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Novel agents in cystic fibrosis

Nature Reviews Drug Discovery volume 7, pages 555–556 (2008)Cite this article

Current medical treatments

Patients with CF receive a combination of physiotherapy, postural drainage and antibiotics, which help remove mucus from their lungs, improve their ability to breathe, and treat chronic lung infections. Therapeutic agents are also available to help clear their mucus. Nebulized hypertonic saline solution can improve the hydration of mucus and improve clearance1. Genentech's Pulmozyme (dornase alfa) is a recombinant human DNase, which hydrolyses the large amounts of DNA found in CF sputum and reduces mucus viscosity2. However, only a subset of patients with CF respond to Pulmozyme and there are no reliable markers to predict which patients will receive the benefit. There is a desperate need for improved therapies, and several promising compounds and new drug classes are currently in development (Table 1).

Table 1 Key cystic fibrosis drug development programmes
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Figure 1: Prevalence of cystic fibrosis (CF) in selected markets.
Figure 2: Forecasts for the global cystic fibrosis (CF) pharmacotherapy market.

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Author information

Authors and Affiliations

  1. Shane Storey and Graeme Wald are senior biotech analysts with Wilson HTM Investment Group, Level 38, 71 Eagle Street, Brisbane, 4000, Queensland, Australia

    Shane Storey & Graeme Wald

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  1. Shane Storey
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  2. Graeme Wald
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Corresponding author

Correspondence to Shane Storey.

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Competing interests

Graeme Wald owns shares in Pharmaxis.

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FURTHER INFORMATION

US Cystic Fibrosis Foundation

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Storey, S., Wald, G. Novel agents in cystic fibrosis. Nat Rev Drug Discov 7, 555–556 (2008). https://doi.org/10.1038/nrd2603

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