Disease Overview
Cystic fibrosis, caused by mutations in cystic fibrosis transmembrane conductance regulator (CFTR) protein, is a rare and life-limiting genetic disorder that is characterized by thick, sticky mucus and pulmonary complications. The prognosis of patients has improved markedly over recent decades with the advent of new treatments, increasing the mean life expectancy of patients to 37 years. These include inhaled antibiotics for managing chronic lung infections, as well as mucolytics that facilitate the breakdown and clearance of mucus from the lungs, improving airflow and decreasing the risk of infection.
Recent events and opinion
Cystic fibrosis, caused by mutations in cystic fibrosis transmembrane conductance regulator (CFTR) protein, is a rare and life-limiting genetic disorder that is characterized by thick, sticky mucus and pulmonary complications. The prognosis of patients has improved markedly over recent decades with the advent of new treatments, increasing the mean life expectancy of patients to 37 years. These include inhaled antibiotics for managing chronic lung infections, as well as mucolytics that facilitate the breakdown and clearance of mucus from the lungs, improving airflow and decreasing the risk of infection.
Recent events and opinion
- Vertex is building a durable $7bn portfolio of CFTR modulators.
- Uptake of latest wave of therapies will uphold improvements in prognosis and life expectancy seen in recent decades.
- Prevalent cases are expected to increase over the forecast period mainly due to improving survival rates.
- Vertex’s dominance of the cystic fibrosis market is set to continue.
- Triple combination therapies with Kalydeco-like efficacy and broad label coverage set foundation for blockbuster status.
Table of Contents
FORECAST: CYSTIC FIBROSIS (Published on 03 September 2018)
Overview
Market Dynamics
Forecast And Future Trends
Market Definition And Methodology
Primary Research Methodology
Bibliography
Product Profile: Bronchitol
Product Profile: Cayston
Product Profile: Colobreathe
Product Profile: Kalydeco
Product Profile: Orkambi
Product Profile: Pulmozyme
Product Profile: Quinsair
Product Profile: Symdeko
Product Profile: Tobi/Tobi Podhaler
Product Profile (Late Stage): Vertex Triple Combination Program
Market Dynamics
Forecast And Future Trends
Market Definition And Methodology
Primary Research Methodology
Bibliography
Product Profile: Bronchitol
Product Profile: Cayston
Product Profile: Colobreathe
Product Profile: Kalydeco
Product Profile: Orkambi
Product Profile: Pulmozyme
Product Profile: Quinsair
Product Profile: Symdeko
Product Profile: Tobi/Tobi Podhaler
Product Profile (Late Stage): Vertex Triple Combination Program
TREATMENT: CYSTIC FIBROSIS (Published on 03 September 2018)
Overview
Executive Summary
Primary Research Methodology
Disease Definition And Diagnosis
Patient Segmentation
Current Treatment Options
Prescribing Trends
Unmet Needs In Cystic Fibrosis
Executive Summary
Primary Research Methodology
Disease Definition And Diagnosis
Patient Segmentation
Current Treatment Options
Prescribing Trends
Unmet Needs In Cystic Fibrosis
EPIDEMIOLOGY: CYSTIC FIBROSIS (Published on 06 September 2018)
Overview
Disease Background
Methodology
Forecast
Bibliography
Appendix
Disease Background
Methodology
Forecast
Bibliography
Appendix
MARKETED DRUGS: CYSTIC FIBROSIS (Published on 03 September 2018)
Overview
Product Overview
Product Profile: Bronchitol
Product Profile: Cayston
Product Profile: Colobreathe
Product Profile: Kalydeco
Product Profile: Orkambi
Product Profile: Pulmozyme
Product Profile: Quinsair
Product Profile: Symdeko
Product Profile: Tobi/Tobi Podhaler
Product Overview
Product Profile: Bronchitol
Product Profile: Cayston
Product Profile: Colobreathe
Product Profile: Kalydeco
Product Profile: Orkambi
Product Profile: Pulmozyme
Product Profile: Quinsair
Product Profile: Symdeko
Product Profile: Tobi/Tobi Podhaler
PIPELINE: CYSTIC FIBROSIS (Published on 03 September 2018)
Overview
Clinical Pipeline Overview
Additional Pharma Intelligence Pipeline Resources
Product Profile (Late Stage): Arikayce
Product Profile (Late Stage): Vertex Triple Combination Program
Clinical Pipeline Overview
Additional Pharma Intelligence Pipeline Resources
