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Cystic Fibrosis Therapeutics in Major Developed Markets to 2019 - CFTR Modulators Initiate Drive towards Personalized Treatment and Market Growth
[February 14, 2014]

Cystic Fibrosis Therapeutics in Major Developed Markets to 2019 - CFTR Modulators Initiate Drive towards Personalized Treatment and Market Growth

(M2 PressWIRE Via Acquire Media NewsEdge) Dublin - Research and Markets ( has announced the addition of the "Cystic Fibrosis Therapeutics in Major Developed Markets to 2019 - CFTR Modulators Initiate Drive towards Personalized Treatment and Market Growth" report to their offering.

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.

Key Topics Covered 1 Table of Contents 2 Introduction 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 3.3 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 4.4 Promising Pipeline Molecules 4.5 Heat Map of Pipeline Products 4.6 Conclusion 5 Market Forecast to 2019 5.1 Geographical Markets 5.2 Drivers and Barriers 6 Strategic Consolidations 6.1 Major Co-Development Deals 6.2 Major Licensing Deals 7 Appendix Companies Mentioned - Aptalis Pharma - Chiesi Farmaceutici - Forest Laboratories - Genentech - Gilead Sciences - Insmed - Novartis - PTC Therapeutics - Pharmaxis - Vertex Pharmaceuticals For more information visit About Research and Markets Research and Markets is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products.

CONTACT: Research and Markets, Laura Wood, Senior Manager. Fax from USA: 646-607-1907 Fax from rest of the world: +353-1-481-1716 Sector: Healthcare and Medical Devices (, Pharmaceuticals ( .


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