Product Code: GBIHC323MR
GBI Research, a leading business intelligence provider, has released its latest research report "Cystic Fibrosis Therapeutics in Major Developed Markets to 2019 - CFTR Modulators Initiate Drive towards Personalized Treatment and Market Growth". The value of the Cystic Fibrosis (CF) market is expected to increase significantly in value over the forecast period across the leading eight developed nations, from $695.6 million in 2012 to almost $4.5 billion in 2019. This equates to a Compound Annual Growth Rate (CAGR) of 30.4%. Novel treatments with disease-modifying mechanisms of action are the primary factor driving the growth of the value of the market. The positive impact of new market entrants will offset the effects of key patent losses during the forecast period.
Cystic Fibrosis Transmembrane conductance Regulators (CFTR) modulators are the first type of CF therapy to treat the root cause of the disease rather than the symptoms, and as a result, they offer patients improved relief and quality of life. Kalydeco (ivacaftor), which has been marketed since 2012, was the first CFTR modulator to reach the market, and despite only treating a small proportion of the CF population, it had a large impact on the value of the market. This effect will be repeated on a larger scale when lumacaftor and Ataluren (PTC124) enter the market, as these treatments can be used for a much larger proportion of the CF population. Vertex Pharmaceuticals Inc. are the developers of Kalydeco, lumacaftor and other early phase CFTR modulators, and are therefore expected to play a key role in the development of the future CF market.
The pricing of CFTR modulators will be premium due to the clinical benefits and disease-modifying effects that they represent. Symptomatic therapies will be used in conjunction with CFTR modulators, and will still be a necessary part of the CF treatment plan. The antibiotics segment in particular is expected to become overcrowded and competitive over the forecast period, due to cheap, generic tobramycin products and novel market entrants competing for market share.
The report assesses the current CF market, and predicts market trends to 2019, analyzing key drivers and barriers. The areas covered include -
- A disease introduction, which defines the disease, including symptoms, diagnosis and treatment.
- An analysis of the CF marketed landscape, including a comparison of the efficacy and safety of the most prominent brands, as well as the unmet needs of CF treatment.
- A detailed analysis of the CF pipeline, detailing, among other parameters, drug distribution by phase, molecule type and mechanism of action. The CF clinical trial landscape is then analyzed, with a particular emphasis on failure rates across different trial phases as well as the trends in clinical trial size and duration. This section also includes profiles and single product forecasts for the most promising pipeline drugs.
- An epidemiological forecast of the major CF markets. The projected values include total and treated populations.
- An in-depth forecasting model for the CF market, which considers the current marketed therapies, in addition to the potential entry of new products into the market. The model consists of a projected outcome, with high and low variance results, depending on the potential performance of pipeline therapies.
- Analysis of strategic consolidations within the CF indication, including co-development and licensing agreements.
- An overview of the drivers and barriers for the CF market is also included.
Reasons to buy
- The report enables clients to develop a strong understanding of CF as a disease and a market. The current therapeutic needs and future market trends contained in this report will highlight market opportunities and the scope of competition between CF therapeutics. Primarily, the report will allow clients to -
- Understand the CF pipeline and the clinical needs it is addressing. In particular, the importance and benefits of CFTR modulators are highlighted and compared with symptomatic treatments.
- Develop their knowledge of key products that may enter the market before 2019. Detailed profiles of these products are provided, with a focus on their clinical trial performance, as well as how they can be incorporated into the CF treatment plan and what competition they face.
- Assess the risk associated with CF clinical trials compared with both industry and therapy area averages. In addition, in order to understand the trends in both trial size and duration according to mechanism of action or molecule type.
- Identify patterns of growth in the CF market over the forecast period and understand the underlying causes, as well as observing the contributions made by each major market to this growth.
CFTR Modulators Instigate Cystic Fibrosis Market Surge
The Cystic Fibrosis (CF) market will grow rapidly over the forecast period (2012-2019) at a Compound Annual Growth Rate (CAGR) of 30.6%. The sharp rise in market value is due to powerful new drugs entering the market, in particular the novel disease-modifying Cystic Fibrosis Transmembrane conductance Regulator (CFTR) modulators.
Although very expensive, CFTR modulators are the first to act directly on the defective CFTR protein, which is the root cause of CF. As a result they represent the closest therapy to a cure currently available for CF patients. One CFTR modulator, Kalydeco (ivacaftor), is currently on the market, although it can only treat 5% of people with a specific mutation (O'Reilly and Elphick, 2013). Despite the low treatment population, it has already become one of the top-grossing drugs since its launch in 2012. A second CFTR modulator, lumacaftor, is expected to be launched in 2015. This drug is used in combination with Kalydeco and it is expected to be usable in over 80% of the CF population, which, combined with its large cost and uptake is expected to be the primary driver in the CF market surge.