Product Profile (Late Stage): Arikayce
Product Profile (Late Stage): Vertex Triple Combination Program
LIST OF FIGURES
Figure 1: Cystic fibrosis – current and future market dynamics analysis
Figure 2: The authors assessment summary of key marketed and pipeline drugs for cystic fibrosis
Figure 3: Cystic fibrosis drug sales across the US and five major EU markets, by country, 2016–25
Figure 4: Growth dynamics of the cystic fibrosis market in the US and five major EU markets, by country, 2016–25
Figure 5: Cystic fibrosis drug sales across the US and five major EU markets, by drug class, 2016–25
Figure 6: Methodology flow for The authors cystic fibrosis forecast
Figure 7: Price sources and calculations, by country
Figure 8: Bronchitol for cystic fibrosis – SWOT analysis
Figure 9: The authors drug assessment summary of Bronchitol for cystic fibrosis
Figure 10: The authors drug assessment summary of Bronchitol for cystic fibrosis
Figure 11: Bronchitol sales for cystic fibrosis across the US, Germany, and the UK, by country, 2016–25
Figure 12: Cayston for cystic fibrosis – SWOT analysis
Figure 13: The authors drug assessment summary of Cayston for cystic fibrosis
Figure 14: The authors drug assessment summary of Cayston for cystic fibrosis
Figure 15: Cayston sales for cystic fibrosis across the US and five major EU markets, by country, 2016–25
Figure 16: Colobreathe for cystic fibrosis – SWOT analysis
Figure 17: The authors drug assessment summary of Colobreathe for cystic fibrosis
Figure 18: The authors drug assessment summary of Colobreathe for cystic fibrosis
Figure 19: Colobreathe sales for cystic fibrosis across the five major EU markets, by country, 2016–25
Figure 20: Kalydeco for cystic fibrosis – SWOT analysis
Figure 21: The authors drug assessment summary of Kalydeco for cystic fibrosis
Figure 22: The authors drug assessment summary of Kalydeco for cystic fibrosis
Figure 23: Kalydeco sales for cystic fibrosis across the US and five major EU markets, by country, 2016–25
Figure 24: Orkambi for cystic fibrosis – SWOT analysis
Figure 25: The authors drug assessment summary of Orkambi for cystic fibrosis
Figure 26: The authors drug assessment summary of Orkambi for cystic fibrosis
Figure 27: Orkambi sales for cystic fibrosis across the US and five major EU markets, by country, 2016–25
Figure 28: Pulmozyme for cystic fibrosis – SWOT analysis
Figure 29: The authors drug assessment summary of Pulmozyme for cystic fibrosis
Figure 30: The authors drug assessment summary of Pulmozyme for cystic fibrosis
Figure 31: Pulmozyme sales for cystic fibrosis across the US and five major EU markets, by country, 2016–25
Figure 32: Quinsair for cystic fibrosis – SWOT analysis
Figure 33: The authors drug assessment summary of Quinsair for cystic fibrosis
Figure 34: The authors drug assessment summary of Quinsair for cystic fibrosis
Figure 35: Quinsair sales for cystic fibrosis across the five major EU markets, by country, 2016–25
Figure 36: Symdeko for cystic fibrosis – SWOT analysis
Figure 37: The authors drug assessment summary of Symdeko for cystic fibrosis
Figure 38: The authors drug assessment summary of Symdeko for cystic fibrosis
Figure 39: Symdeko sales for cystic fibrosis across the US and five major EU markets, by country, 2016–25
Figure 40: Tobi/Tobi Podhaler for cystic fibrosis – SWOT analysis
Figure 41: The authors drug assessment summary of Tobi/Tobi Podhaler for cystic fibrosis
Figure 42: The authors drug assessment summary of Tobi/Tobi Podhaler for cystic fibrosis
Figure 43: Tobi/Tobi Podhaler sales for cystic fibrosis across the US and five major EU markets, by country, 2016–25
Figure 44: Vertex triple combination program for cystic fibrosis – SWOT analysis
Figure 45: The authors drug assessment summary of Vertex triple combination program for cystic fibrosis
Figure 46: The authors drug assessment summary of Vertex triple combination program for cystic fibrosis
Figure 47: Vertex triple combination program sales for cystic fibrosis across the US and five major EU markets, by country, 2016–25
Figure 48: Proportion of cystic fibrosis patients with an unknown mutation status, 2016
Figure 49: Prevalence of F508del mutations among surveyed pulmonologists, by country
