The Food and Drug Administration (FDA) has approved Orkambi (lumacaftor/ivacaftor; Vertex), the first and only drug indicated for the treatment of cystic fibrosis (CF) in patients aged ≥12 who are homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Orkambi contains lumacaftor, which increases the amount of functional protein at the cell surface by addressing the processing and trafficking defect of the protein, and ivacaftor, which enhances the function of the CFTR protein once it reaches the cell surface.

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Orkambi was granted Breakthrough Therapy designation in 2012 and its approval was recommended by the FDA’s Pulmonary-Allergy Drugs Advisory Committee (PADAC).

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