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Stakeholder Opinions: Cystic Fibrosis - A pipeline full of hope
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
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Disease background,
segmentation and epidemiology of cystic fibrosis in the US and
five major European countries
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Summary of the drug classes
currently used for the treatment of cystic fibrosis lung disease
and cystic fibrosis-related pancreatic insufficiency
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An overview of unmet needs, the
cystic fibrosis pipeline and clinical trial design
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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
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Understand and capitalize on
clinical unmet needs in the market through both lifecycle
management of marketed drugs and new product development
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Assess the cystic fibrosis
pipeline both through background reading and via the interactive
Excel-based pipeline summary
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Identify physician awareness
and perceptions surrounding future treatments and new
developments for cystic fibrosis
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