Cystic Fibrosis (CF) is an autosomal recessive disease affecting up to 90,000 
people worldwide. Approximately 73% of patients are homozygous for the F508del 
cystic fibrosis transmembrane conductance regulator [CFTR] mutation. 
Traditionally treatment has only included supportive care. Therefore, there is a 
need for safe and effective novel therapies targeting the underlying molecular 
defects seen with CF. Areas covered: In 2016, the Food and Drug Administration 
and the European Commission approved LUM/IVA (Orkambi), a CFTR modulator that 
includes both a CFTR corrector and potentiator, for CF patients homozygous for 
the F508del CFTR mutation. This article reviews the pharmacologic features, 
clinical efficacy, and safety of LUM/IVA and summarize the available 
pre-clinical and clinical data of LUM/IVA use. Expert commentary: LUM/IVA showed 
modest, but significant improvements from baseline in percent predicted FEV1 
(ppFEV1) as well as a reduction in pulmonary exacerbations by 35% It was shown 
to be safe for short- and long-term use. Currently, LUM/IVA is the only oral 
agent in its class available and represents a milestone the development of 
therapies for the management of CF. Nonetheless, pharmacoeconomic data are 
necessary to justify its high cost before is use becomes standard of care.