Increasing Life Expectancy Causes Rise in Prevalence
The prevalence of CF will rise slowly over the forecast period by an estimated 10,000 cases. The primary cause of this is the increasing life expectancy of CF patients, which has rapidly grown over the past three decades as the understanding of the causes of CF has developed (Davies et al., 2007).
CF incidence remains relatively constant as it is a hereditary disease; however the mortality rate is decreasing due to improvements in treatments and disease management. Therefore, the overall mortality rate is lower than the birth rate of CF patients and so the CF population is increasing. This will eventually reach equilibrium.
The increasing prevalence is also a factor in terms of driving market growth, as many CF therapeutics must be taken indefinitely and on a regular basis.
Key Drug Expiries Instigate Competition
Several key drug expirations will occur in the forecast period. The drugs set to lose their patent protection are the mucolytic Pulmozyme (dornase alfa) and the antibiotics TOBI (tobramycin) and TOBI Podhaler (tobramycin). Cheap, generic versions of TOBI and TOBI Podhaler are expected to enter the market following their patent expiration, leading to an increase in the number of antibiotics on the market and an increase in competition. The number of Pulmozyme biosimilars and the impact on pricing is expected to be limited.
The antibiotics segment of the market is predicted to become overcrowded in the forecast period, following the launch of several products with similar safety and efficacy profiles and the introduction of generic TOBI and TOBI Podhaler. Rather than one product dominating the antibiotics segment as is currently the case with TOBI, it is likely that each new product will acquire a smaller but more equal market share.
Table of Contents
1. Table of Contents
- 1.1. List of Tables
- 1.2. List of Figures
- 2.1. Disease Introduction
- 2.2. Symptoms
- 2.3. Etiology
- 2.4. Pathophysiology
- 2.5. Diagnosis
- 2.6. Prognosis
- 2.7. Assessment of Disease Severity
- 2.8. Treatment
3. Marketed Products
- 3.1. Overview
- 3.2. Product Profiles
- 3.2.1. Pulmozyme (dornase alfa) - Genentech
- 3.2.2. Bronchitol (mannitol) - Pharmaxis
- 3.2.3. TOBI (tobramycin) - Novartis
- 3.2.4. TOBI Podhaler (tobramycin) - Novartis
- 3.2.5. Bramitob/Bethkis (tobramycin) - Chiesi Farmaceutici
- 3.2.6. Colobreathe (colistimethate sodium) - Forest Laboratories
- 3.2.7. Cayston (aztreonam) - Gilead Sciences
- 3.2.8. Kalydeco (ivacaftor) - Vertex Pharmaceuticals
- 3.2.9. Pancreatic Enzyme Replacement Therapies
- 3.2.10. Other Successful Products
- 3.3. Heat Map of Marketed Products
- 3.4. Conclusion
4. Developmental Pipeline
- 4.1. Overview
- 4.2. Mechanisms of Action in the Pipeline
- 4.3. Clinical Trials
- 4.3.1. Failure Rate
- 4.3.2. Clinical Trial Duration
- 4.3.3. Clinical Trial Size
- 4.4. Promising Pipeline Molecules
- 4.4.1. Lumacaftor (VX809) - Vertex Pharmaceuticals
- 4.4.2. Ataluren (PTC124) - PTC Therapeutics
- 4.4.3. Aeroquin (levofloxacin) - Aptalis Pharma
- 4.4.4. Arikace (amikacin liposomal) - Insmed
- 4.4.5. Other Promising Molecules
- 4.5. Heat Map of Pipeline Products
- 4.6. Conclusion
5. Market Forecast to 2019
- 5.1. Geographical Markets
- 5.1.1. Global Markets
- 5.1.2. North America
- 5.1.3. Top Five EU Countries
- 5.2. Drivers and Barriers
- 5.2.1. Drivers
- 5.2.2. Barriers
6. Strategic Consolidations
- 6.1. Major Co-Development Deals
- 6.2. Major Licensing Deals
- 7.1. References
- 7.1.1. Marketed Product Heat Map References
- 7.1.2. Pipeline Product Heat Map References
- 7.2. Market Definition
- 7.3. Abbreviations
- 7.4. Methodology
