Introduction

With cystic fibrosis patients still dying prematurely and only two products currently approved for the treatment of this disease, the market is wide open for new entrants. Not only will novel symptomatic treatments and re-formulations lead to improved treatment paradigms, truly disease-modifying compounds are in late-stage development and will significantly change the market.

Scope

• Disease background, segmentation and epidemiology of cystic fibrosis in the US and five major European countries
• Summary of the drug classes currently used for the treatment of cystic fibrosis lung disease and cystic fibrosis-related pancreatic insufficiency
• An overview of unmet needs, the cystic fibrosis pipeline and clinical trial design
• In-depth outline of around 85 compounds studied for the treatment of cystic fibrosis by the pharmaceutical industry and academic institutes

Highlights

It is clear that, although cystic fibrosis only affects around 52,000 patients in the five major European countries and the US, the pipeline is highly active with over 50 pharmaceutical companies involved. As many as 13 of the compounds studied by the pharmaceutical industry are currently in late-stage development.

The promise of gene therapy has yet to materialize, and mutation specific correcting therapy is eagerly anticipated. There are at least four protein repair treatments in clinical development for three different classes of mutations, although it is likely that combination strategies are required in order to enhance their efficacy.

Disease-modifying treatments that are not mutation-specific include alternative chloride channel activators and epithelial sodium channel inhibitors. Compounds in the former class are furthest along in development; however, it is likely that a combination of the two classes will be necessary for full correction of the airway surface liquid.

Reasons to Purchase

• Understand and capitalize on clinical unmet needs in the market through both lifecycle management of marketed drugs and new product development
• Assess the cystic fibrosis pipeline both through background reading and via the interactive Excel-based pipeline summary
• Identify physician awareness and perceptions surrounding future treatments and new developments for cystic fibrosis

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