Gap Analysis
7.3.3 Opportunity
7.4 Increased Treatment Compliance
7.4.1 Unmet Need
7.4.2 Gap Analysis
7.4.3 Opportunity
7.5 Development of Mucolytic Therapies
7.5.1 Unmet Need
7.5.2 Gap Analysis
7.5.3 Opportunity
8 R&D Strategies
8.1 Overview
8.1.1 Reformulation Strategies
8.1.2 Personalized Treatment Approach
8.1.3 Development of Agents with Novel Mechanisms of Action
8.1.4 Licensing and Alliances
8.2 Clinical Trial Design
8.2.1 Current Clinical Trials
8.2.2 CF Transmembrane Conductance Regulator Modulator Clinical Trials
9 Pipeline Assessment
9.1 Overview
9.2 Promising Drugs in Clinical Development
9.3 Promising Drugs in Clinical Development, Inhaled Antibiotics
9.3.1 Arikace (liposomal amikacin)
9.4 Promising Drugs in Clinical Development, CF Transmembrane Conductance Regulator Modulators
9.4.1 Ivacaftor/Tezacaftor
9.5 Innovative Early-Stage Approaches
10 Pipeline Valuation Analysis
10.1 Clinical Benchmark of Key Pipeline Drugs
10.2 Commercial Benchmark of Key Pipeline Drugs
10.3 Competitive Assessment
10.4 Top-Line 10-Year Forecast
10.4.1 US
10.4.2 5EU
10.4.3 Canada
11 Appendix
11.1 Bibliography
11.2 Abbreviations
11.3 Methodology
11.4 Forecasting Methodology
11.4.1 Diagnosed CF Patients
11.4.2 Percent Drug-Treated Patients
11.4.3 Drugs Included in Each Therapeutic Class
11.4.4 Launch and Patent Expiry Dates
11.4.5 General Pricing Assumptions
11.4.6 Drug Assumptions
11.4.7 Pricing of Pipeline Agents
11.5 Primary Research - Key Opinion Leaders Interviewed for This Report
11.6 Primary Research - Prescriber Survey
11.7 About the Authors
11.7.1 Analyst
11.7.2 Reviewer
11.7.3 Therapy Area Director
11.7.4 Epidemiologist
11.7.5 Managing Epidemiologist
11.7.6 Global Director of Therapy Analysis and Epidemiology
11.7.7 Global Head of Healthcare
11.8 About GlobalData
11.9 Contact Us
11.10 Disclaimer
1.1 List of Tables
Table 1: Cystic Fibrosis: Key Metrics in the Seven Major Pharmaceutical Markets
Table 2: Onset of CF by Approximate Age
Table 3: Classification and Criteria of the CFTR Gene Mutations in CF
Table 4: Symptoms of CF
Table 5: Risk Factors and Comorbidities of CF
Table 6: 7MM, Sources Used to Forecast the Diagnosed Incident Cases of CF (Based on
Registry Data)
Table 7: 7MM, Sources Used to Forecast the Diagnosed Prevalent Cases of CF (Based on Registry Data)
Table 8: 7MM, Sources Used for Segmentation of Diagnosed Prevalent Cases of CF by Specific Mutations (Based on Registry Data)
Table 9: 7MM, Sources Used to Forecast the Proportion of Diagnosed Prevalent Cases of CF by Chronic MRSA Infection (Based on Registry Data)
Table 10: 7MM, Sources Used to Forecast CF Deaths (Based on Registry Data)
Table 11: 7MM, Proportion of CF Cases Reported to Respective CF Registries (Primary Research)
Table 12: 7MM, Sources Not Used in the Epidemiological Analysis of CF
Table 13: 7MM, Diagnosed Incident Cases of CF, All Ages, Both Sexes, N, Selected Years 2015-2025 (Based on Registry Data)
Table 14: 7MM, Sex-Specific Diagnosed Incident Cases of CF, All Ages, N (Row %), 2015 (Based on Registry Data)
Table 15: 7MM, Diagnosed Prevalent Cases of CF, All Ages, Both Sexes, N, Selected Years 2015-2025 (Based on Registry Data)