Figure 50: Use of airway clearance techniques among surveyed pulmonologists’ cystic fibrosis patients, by country, 2016
Figure 51: Treatments for pancreatic symptoms among surveyed pulmonologists’ cystic fibrosis patients, by country, 2016
Figure 52: Treatment strategies for pulmonary symptoms among surveyed pulmonologists’ cystic fibrosis patients, by country, 2016
Figure 53: Top five therapies among cystic fibrosis patients receiving chronic antibiotics, 2016
Figure 54: Top therapies among cystic fibrosis patients receiving mucolytics, 2016
Figure 55: Top therapies among cystic fibrosis patients receiving CFTR modulators, 2016
Figure 56: Top five therapies among cystic fibrosis patients receiving bronchodilators, 2016
Figure 57: Compliance rates of cystic fibrosis patients, by therapy type and country, 2016
Figure 58: Treatment rates for non-pulmonary symptoms among surveyed pulmonologists’ cystic fibrosis patients, by country, 2016
Figure 59: Key unmet needs for cystic fibrosis, 2016
Figure 60: Trends in diagnosed prevalent cases of cystic fibrosis in the US and five major EU markets, by country, 2017–37
Figure 61: Bronchitol for cystic fibrosis – SWOT analysis
Figure 62: The authors drug assessment summary of Bronchitol for cystic fibrosis
Figure 63: The authors drug assessment summary of Bronchitol for cystic fibrosis
Figure 64: Bronchitol sales for cystic fibrosis across the US, Germany, and the UK, by country, 2016–25
Figure 65: Cayston for cystic fibrosis – SWOT analysis
Figure 66: The authors drug assessment summary of Cayston for cystic fibrosis
Figure 67: The authors drug assessment summary of Cayston for cystic fibrosis
Figure 68: Cayston sales for cystic fibrosis across the US and five major EU markets, by country, 2016–25
Figure 69: Colobreathe for cystic fibrosis – SWOT analysis
Figure 70: The authors drug assessment summary of Colobreathe for cystic fibrosis
Figure 71: The authors drug assessment summary of Colobreathe for cystic fibrosis
Figure 72: Colobreathe sales for cystic fibrosis across the five major EU markets, by country, 2016–25
Figure 73: Kalydeco for cystic fibrosis – SWOT analysis
Figure 74: The authors drug assessment summary of Kalydeco for cystic fibrosis
Figure 75: The authors drug assessment summary of Kalydeco for cystic fibrosis
Figure 76: Kalydeco sales for cystic fibrosis across the US and five major EU markets, by country, 2016–25
Figure 77: Orkambi for cystic fibrosis – SWOT analysis
Figure 78: The authors drug assessment summary of Orkambi for cystic fibrosis
Figure 79: The authors drug assessment summary of Orkambi for cystic fibrosis
Figure 80: Orkambi sales for cystic fibrosis across the US and five major EU markets, by country, 2016–25
Figure 81: Pulmozyme for cystic fibrosis – SWOT analysis
Figure 82: The authors drug assessment summary of Pulmozyme for cystic fibrosis
Figure 83: The authors drug assessment summary of Pulmozyme for cystic fibrosis
Figure 84: Pulmozyme sales for cystic fibrosis across the US and five major EU markets, by country, 2016–25
Figure 85: Quinsair for cystic fibrosis – SWOT analysis
Figure 86: The authors drug assessment summary of Quinsair for cystic fibrosis
Figure 87: The authors drug assessment summary of Quinsair for cystic fibrosis
Figure 88: Quinsair sales for cystic fibrosis across the five major EU markets, by country, 2016–25
Figure 89: Symdeko for cystic fibrosis – SWOT analysis
Figure 90: The authors drug assessment summary of Symdeko for cystic fibrosis
Figure 91: The authors drug assessment summary of Symdeko for cystic fibrosis
Figure 92: Symdeko sales for cystic fibrosis across the US and five major EU markets, by country, 2016–25
Figure 93: Tobi/Tobi Podhaler for cystic fibrosis – SWOT analysis
Figure 94: The authors drug assessment summary of Tobi/Tobi Podhaler for cystic fibrosis
Figure 95: The authors drug assessment summary of Tobi/Tobi Podhaler for cystic fibrosis
Figure 96: Tobi/Tobi Podhaler sales for cystic fibrosis across the US and five major EU markets, by country, 2016–25
Figure 97: Arikayce for cystic fibrosis – SWOT analysis
Figure 98: The authors drug assessment summary of Arikayce for cystic fibrosis
Figure 99: The authors drug assessment summary of Arikayce for cystic fibrosis
