Abstract
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 of this research
- 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
Research and analysis 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.
Key reasons to purchase this research
- 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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