Table 16: 7MM, Age-Specific Diagnosed Prevalent Cases of CF, Both Sexes, N (Row %), 2015 (Based on Registry Data)
Table 17: 7MM, Sex-Specific Diagnosed Prevalent Cases of CF, All Ages, N (Row %), 2015 (Based on Registry Data)
Table 18: 7MM, Diagnosed Prevalent Cases of CF with Chronic MRSA Infection, All Ages, Both Sexes, N, Selected Years 2015-2025 (Based on Registry Data)
Table 19: 7MM, CF Deaths, All Ages, Both Sexes, N, Selected Years 2015-2025 (Based on Registry Data)
Table 20: 7MM, Sex-Specific CF Deaths, All Ages, N (Row %), 2015 (Based on Registry Data)
Table 21: 7MM, Diagnosed Incident Cases of CF, All Ages, Both Sexes, N, Selected Years 2015-2025 (Adjusted for Underestimation)
Table 22: 7MM, Sex-Specific Diagnosed Incident Cases of CF, All Ages, N (Row %), 2015 (Adjusted for Underestimation)
Table 23: 7MM, Diagnosed Prevalent Cases of CF, All Ages, Both Sexes, N, Selected Years 2015-2025 (Adjusted for Underestimation)
Table 24: 7MM, Age-Specific Diagnosed Prevalent Cases of CF, Both Sexes, N (Row %), 2015 (Adjusted for Underestimation)
Table 25: 7MM, Sex-Specific Diagnosed Prevalent Cases of CF, All Ages, N (Row %), 2015 (Adjusted for Underestimation)
Table 26: 7MM, Diagnosed Prevalent Cases of CF with Chronic MRSA Infection, All Ages, Both Sexes, N, Selected Years 2015-2025 (Adjusted for Underestimation)
Table 27: 7MM, CF Deaths, All Ages, Both Sexes, N, Selected Years 2015-2025 (Adjusted for Underestimation)
Table 28: 7MM, Sex-Specific CF Deaths, All Ages, N (Row %), 2015 (Adjusted for Underestimation)
Table 29: Treatment Guidelines for CF
Table 30: Leading Treatments for CF, 2016
Table 31: Product Profile - TOBI
Table 32: TOBI SWOT Analysis, 2016
Table 33: Product Profile - TOBI Podhaler
Table 34: TOBI Podhaler SWOT Analysis, 2016
Table 35: Product Profile - Quinsair
Table 36: Quinsair SWOT Analysis, 2016
Table 37: Product Profile - Cayston
Table 38: Cayston SWOT Analysis, 2016
Table 39: Product Profile - Bramitob
Table 40: Bramitob SWOT Analysis, 2016
Table 41: Product Profile - Colobreathe
Table 42: Colobreathe SWOT Analysis, 2016
Table 43: Product Profile - Pulmozyme
Table 44: Pulmozyme SWOT Analysis, 2016
Table 45: Product Profile - Bronchitol
Table 46: Bronchitol SWOT Analysis, 2016
Table 47: Product Profile - Kalydeco
Table 48: Kalydeco SWOT Analysis, 2016
Table 49: Product Profile - Orkambi
Table 50: Orkambi SWOT Analysis, 2016
Table 51: Pancreatic Enzyme Replacement Therapies (PERTs) SWOT Analysis, 2016
Table 52: Unmet Need and Opportunity in CF, 2016
Table 53: Key Late-Stage Pipeline Agents for CF, 2016
Table 54: Comparison of Therapeutic Classes in Development for CF, 2015-2025
Table 55: Product Profile - Arikace
Table 56: Insmed’s Arikace - Phase III Clinical Trials
Table 57: Arikace SWOT Analysis, 2016
Table 58: Product Profile - ivacaftor/tezacaftor
Table 59: Vertex’s ivacaftor/tezacaftor - Phase III Clinical Trials
Table 60: Efficacy of Ivacaftor + Tezacaftor
Table 61: Ivacaftor + Tezacaftor SWOT Analysis, 2016
Table 62: Drugs in Development for CF, 2016
Table 63: Clinical Benchmark of Key Pipeline Drugs - Inhaled Antibiotics
Table 64: Clinical Benchmark of Key Pipeline Drugs - CFTR Modulators
Table 65: Commercial Benchmark of Key Pipeline Drugs - Inhaled Antibiotics