Figure 100: Vertex triple combination program for cystic fibrosis – SWOT analysis
Figure 101: The authors drug assessment summary of Vertex triple combination program for cystic fibrosis
Figure 102: The authors drug assessment summary of Vertex triple combination program for cystic fibrosis
Figure 103: Vertex triple combination program sales for cystic fibrosis across the US and five major EU markets, by country, 2016–25
Figure 2: The authors assessment summary of key marketed and pipeline drugs for cystic fibrosis
Figure 3: Cystic fibrosis drug sales across the US and five major EU markets, by country, 2016–25
Figure 4: Growth dynamics of the cystic fibrosis market in the US and five major EU markets, by country, 2016–25
Figure 5: Cystic fibrosis drug sales across the US and five major EU markets, by drug class, 2016–25
Figure 6: Methodology flow for The authors cystic fibrosis forecast
Figure 7: Price sources and calculations, by country
Figure 8: Bronchitol for cystic fibrosis – SWOT analysis
Figure 9: The authors drug assessment summary of Bronchitol for cystic fibrosis
Figure 10: The authors drug assessment summary of Bronchitol for cystic fibrosis
Figure 11: Bronchitol sales for cystic fibrosis across the US, Germany, and the UK, by country, 2016–25
Figure 12: Cayston for cystic fibrosis – SWOT analysis
Figure 13: The authors drug assessment summary of Cayston for cystic fibrosis
Figure 14: The authors drug assessment summary of Cayston for cystic fibrosis
Figure 15: Cayston sales for cystic fibrosis across the US and five major EU markets, by country, 2016–25
Figure 16: Colobreathe for cystic fibrosis – SWOT analysis
Figure 17: The authors drug assessment summary of Colobreathe for cystic fibrosis
Figure 18: The authors drug assessment summary of Colobreathe for cystic fibrosis
Figure 19: Colobreathe sales for cystic fibrosis across the five major EU markets, by country, 2016–25
Figure 20: Kalydeco for cystic fibrosis – SWOT analysis
Figure 21: The authors drug assessment summary of Kalydeco for cystic fibrosis
Figure 22: The authors drug assessment summary of Kalydeco for cystic fibrosis
Figure 23: Kalydeco sales for cystic fibrosis across the US and five major EU markets, by country, 2016–25
Figure 24: Orkambi for cystic fibrosis – SWOT analysis
Figure 25: The authors drug assessment summary of Orkambi for cystic fibrosis
Figure 26: The authors drug assessment summary of Orkambi for cystic fibrosis
Figure 27: Orkambi sales for cystic fibrosis across the US and five major EU markets, by country, 2016–25
Figure 28: Pulmozyme for cystic fibrosis – SWOT analysis
Figure 29: The authors drug assessment summary of Pulmozyme for cystic fibrosis
Figure 30: The authors drug assessment summary of Pulmozyme for cystic fibrosis
Figure 31: Pulmozyme sales for cystic fibrosis across the US and five major EU markets, by country, 2016–25
Figure 32: Quinsair for cystic fibrosis – SWOT analysis
Figure 33: The authors drug assessment summary of Quinsair for cystic fibrosis
Figure 34: The authors drug assessment summary of Quinsair for cystic fibrosis
Figure 35: Quinsair sales for cystic fibrosis across the five major EU markets, by country, 2016–25
Figure 36: Symdeko for cystic fibrosis – SWOT analysis
Figure 37: The authors drug assessment summary of Symdeko for cystic fibrosis
Figure 38: The authors drug assessment summary of Symdeko for cystic fibrosis
Figure 39: Symdeko sales for cystic fibrosis across the US and five major EU markets, by country, 2016–25
Figure 40: Tobi/Tobi Podhaler for cystic fibrosis – SWOT analysis
Figure 41: The authors drug assessment summary of Tobi/Tobi Podhaler for cystic fibrosis
Figure 42: The authors drug assessment summary of Tobi/Tobi Podhaler for cystic fibrosis
Figure 43: Tobi/Tobi Podhaler sales for cystic fibrosis across the US and five major EU markets, by country, 2016–25
Figure 44: Vertex triple combination program for cystic fibrosis – SWOT analysis
Figure 45: The authors drug assessment summary of Vertex triple combination program for cystic fibrosis
Figure 46: The authors drug assessment summary of Vertex triple combination program for cystic fibrosis
Figure 47: Vertex triple combination program sales for cystic fibrosis across the US and five major EU markets, by country, 2016–25
