Can cystic fibrosis patients finally catch a breath with lumacaftor/ivacaftor?

E K Schneider, Felisa Reyes-Ortega, Jian Li, Tony Velkov

Research output: Contribution to journalReview ArticleResearchpeer-review

30 Citations (Scopus)


Cystic fibrosis (CF) is a life-limiting disease caused by defective or deficient cystic fibrosis transmembrane conductance regulator (CFTR) activity. The recent US Food and Drug Administration (FDA) approval of lumacaftor combined with ivacaftor (Orkambi) targets patients with the F508del-CFTR. The question remains: Is this breakthrough combination therapy the “magic-bullet” cure for the vast majority of patients with CF? This review covers the contemporary clinical and scientific knowledge-base for lumacaftor/ivacaftor and highlights the emerging issues from recent conflicting literature reports.

Original languageEnglish
Pages (from-to)130-141
Number of pages12
JournalClinical Pharmacology & Therapeutics
Issue number1
Publication statusPublished - 1 Jan 2017

Cite this