Table 66: Commercial Benchmark of Key Pipeline Drugs - CFTR Modulators
Table 67: Top-Line Sales Forecasts ($m) for CF, 2015-2025
Table 68: Key Events Impacting Sales for CF, 2015-2025
Table 69: CF Market - Global Drivers and Barriers, 2015‒2025
Table 70: Key Historical and Projected Launch Dates
Table 71: Key Historical and Projected Patent Expiry Dates
Table 72: High-Prescribing Physicians (Non-KOLs) Surveyed, By Country
1.2 List of Figures
Figure 1: Global Sales for CF by Country/Region, 2015 and 2025
Figure 2: Classes of CFTR Gene Mutations in CF
Figure 3: 7MM, Diagnosed Incident Cases of CF, All Ages, Both Sexes, N, 2015-2025 (Based on Registry Data)
Figure 4: 7MM, Sex-Specific Diagnosed Incident Cases of CF, All Ages, N, 2015 (Based on Registry Data)
Figure 5: 7MM, Diagnosed Prevalent Cases of CF, All Ages, Both Sexes, N, Selected Years 2015-2025 (Based on Registry Data)
Figure 6: 7MM, Age-Specific Diagnosed Prevalent Cases of CF, Both Sexes, N, 2015 (Based on Registry Data)
Figure 7: 7MM, Sex-Specific Diagnosed Prevalent Cases of CF, All Ages, N, 2015 (Based on Registry Data)
Figure 8: 7MM, Age-Standardized Diagnosed Prevalence of CF, All Ages, N, 2015 (Based on Registry Data)
Figure 9: 7MM, Diagnosed Prevalent Cases of CF with Specific Mutations, Both Sexes, All Ages, N, 2015 (Based on Registry Data)
Figure 10: 7MM, Diagnosed Prevalent Cases of CF with Chronic MRSA Infection, Both Sexes, All Ages, N, 2015 (Based on Registry Data)
Figure 11: 7MM, CF Deaths, All Ages, Both Sexes, N, Selected Years 2015-2025 (Based on Registry Data)
Figure 12: 7MM, Sex-Specific CF Deaths, All Ages, N, 2015 (Based on Registry Data)
Figure 13: 7MM, Diagnosed Incident Cases of CF, All Ages, Both Sexes, N, 2015-2025 (Adjusted for Underestimation)
Figure 14: 7MM, Sex-Specific Diagnosed Incident Cases of CF, All Ages, N, 2015 (Adjusted for Underestimation)
Figure 15: 7MM, Diagnosed Prevalent Cases of CF, All Ages, Both Sexes, N, Selected Years 2015-2025 (Adjusted for Underestimation)
Figure 16: 7MM, Age-Specific Diagnosed Prevalent Cases of CF, Both Sexes, N, 2015 (Adjusted for Underestimation)
Figure 17: 7MM, Sex-Specific Diagnosed Prevalent Cases of CF, All Ages, N, 2015 (Adjusted for Underestimation)
Figure 18: 7MM, Diagnosed Prevalent Cases of CF with Specific Mutations, Both Sexes, All Ages, N, 2015 (Adjusted for Underestimation)
Figure 19: 7MM, Diagnosed Prevalent Cases of CF with Chronic MRSA Infection, Both Sexes, All Ages, N, 2015 (Adjusted for Underestimation)
Figure 20: 7MM, CF Deaths, All Ages, Both Sexes, N, Selected Years 2015-2025 (Adjusted for Underestimation)
Figure 21: 7MM, Sex-Specific CF Deaths, All Ages, N, 2015 (Adjusted for Underestimation)
Figure 22: Competitive Assessment of Marketed and Pipeline Agents in CF, 2015-2025
Figure 23: Competitive Assessment of Marketed and Pipeline Agents in CF, 2015-2025
Figure 24: Top-Line Sales for CF by Country/Region, 2015 and 2025
Figure 25: Global Sales for CF by Drug Class, 2015 and 2025
Figure 26: Sales for CF by Drug Class in the US, 2015 and 2025
Figure 27: Sales for CF by Drug Class in the 5EU, 2015 and 2025
Figure 28: Sales for CF by Drug Class in Canada, 2015 and 2025

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