Figure 48: Proportion of cystic fibrosis patients with an unknown mutation status, 2016
Figure 49: Prevalence of F508del mutations among surveyed pulmonologists, by country
Figure 50: Use of airway clearance techniques among surveyed pulmonologists’ cystic fibrosis patients, by country, 2016
Figure 51: Treatments for pancreatic symptoms among surveyed pulmonologists’ cystic fibrosis patients, by country, 2016
Figure 52: Treatment strategies for pulmonary symptoms among surveyed pulmonologists’ cystic fibrosis patients, by country, 2016
Figure 53: Top five therapies among cystic fibrosis patients receiving chronic antibiotics, 2016
Figure 54: Top therapies among cystic fibrosis patients receiving mucolytics, 2016
Figure 55: Top therapies among cystic fibrosis patients receiving CFTR modulators, 2016
Figure 56: Top five therapies among cystic fibrosis patients receiving bronchodilators, 2016
Figure 57: Compliance rates of cystic fibrosis patients, by therapy type and country, 2016
Figure 58: Treatment rates for non-pulmonary symptoms among surveyed pulmonologists’ cystic fibrosis patients, by country, 2016
Figure 59: Key unmet needs for cystic fibrosis, 2016
Figure 60: Trends in diagnosed prevalent cases of cystic fibrosis in the US and five major EU markets, by country, 2017–37
Figure 61: Bronchitol for cystic fibrosis – SWOT analysis
Figure 62: The authors drug assessment summary of Bronchitol for cystic fibrosis
Figure 63: The authors drug assessment summary of Bronchitol for cystic fibrosis
Figure 64: Bronchitol sales for cystic fibrosis across the US, Germany, and the UK, by country, 2016–25
Figure 65: Cayston for cystic fibrosis – SWOT analysis
Figure 66: The authors drug assessment summary of Cayston for cystic fibrosis
Figure 67: The authors drug assessment summary of Cayston for cystic fibrosis
Figure 68: Cayston sales for cystic fibrosis across the US and five major EU markets, by country, 2016–25
Figure 69: Colobreathe for cystic fibrosis – SWOT analysis
Figure 70: The authors drug assessment summary of Colobreathe for cystic fibrosis
Figure 71: The authors drug assessment summary of Colobreathe for cystic fibrosis
Figure 72: Colobreathe sales for cystic fibrosis across the five major EU markets, by country, 2016–25
Figure 73: Kalydeco for cystic fibrosis – SWOT analysis
Figure 74: The authors drug assessment summary of Kalydeco for cystic fibrosis
Figure 75: The authors drug assessment summary of Kalydeco for cystic fibrosis
Figure 76: Kalydeco sales for cystic fibrosis across the US and five major EU markets, by country, 2016–25
Figure 77: Orkambi for cystic fibrosis – SWOT analysis
Figure 78: The authors drug assessment summary of Orkambi for cystic fibrosis
Figure 79: The authors drug assessment summary of Orkambi for cystic fibrosis
Figure 80: Orkambi sales for cystic fibrosis across the US and five major EU markets, by country, 2016–25
Figure 81: Pulmozyme for cystic fibrosis – SWOT analysis
Figure 82: The authors drug assessment summary of Pulmozyme for cystic fibrosis
Figure 83: The authors drug assessment summary of Pulmozyme for cystic fibrosis
Figure 84: Pulmozyme sales for cystic fibrosis across the US and five major EU markets, by country, 2016–25
Figure 85: Quinsair for cystic fibrosis – SWOT analysis
Figure 86: The authors drug assessment summary of Quinsair for cystic fibrosis
Figure 87: The authors drug assessment summary of Quinsair for cystic fibrosis
Figure 88: Quinsair sales for cystic fibrosis across the five major EU markets, by country, 2016–25
Figure 89: Symdeko for cystic fibrosis – SWOT analysis
Figure 90: The authors drug assessment summary of Symdeko for cystic fibrosis
Figure 91: The authors drug assessment summary of Symdeko for cystic fibrosis
Figure 92: Symdeko sales for cystic fibrosis across the US and five major EU markets, by country, 2016–25
Figure 93: Tobi/Tobi Podhaler for cystic fibrosis – SWOT analysis
Figure 94: The authors drug assessment summary of Tobi/Tobi Podhaler for cystic fibrosis
Figure 95: The authors drug assessment summary of Tobi/Tobi Podhaler for cystic fibrosis
Figure 96: Tobi/Tobi Podhaler sales for cystic fibrosis across the US and five major EU markets, by country, 2016–25
