Potentiators and Correctors in Paediatric Cystic Fibrosis Patients: A Narrative Review
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Cystic fibrosis is the most common inherited condition in the Caucasian population and is associated with significantly reduced life expectancy. Recent advances in treatment have focussed on addressing the underlying cause of the condition, the defective production, expression and function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. Several drugs with different modes of action have produced promising results in clinical trials, and some have been incorporated into routine clinical care for specific patients in many countries worldwide. Further trials continue to explore the safety and efficacy of these drugs in the youngest age groups and to search for more effective therapies to treat the most common disease-causing gene mutations in an ever-expanding drug pipeline. As evidence mounts for the early onset of disease in young patients, the prospect of introducing disease-modifying therapy in early life becomes more pertinent, although the cost implications of these expensive drugs are significant. In this review, we summarise these new therapy advances and review those currently being explored in clinical trials.
Compliance with Ethical Standards
Conflict of interest
RD, CE, DH, and IM have no conflicts of interest and have received no funding in relation to this manuscript. JCD has performed clinical trial leadership, advisory board and educational roles for the following companies for which fees have been paid to Imperial College London or Royal Brompton Hospital: Vertex Pharmaceuticals, PTC, Proteostasis, Galapagos, Abbvie, Chiesi, Eloxx, Ionis, Enterprise Therapeutics, Pulmocide, Novartis, Algipharma. JCD has received no funding in relation to this manuscript.
No sources of funding were used to conduct this study or prepare this manuscript.
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