Journal article

Can Cystic Fibrosis Patients Finally Catch a Breath With Lumacaftor/Ivacaftor?

EK Schneider, F Reyes-Ortega, J Li, T Velkov

CLINICAL PHARMACOLOGY & THERAPEUTICS | WILEY | Published : 2017

Abstract

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.

Grants

Awarded by National Institute of Allergy and Infectious Diseases (NIAID) of the National Institutes of Health


Awarded by NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES


Funding Acknowledgements

This study is supported by a research grant to J.L and T.V. from the National Institute of Allergy and Infectious Diseases (NIAID) of the National Institutes of Health (R01AI111965). The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institute of Allergy and Infectious Diseases or the National Institutes of Health. J.L. is an Australian National Health and Medical Research Council (NHMRC) Senior Research Fellow, and T.V. is an Australian NHMRC Industry Career Development Level 2 Research Fellow.