Figure 97: Arikayce for cystic fibrosis – SWOT analysis
Figure 98: The authors drug assessment summary of Arikayce for cystic fibrosis
Figure 99: The authors drug assessment summary of Arikayce for cystic fibrosis
Figure 100: Vertex triple combination program for cystic fibrosis – SWOT analysis
Figure 101: The authors drug assessment summary of Vertex triple combination program for cystic fibrosis
Figure 102: The authors drug assessment summary of Vertex triple combination program for cystic fibrosis
Figure 103: Vertex triple combination program sales for cystic fibrosis across the US and five major EU markets, by country, 2016–25
LIST OF TABLES
Table 1: Cystic fibrosis drug sales across the US and five major EU markets, by country ($m), 2016–25
Table 2: Cystic fibrosis drug sales across the US and five major EU markets, by drug class ($m), 2016–25
Table 3: Exchange rates used for calculating prices
Table 4: Pulmonologists and pediatric pulmonologists surveyed for the cystic fibrosis primary research study, 2016
Table 5: Therapies included within cystic fibrosis key opinion leader discussions
Table 6: Bronchitol drug profile
Table 7: Bronchitol Phase III data in cystic fibrosis
Table 8: Bronchitol sales for cystic fibrosis across the US, Germany, and the UK, by country ($m), 2016–25
Table 9: Bronchitol sales for cystic fibrosis across the US, Germany, and the UK, by country ($m), 2016–25
Table 10: Cayston drug profile
Table 11: Cayston Phase III data in cystic fibrosis
Table 12: Cayston sales for cystic fibrosis across the US and five major EU markets, by country ($m), 2016–25
Table 13: Colobreathe drug profile
Table 14: Colobreathe Phase III data in cystic fibrosis
Table 15: Colobreathe sales for cystic fibrosis across the five major EU markets, by country ($m), 2016–25
Table 16: Kalydeco drug profile
Table 17: Kalydeco Phase III data in cystic fibrosis
Table 18: Kalydeco sales for cystic fibrosis across the US and five major EU markets, by country ($m), 2016–25
Table 19: Orkambi drug profile
Table 20: Orkambi Phase III data in cystic fibrosis
Table 21: Orkambi sales for cystic fibrosis across the US and five major EU markets, by country ($m), 2016–25
Table 22: Pulmozyme drug profile
Table 23: Pulmozyme pivotal trial data in cystic fibrosis
Table 24: Pulmozyme sales for cystic fibrosis across the US and five major EU markets, by country ($m), 2016–25
Table 25: Quinsair drug profile
Table 26: Quinsair Phase IIb/III data in cystic fibrosis
Table 27: Quinsair sales for cystic fibrosis across the five major EU markets, by country ($m), 2016–25
Table 28: Symdeko drug profile
Table 29: Symdeko Phase III data in cystic fibrosis
Table 30: Symdeko sales for cystic fibrosis across the US and five major EU markets, by country ($m), 2016–25
Table 31: Tobi/Tobi Podhaler drug profile
Table 32: Tobi/Tobi Podhaler Phase III data in cystic fibrosis
Table 33: Tobi/Tobi Podhaler sales for cystic fibrosis across the US and five major EU markets, by country ($m), 2016–25
Table 34: Vertex triple combination program drug profile
Table 35: Vertex triple combination program Phase II data in cystic fibrosis
Table 36: Vertex triple combination program Phase III data in cystic fibrosis
Table 37: Vertex triple combination program sales for cystic fibrosis across the US and five major EU markets, by country ($m), 2016–25
Table 38: Pulmonologists and pediatric pulmonologists surveyed for the cystic fibrosis primary research study, 2016
Table 39: CFTR mutational classes and their impact on CFTR structure and function
Table 40: Phenotypic features consistent with a diagnosis of cystic fibrosis
Table 41: Approved treatments available for cystic fibrosis across the US and five major EU markets
Table 42: Cystic Fibrosis Foundation major treatment recommendations
Table 43: Patient shares of approved antibiotics for cystic fibrosis among patients receiving chronic antibiotic treatment, by country (%), 2016
Table 44: Patient shares of therapies among patients receiving treatment for pancreatic symptoms (%), 2016
Table 45: Sources used for epidemiological analysis of cystic fibrosis in the US and five major EU markets, by country
Table 46: Diagnosed prevalent cases of cystic fibrosis in the US and five major EU markets, by country, 2017–37
