Vertex announced that the Food and Drug Administration’s (FDA) Pulmonary-Allergy Drugs Advisory Committee (PADAC) has voted 12 to 1 to recommend that the FDA approve Orkambi (lumacaftor/ivacaftor) for people with cystic fibrosis (CF) aged ≥12 who have two copies of 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. If approved, Orkambi would be the first and only drug indicated for the treatment of CF in patients aged ≥12 who have two copies of the F508del mutation in the CFTR gene.
Orkambi was granted Breakthrough Therapy designation in 2012 and a New Drug Application (NDA) was submitted to the FDA in November 2014. The FDA is expected to make a decision regarding the drug approval by July 5, 2015.
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