- 7.5. Coverage
- 7.6. Secondary Research
- 7.7. Therapeutic Landscape
- 7.8. Epidemiology-Based Forecasting
- 7.9. Market Size by Geography
- 7.10. Geographical Landscape
- 7.11. Pipeline Analysis
- 7.12. Competitive Landscape
- 7.12.1. Expert Panel Validation
- 7.13. Contact Us
- 7.14. Disclaimer
List of Tables
- Table 1: Cystic Fibrosis Market, Global, Treatment Plan
- Table 2: Cystic Fibrosis Market, Global, Pipeline, Preclinical
- Table 3: Cystic Fibrosis Market, Global, Pipeline, Phase I
- Table 4: Cystic Fibrosis Market, Global, Pipeline, Phase II
- Table 5: Cystic Fibrosis Market, Global, Pipeline, Phase III
- Table 6: Cystic Fibrosis Market, Global, Pipeline, Pre-Registration
- Table 7: Cystic Fibrosis Market, Global, Pipeline, Undisclosed
- Table 8: Cystic Fibrosis Market, Global, Market Forecast, 2012-2019
- Table 9: Cystic Fibrosis Market, US, Market Forecast, 2012-2019
- Table 10: Cystic Fibrosis Market, Canada, Market Forecast, 2012-2019
- Table 11: Cystic Fibrosis Market, UK, Market Forecast, 2012-2019
- Table 12: Cystic Fibrosis Market, France, Market Forecast, 2012-2019
- Table 13: Cystic Fibrosis Market, Germany, Market Forecast, 2012-2019
- Table 14: Cystic Fibrosis Market, Italy, Market Forecast, 2012-2019
- Table 15: Cystic Fibrosis Market, Spain, Market Forecast, 2012-2019
List of Figures
- Figure 1: Cystic Fibrosis Market, Global, Marketed, Heat Map
- Figure 2: Cystic Fibrosis Market, Global, Pipeline Summary
- Figure 3: Cystic Fibrosis Market, Global, Pipeline Mechanisms of Action
- Figure 4: Cystic Fibrosis Market, Global, Clinical Trial Failure Rate
- Figure 5: Cystic Fibrosis Market, Global, Pipeline Trial Duration
- Figure 6: Cystic Fibrosis Market, Global, Pipeline, Trial Duration by Mechanism of Action
- Figure 7: Cystic Fibrosis Market, Global, Pipeline Trial Size
- Figure 8: Cystic Fibrosis Market, Global, Pipeline, Trial Size by Mechanism of Action
- Figure 9: Cystic Fibrosis Market, Global, Pipeline, Promising Pipeline Products, Lumacaftor Forecast, 2015-2019
- Figure 10: Cystic Fibrosis Market, Global, Pipeline, Promising Pipeline Products, Ataluren Forecast, 2016-2019
- Figure 11: Cystic Fibrosis Market, Global, Pipeline Promising Pipeline Products, Aeroquin Forecast, 2014-2019
- Figure 12: Cystic Fibrosis Market, Global, Pipeline, Promising Pipeline Products, Arikace Forecast, 2015-2019
- Figure 13: Cystic Fibrosis Market, Global, Pipeline, Heat Map
- Figure 14: Cystic Fibrosis Market, Global, Treatment Patterns, 2012-2019
- Figure 15: Cystic Fibrosis Market, Global, Market Size, 2012-2019
- Figure 16: Cystic Fibrosis Market, North America, Treatment Patterns, 2012-2019
- Figure 17: Cystic Fibrosis Market, North America, Annual Cost of Treatment, 2012-2019
- Figure 18: Cystic Fibrosis Market, North America, Market Size, 2012-2019
- Figure 19: Cystic Fibrosis Market, Top Five EU Countries, Treatment Patterns, 2012-2019
- Figure 20: Cystic Fibrosis Market, Top Five EU Countries, Annual Cost of Treatment, 2012-2019
- Figure 21: Cystic Fibrosis Market, Top Five EU Countries, Market Size, 2012-2019
- Figure 22: Cystic Fibrosis Market, Global, Major Co-developments by Region and Value, 2006-2013
- Figure 23: Cystic Fibrosis Market, Global, Major Co-development Deals by Year, 2006-2013
- Figure 24: Cystic Fibrosis Market, Global, Co-development Deals by Phase, 2006-2013
- Figure 25: Cystic Fibrosis Market, Global, Licensing Deals by Region, 2006-2013
- Figure 26: Cystic Fibrosis Market, Global, Licensing Deals by Year, 2006-2013
- Figure 27: Cystic Fibrosis Market, Global, Licensing Deals by Phase, 2006-2013
- Figure 28: Forecasting Model for Therapeutic Areas