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Improving complex medical care while awaiting next‐generation CFTR potentiators and correctors: The current pipeline of therapeutics
Author(s) -
Goralski Jennifer L.,
Davis Stephanie D.
Publication year - 2015
Publication title -
pediatric pulmonology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.866
H-Index - 106
eISSN - 1099-0496
pISSN - 8755-6863
DOI - 10.1002/ppul.23253
Subject(s) - potentiator , ivacaftor , medicine , cystic fibrosis , cystic fibrosis transmembrane conductance regulator , intensive care medicine , population , pharmacology , environmental health
Summary While a major target in cystic fibrosis (CF) research in recent years has been the development of corrector and potentiator drugs targeting the cystic fibrosis transmembrane conductance regulator (CFTR) protein, these therapies have not yet proven robust enough to replace or eliminate other therapies that have demonstrated improved health outcomes and quality of life in patients with CF. Further, ivacaftor is only indicated for approximately 5% of the US CF population, although the FDA has recently approved lumacaftor/ivacaftor, a combination therapy intended for those homozygous for Phe508del, which should reach a much larger number of patients. This review appraises therapeutics currently available or being studied while we await the next generation of CFTR potentiators and correctors. Pediatr Pulmonol. 2015; 50:S66–S73. © 2015 Wiley Periodicals, Inc.