Product Code: GDHC003EPIDR
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. CF patients have a limited number of available treatment options and significant unmet needs still exist. Opportunities are significant for new therapies that will improve symptoms, change the course of this disease, as well as increase therapy options. The introduction of the first disease modifying therapy, Kalydeco (ivacaftor), by Vertex in 2012, paved the way for a new class of therapies known as cystic fibrosis transmembrane conductance regulator (CFTR) modulators. On June 24, 2014, Vertex announced positive Phase III clinical trial data for lumacaftor in combination with Kalydeco in patients with CF who have two copies of the F508del mutation. This novel combination therapy of CFTR modulators has the potential to treat the underlying cause of the disease for approximately 50% of all CF patients and drive rapid growth in the CF market.
Key Questions Answered
- What are the pressing unmet needs and remaining opportunities in the CF market?
- What R&D strategies are drug developers pursuing in the CF therapeutics space?
- What is the significance of CFTR modulators in the CF market?
- What is the clinical and commercial positioning of key pipeline products in the CF market?
- What are the innovative early-stage treatment approaches in the CF market?
- Rapid growth in the CF market is expected from 2012 to 2018.
- 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 novel disease modifying drugs.
- 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 2018.
- 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.
Reasons to buy
- Identify the unmet needs and remaining opportunities in the CF therapeutics market.
- Develop business strategies by understanding the trends shaping and driving the US and five major EU CF therapeutics market.
- Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.
- Assess the clinical and commercial viability of promising pipeline products.
- Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies.
- Formulate effective sales and marketing strategies by understanding the competitive landscape and by analyzing the performance of various emerging therapies.
- Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments and strategic partnerships.
- Drive revenues by understanding the key trends, innovative products and technologies, market and segments likely to impact the US and five major EU CF therapeutics market in future.
Table of Contents
1. Table of Contents
- 1.1. List of Tables
- 1.2. List of Figures
- 2.1. Catalyst
- 2.2. Related Reports
- 2.3. Upcoming Related Reports
3. Disease Overview
- 3.1. Etiology and Pathophysiology
- 3.1.1. Etiology
- 3.1.2. Pathophysiology
- 3.1.3. Prognosis
- 3.1.4. Quality of Life
- 3.2. Symptoms
- 4.1. Risk Factors and Co-morbidities
- 4.2. Global and Historical Trends
- 4.2.1. US
- 4.2.2. France
- 4.2.3. Germany
- 4.2.4. Italy
- 4.2.5. Spain
- 4.2.6. UK
- 4.3. Forecast Methodology
- 4.3.1. Sources Used
- 4.3.2. Forecast Assumptions and Methods
- 4.3.3. Sources Not Used
- 4.4. Epidemiology Forecast
- 4.4.1. Total Prevalent Cases of Cystic Fibrosis
- 4.4.2. Total Prevalent Cases of Cystic Fibrosis Segmented by Age
- 4.4.3. Total Prevalent Cases of Cystic Fibrosis Segmented by Sex
- 4.4.4. Total Prevalent Cases of Cystic Fibrosis by Mutation Type
- 4.5. Discussion
- 4.5.1. Conclusion on Epidemiology Trends
- 4.5.2. Limitations of the Analysis
- 4.5.3. Strengths of the Analysis
5. Current Treatment Options
- 5.1. Overview
- 5.2. Product Profiles - Major Brands, Inhaled Antibiotics
- 5.2.1. TOBI (tobramycin)
- 5.2.2. TOBI Podhaler (tobramycin inhalation powder)
- 5.2.3. Bramitob (tobramycin)
- 5.2.4. Colistimethate Sodium (nebulized; numerous generic names)
- 5.2.5. Colobreathe (colistimethate sodium dry powder)
- 5.2.6. Cayston (aztreonam)
- 5.3. Product Profiles - Major Brands, Mucolytics
- 5.3.1. Pulmozyme (dornase alfa)
- 5.3.2. Bronchitol (mannitol)
- 5.4. Product Profiles - Major Brands, CFTR Modulators
- 5.4.1. Kalydeco (ivacaftor, VX-770)
- 5.5. Product Profiles - Major Brands, Other Therapies
- 5.5.1. Pancreatic Enzyme Replacement Therapies (PERTs)
6. Unmet Needs Assessment and Oppportunity Analysis
- 6.1. Overview
- 6.2. Unmet Needs Analysis
- 6.2.1. The Development of Curative Therapies
- 6.2.2. Improving Treatment of CF-related Lung Infections
