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From pipeline to patient: new developments in cystic fibrosis therapeutics

Andrew P Prayle1, Alan R Smyth

  • 1University of Nottingham, School of Clinical Science, Queens Medical Centre, Child Health, Nottingham, UK.

Expert Opinion on Pharmacotherapy
|February 5, 2013
PubMed
Abstract

Insights

New cystic fibrosis (CF) treatments targeting the basic defect are now available for specific genotypes. Ongoing research also shows modest success in modulating airway surface liquid and managing CF complications.

Area of Science:

  • Pulmonology
  • Genetics
  • Pharmacology

Background:

  • Cystic Fibrosis (CF) is a rare inherited disorder causing recurrent lung infections and pancreatic malabsorption.
  • Recent advancements have introduced targeted therapies for CF, with some now in clinical use.

Purpose of the Study:

  • To review recent clinical trial outcomes in Cystic Fibrosis (CF).
  • To highlight significant developments in CF therapeutics from the past three years.

Main Methods:

  • Conducted a literature and trials registry search for CF trials and meta-analyses.
  • Selected key findings from ongoing and recently reported CF clinical trials.

Main Results:

  • Targeted therapies addressing the fundamental defect in CF have been approved for specific genotypes.
  • Modulators of airway surface liquid, new pancreatic enzyme supplements, antibiotics, and treatments for endocrine complications show modest progress.

Conclusions:

  • The advent of genotype-specific therapies marks a significant milestone in CF treatment.
  • Continued development in traditional therapeutic areas offers ongoing, albeit incremental, benefits for CF patients.

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