Vertex has announced that the Food and Drug Administration (FDA) has accepted for filing and granted Priority Review to their supplemental New Drug Application (sNDA) for Orkambi (lumacaftor/ivacaftor). The sNDA proposes the use of Orkambi in patients with cystic fibrosis (CF) between the ages of 6 and 11 who have two copies of F508del mutation.

Patients with two copies of F508del mutation have little to no cystic fibrosis transmembrane conductance regulator (CFTR) protein at the cell surface. Orkambi is a combination of lumacaftor, which is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del CFTR protein, and ivacaftor, which is designed to enhance 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 in 2015 it was approved to treat CF patients aged ≥12, who are homozygous for the F508del gene mutation.

Data from a recent open label Phase 3 clinical study that examined the safety of Orkambi is included in the sNDA. The FDA have set a target review date for the application of September 30, 2016, under the Prescription Drug User Free Act (PDUFA).  

Vertex is also currently running a 6-month efficacy trial of Orkambi in Europe, with participants aged 6 to 11. The results will be used to support a submission for approval in Europe in early 2017.

Results of the U.S. Phase 3 clinical study cited in the sNDA will be presented next week at the European Cystic Fibrosis Society conference

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