Skip to main content

Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.

  • From the Analyst's Couch
  • Published:

The cystic fibrosis drug market

Nature Reviews Drug Discovery volume 13, pages 721–722 (2014)Cite this article

Subjects

The disease is caused by mutations in the gene encoding the CF transmembrane conductance regulator (CFTR), an anion channel that helps maintain fluid- and electrolyte-homeostasis in multiple organs, including the lungs and pancreas. In individuals with CF this homeostasis is disrupted, leading to the accumulation of hyperviscous mucus. In the lung, this blocks the airways, resulting in a cycle of inflammation and chronic infection, particularly with the bacterium Pseudomonas aeruginosa. In the pancreas, hyperviscous mucus obstructs the movement of digestive enzymes.

To date, more than 1,700 different CFTR mutations have been described. The most common mutation, found in almost 70% of individuals with CF, is a deletion of three nucleotides encoding phenylalanine at position 508 (ΔPhe508), resulting in the expression of a defective CFTR protein.

This is a preview of subscription content, access via your institution

Access options

Buy this article

  • Purchase on Springer Link
  • Instant access to full article PDF
Buy now

Prices may be subject to local taxes which are calculated during checkout

Figure 1: Global sales of cystic fibrosis drugs.

Author information

Authors and Affiliations

  1. IMS Health Ltd, 210 Pentonville Road, London, N1 9JY, UK

    Basharut A. Syed & Bashar Hamad

Authors
  1. Basharut A. Syed
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. Bashar Hamad
    View author publications

    You can also search for this author in PubMed Google Scholar

Corresponding authors

Correspondence to Basharut A. Syed or Bashar Hamad.

Ethics declarations

Competing interests

The authors declare no competing financial interests.

Related links

Further information

IMS Health

PowerPoint slides

Rights and permissions

Reprints and permissions

About this article

Check for updates. Verify currency and authenticity via CrossMark

Cite this article

Syed, B., Hamad, B. The cystic fibrosis drug market. Nat Rev Drug Discov 13, 721–722 (2014). https://doi.org/10.1038/nrd4434

Download citation

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1038/nrd4434

This article is cited by

Search

Advanced search

Quick links

Nature Briefing: Translational Research

Sign up for the Nature Briefing: Translational Research newsletter — top stories in biotechnology, drug discovery and pharma.

Get what matters in translational research, free to your inbox weekly. Sign up for Nature Briefing: Translational Research