Can Cystic Fibrosis Patients Finally Catch a Breath With Lumacaftor/Ivacaftor?
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.
Drug Delivery, Disposition and Dynamics, Monash Institute of Pharmaceutical Sciences; Monash University, Parkville, Victoria, Australia.
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Schneider
Drug Delivery, Disposition and Dynamics, Monash Institute of Pharmaceutical Sciences; Monash University, Parkville, Victoria, Australia.
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Reyes-Ortega
Drug Delivery, Disposition and Dynamics, Monash Institute of Pharmaceutical Sciences; Monash University, Parkville, Victoria, Australia.|Monash Biomedicine Discovery Institute, Department of Microbiology, Monash University, Clayton, Victoria, Australia.
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Li
Drug Delivery, Disposition and Dynamics, Monash Institute of Pharmaceutical Sciences; Monash University, Parkville, Victoria, Australia.
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Velkov
D000627:Aminophenols / Q000494:pharmacology / Q000627:therapeutic use*; D000631:Aminopyridines / Q000494:pharmacology / Q000627:therapeutic use*; D052117:Benzodioxoles / Q000494:pharmacology / Q000627:therapeutic use*; D003550:Cystic Fibrosis / Q000188:drug therapy* / Q000235:genetics / Q000503:physiopathology; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000235:genetics*; D017277:Drug Approval; D004338:Drug Combinations; D006801:Humans; D015363:Quinolones / Q000494:pharmacology / Q000627:therapeutic use*; D014481:United States; D014486:United States Food and Drug Administration
D000627:Aminophenols; D000631:Aminopyridines; D052117:Benzodioxoles; D004338:Drug Combinations; D015363:Quinolones; C000599212:lumacaftor, ivacaftor drug combination; D019005:Cystic Fibrosis Transmembrane Conductance Regulator