From pipeline to patient: new developments in cystic fibrosis therapeutics

Introduction: Cystic fibrosis (CF) is an inherited rare disease characterised by recurrent pulmonary infection, pancreatic malabsorption and a number of other multisystem effects. In the past few years new drugs specifically designed to treat CF have entered the pipeline, and some are now used in clinical practice. Areas covered: Clinical trial results from CF trials reported or ongoing within the last 3 years are discussed. A literature and trials registry search of trials and meta-analyses involving patients with CF was conducted, from which the most exciting developments were selected. Expert opinion: Drugs to address the basic defect in CF have finally come to market, albeit for a limited number of patients with a specific genotype. Traditional areas of CF therapeutics continue to be developed, with modest success, including drugs to modulate the airway surface liquid, new pancreatic supplements, antibiotics and new treatments for endocrine complications of CF.