- 6.2.3. Improving Airway Clearance with Mucolytic Agents
- 6.2.4. Improving Treatment Compliance
- 6.2.5. The Development of Safe Anti-inflammatory Therapies
- 6.3. Opportunity Analysis
- 6.3.1. Therapies that Target CFTR Protein Function
- 6.3.2. New Classes and Formulations of Inhaled Antibiotics
- 6.3.3. Novel Mucolytic Agents
- 6.3.4. Novel Anti-inflammatory Agents
7. R&D Strategies
- 7.1. Overview
- 7.1.1. Reformulation Strategies
- 7.1.2. Personalized Treatment Approach
- 7.1.3. Diverse Proof-of-Concept Research
- 7.1.4. Licensing and Alliances
- 7.2. Clinical Trial Design
8. Pipeline Assessment
- 8.1. Overview
- 8.2. Promising Drugs in Clinical Development
- 8.3. Promising Drugs in Clinical Development, Inhaled Antibiotics
- 8.3.1. Arikayce (amikacin)
- 8.4. Promising Drugs in Clinical Development, CFTR Modulators
- 8.4.1. Lumacaftor (VX-809)/Kalydeco (VX-770)
- 8.4.2. Ataluren (PTC124)
- 8.5. Innovative Early-Stage Approaches
9. Pipeline Valuation Analysis
- 9.1. Clinical Benchmark of Key Pipeline Drugs
- 9.2. Commercial Benchmark of Key Pipeline Drugs
- 9.3. Competitive Assessment
- 9.4. Top-Line Six-Year Forecast
- 10.1. Bibliography
- 10.2. Abbreviations
- 10.3. Methodology
- 10.4. Forecasting Methodology
- 10.4.1. Diagnosed CF Patients
- 10.4.2. Percent Drug-treated Patients
- 10.4.3. Drugs Included in Each Therapeutic Class
- 10.4.4. Launch and Patent Expiry Dates
- 10.4.5. General Pricing Assumptions
- 10.4.6. Drug Assumptions
- 10.4.7. Generic Erosion
- 10.4.8. Pricing of Pipeline Agents
- 10.5. Physicians and Specialists Included in this Study
- 10.6. About the Authors
- 10.6.1. Analyst
- 10.6.2. Reviewer
- 10.6.3. Epidemiologist
- 10.6.4. Global Head of Healthcare
- 10.7. About GlobalData
- 10.8. Disclaimer
List of Tables
- Table 1: Classes of the CFTR Gene Mutations in Cystic Fibrosis
- Table 2: Symptoms of Cystic Fibrosis
- Table 3: Six Major Markets, Sources of Cystic Fibrosis Prevalence Data
- Table 4: Six Major Markets, Sources of Cystic Fibrosis-Causing Mutation Data
- Table 5: Six Major Markets, Total Prevalent Cases of Cystic Fibrosis, Ages 0-35 Years, Men and Women, Selected Years, 2012-2022
- Table 6: Six Major Markets, Total Prevalent Cases of Cystic Fibrosis, Ages 0-35 Years, By Sex, 2012, N (%)
- Table 7: Six Major Markets, Cystic Fibrosis Prevalent Cases Segmented by Mutation Type, Ages 0-35 Years, Men and Women, 2012
- Table 8: Leading Treatments for Cystic Fibrosis, 2014
- Table 9: Product Profile - TOBI
- Table 10: Product Profile - TOBI Podhaler
- Table 11: Product Profile - Bramitob
- Table 12: Product Profile - Colistimethate Sodium
- Table 13: Product Profile - Colobreathe
- Table 14: Product Profile - Cayston
- Table 15: Product Profile - Pulmozyme
- Table 16: Product Profile - Bronchitol
- Table 17: Product Profile - Kalydeco
- Table 18: Overall Unmet Needs - Current Level of Attainment
- Table 19: Cystic Fibrosis - Late-Stage Pipeline, 2014
- Table 20: Product Profile - Arikayce
- Table 21: Product Profile - Lumacaftor/Kalydeco
- Table 22: Product Profile - Ataluren
- Table 23: Early-Stage Pipeline Products in Cystic Fibrosis, 2014
- Table 24: Clinical Benchmark of Key Pipeline Drugs - Inhaled Antibiotics
- Table 25: Clinical Benchmark of Key Pipeline Drugs - CFTR Modulators
- Table 26: Commercial Benchmark of Key Pipeline Drugs - Inhaled Antibiotics
- Table 27: Commercial Benchmark of Key Pipeline Drugs - CFTR Modulators
- Table 28: Top-Line Sales Forecasts ($m) for Cystic Fibrosis, 2012-2018
- Table 29: Key Events Impacting Sales for Cystic Fibrosis, 2012-2018
- Table 30: Cystic Fibrosis Market - Drivers and Barriers, 2012-2018
- Table 31: Key Launch Dates
- Table 32: Key Patent Expiries
List of Figures
- Figure 1: Six Major Markets, Total Prevalent Cases of Cystic Fibrosis, Ages 0-35 Years, Men and Women, Selected Years, 2012-2022
- Figure 2: Six Major Markets, Total Prevalent Cases of Cystic Fibrosis, By Age, Men and Women, 2012
- Figure 3: Six Major Markets, Cystic Fibrosis Prevalent Cases Segmented by Mutation Type, Ages 0-35 Years, Men and Women, 2012
- Figure 4: Competitive Assessment of Late-Stage Pipeline Inhaled Antibiotics in Cystic Fibrosis, 2012-2018
- Figure 5: Competitive Assessment of Late-Stage Pipeline CFTR Modulators in Cystic Fibrosis, 2012-2018
- Figure 6: Sales for Cystic Fibrosis by Region, 2012-2018