Table 47: Profiled key marketed drugs for cystic fibrosis
Table 48: Bronchitol drug profile
Table 49: Bronchitol Phase III data in cystic fibrosis
Table 50: Bronchitol sales for cystic fibrosis across the US, Germany, and the UK, by country ($m), 2016–25
Table 51: Bronchitol sales for cystic fibrosis across the US, Germany, and the UK, by country ($m), 2016–25
Table 52: Cayston drug profile
Table 53: Cayston Phase III data in cystic fibrosis
Table 54: Cayston sales for cystic fibrosis across the US and five major EU markets, by country ($m), 2016–25
Table 55: Colobreathe drug profile
Table 56: Colobreathe Phase III data in cystic fibrosis
Table 57: Colobreathe sales for cystic fibrosis across the five major EU markets, by country ($m), 2016–25
Table 58: Kalydeco drug profile
Table 59: Kalydeco Phase III data in cystic fibrosis
Table 60: Kalydeco sales for cystic fibrosis across the US and five major EU markets, by country ($m), 2016–25
Table 61: Orkambi drug profile
Table 62: Orkambi Phase III data in cystic fibrosis
Table 63: Orkambi sales for cystic fibrosis across the US and five major EU markets, by country ($m), 2016–25
Table 64: Pulmozyme drug profile
Table 65: Pulmozyme pivotal trial data in cystic fibrosis
Table 66: Pulmozyme sales for cystic fibrosis across the US and five major EU markets, by country ($m), 2016–25
Table 67: Quinsair drug profile
Table 68: Quinsair Phase IIb/III data in cystic fibrosis
Table 69: Quinsair sales for cystic fibrosis across the five major EU markets, by country ($m), 2016–25
Table 70: Symdeko drug profile
Table 71: Symdeko Phase III data in cystic fibrosis
Table 72: Symdeko sales for cystic fibrosis across the US and five major EU markets, by country ($m), 2016–25
Table 73: Tobi/Tobi Podhaler drug profile
Table 74: Tobi/Tobi Podhaler Phase III data in cystic fibrosis
Table 75: Tobi/Tobi Podhaler sales for cystic fibrosis across the US and five major EU markets, by country ($m), 2016–25
Table 76: Profiled pipeline products in development for cystic fibrosis
Table 77: Arikayce drug profile
Table 78: Arikayce Phase III data in cystic fibrosis
Table 79: Vertex triple combination program drug profile
Table 80: Vertex triple combination program Phase II data in cystic fibrosis
Table 81: Vertex triple combination program Phase III data in cystic fibrosis
Table 82: Vertex triple combination program sales for cystic fibrosis across the US and five major EU markets, by country ($m), 2016–25
Table 2: Cystic fibrosis drug sales across the US and five major EU markets, by drug class ($m), 2016–25
Table 3: Exchange rates used for calculating prices
Table 4: Pulmonologists and pediatric pulmonologists surveyed for the cystic fibrosis primary research study, 2016
Table 5: Therapies included within cystic fibrosis key opinion leader discussions
Table 6: Bronchitol drug profile
Table 7: Bronchitol Phase III data in cystic fibrosis
Table 8: Bronchitol sales for cystic fibrosis across the US, Germany, and the UK, by country ($m), 2016–25
Table 9: Bronchitol sales for cystic fibrosis across the US, Germany, and the UK, by country ($m), 2016–25
Table 10: Cayston drug profile
Table 11: Cayston Phase III data in cystic fibrosis
Table 12: Cayston sales for cystic fibrosis across the US and five major EU markets, by country ($m), 2016–25
Table 13: Colobreathe drug profile
Table 14: Colobreathe Phase III data in cystic fibrosis
Table 15: Colobreathe sales for cystic fibrosis across the five major EU markets, by country ($m), 2016–25
Table 16: Kalydeco drug profile
Table 17: Kalydeco Phase III data in cystic fibrosis
Table 18: Kalydeco sales for cystic fibrosis across the US and five major EU markets, by country ($m), 2016–25
Table 19: Orkambi drug profile
Table 20: Orkambi Phase III data in cystic fibrosis
Table 21: Orkambi sales for cystic fibrosis across the US and five major EU markets, by country ($m), 2016–25
Table 22: Pulmozyme drug profile
Table 23: Pulmozyme pivotal trial data in cystic fibrosis
Table 24: Pulmozyme sales for cystic fibrosis across the US and five major EU markets, by country ($m), 2016–25
Table 25: Quinsair drug profile
Table 26: Quinsair Phase IIb/III data in cystic fibrosis
