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TMCNet:  Research and Markets: 2013 Report on Global Cystic Fibrosis Therapeutics - Opportunity Analysis and Forecasts to 2017

[January 22, 2014]

Research and Markets: 2013 Report on Global Cystic Fibrosis Therapeutics - Opportunity Analysis and Forecasts to 2017

DUBLIN --(Business Wire)--

Research and Markets ( has announced the addition of the "2013 Report on Global Cystic Fibrosis Therapeutics - Opportunity Analysis and Forecasts to 2017" report to their offering.

Cystic Fibrosis is caused by a genetic mutation which leads to the production of thickened sticky secretions in organs with epithelial cell linings, including the respiratory tract. Over time, lung infections lead to airway destruction, respiratory failure and death. The first-in-class CFTR modulator Kalydeco, a CFTR potentiator works to restore the function of the mutated CFTR protein in patients with the G551D gating mutation of CF, allowing an improved flow f salt and fluids on the surface of the lungs.

Only around 4% of CF patients in the US have the G551D gating mutation, and more studies are needed to help determine whether people with other CF gating mutations might also be eligible for Kalydeco. Still, the drug's approval has paved the way for a new class of therapies, which offer a personalized treatment approach to patients and could be highly profitable for its developers.

Vertex (News - Alert) is now developing a combination therapy of potential first-in-class CFTR modulator lumacaftor (VX-809) with Kalydeco (VX-770) for the treatment of CF patients who are homozygous for the F508del mutation in the CFTR gene. The CFTR corrector, lumacaftor increases trafficking of the defective CFTR protein to the cell membrane, where it can exert its functions. The combination therapy is currently in Phase III stage of development, and is expected to gain approval in 2014.

It is estimated that around half of all CF patients are homozygous to the F508del mutation, and Vertex has also announced plans to explore the lumacaftor/Kalydeco combination therapy in patients that are heterozygous to the F508del mutation.

Key Topics Covered:

1 List of Tables and Figures

2 Introduction

3 Disease Overview

4 Epidemiology

5 Current Treatment Options

6 Unmet Needs Assessment and Opportunity Analysis

7 R&D Strategies

8 Pipeline Assessment

9 Pipeline Valuation Analysis

10 Appendix

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