Table 27: Quinsair sales for cystic fibrosis across the five major EU markets, by country ($m), 2016–25
Table 28: Symdeko drug profile
Table 29: Symdeko Phase III data in cystic fibrosis
Table 30: Symdeko sales for cystic fibrosis across the US and five major EU markets, by country ($m), 2016–25
Table 31: Tobi/Tobi Podhaler drug profile
Table 32: Tobi/Tobi Podhaler Phase III data in cystic fibrosis
Table 33: Tobi/Tobi Podhaler sales for cystic fibrosis across the US and five major EU markets, by country ($m), 2016–25
Table 34: Vertex triple combination program drug profile
Table 35: Vertex triple combination program Phase II data in cystic fibrosis
Table 36: Vertex triple combination program Phase III data in cystic fibrosis
Table 37: Vertex triple combination program sales for cystic fibrosis across the US and five major EU markets, by country ($m), 2016–25
Table 38: Pulmonologists and pediatric pulmonologists surveyed for the cystic fibrosis primary research study, 2016
Table 39: CFTR mutational classes and their impact on CFTR structure and function
Table 40: Phenotypic features consistent with a diagnosis of cystic fibrosis
Table 41: Approved treatments available for cystic fibrosis across the US and five major EU markets
Table 42: Cystic Fibrosis Foundation major treatment recommendations
Table 43: Patient shares of approved antibiotics for cystic fibrosis among patients receiving chronic antibiotic treatment, by country (%), 2016
Table 44: Patient shares of therapies among patients receiving treatment for pancreatic symptoms (%), 2016
Table 45: Sources used for epidemiological analysis of cystic fibrosis in the US and five major EU markets, by country
Table 46: Diagnosed prevalent cases of cystic fibrosis in the US and five major EU markets, by country, 2017–37
Table 47: Profiled key marketed drugs for cystic fibrosis
Table 48: Bronchitol drug profile
Table 49: Bronchitol Phase III data in cystic fibrosis
Table 50: Bronchitol sales for cystic fibrosis across the US, Germany, and the UK, by country ($m), 2016–25
Table 51: Bronchitol sales for cystic fibrosis across the US, Germany, and the UK, by country ($m), 2016–25
Table 52: Cayston drug profile
Table 53: Cayston Phase III data in cystic fibrosis
Table 54: Cayston sales for cystic fibrosis across the US and five major EU markets, by country ($m), 2016–25
Table 55: Colobreathe drug profile
Table 56: Colobreathe Phase III data in cystic fibrosis
Table 57: Colobreathe sales for cystic fibrosis across the five major EU markets, by country ($m), 2016–25
Table 58: Kalydeco drug profile
Table 59: Kalydeco Phase III data in cystic fibrosis
Table 60: Kalydeco sales for cystic fibrosis across the US and five major EU markets, by country ($m), 2016–25
Table 61: Orkambi drug profile
Table 62: Orkambi Phase III data in cystic fibrosis
Table 63: Orkambi sales for cystic fibrosis across the US and five major EU markets, by country ($m), 2016–25
Table 64: Pulmozyme drug profile
Table 65: Pulmozyme pivotal trial data in cystic fibrosis
Table 66: Pulmozyme sales for cystic fibrosis across the US and five major EU markets, by country ($m), 2016–25
Table 67: Quinsair drug profile
Table 68: Quinsair Phase IIb/III data in cystic fibrosis
Table 69: Quinsair sales for cystic fibrosis across the five major EU markets, by country ($m), 2016–25
Table 70: Symdeko drug profile
Table 71: Symdeko Phase III data in cystic fibrosis
Table 72: Symdeko sales for cystic fibrosis across the US and five major EU markets, by country ($m), 2016–25
Table 73: Tobi/Tobi Podhaler drug profile
Table 74: Tobi/Tobi Podhaler Phase III data in cystic fibrosis
Table 75: Tobi/Tobi Podhaler sales for cystic fibrosis across the US and five major EU markets, by country ($m), 2016–25
Table 76: Profiled pipeline products in development for cystic fibrosis
Table 77: Arikayce drug profile
Table 78: Arikayce Phase III data in cystic fibrosis
Table 79: Vertex triple combination program drug profile
Table 80: Vertex triple combination program Phase II data in cystic fibrosis
Table 81: Vertex triple combination program Phase III data in cystic fibrosis
Table 82: Vertex triple combination program sales for cystic fibrosis across the US and five major EU markets, by country ($m), 2016–25
