Vertex announced that it has submitted a New Drug Application (NDA) to the Food and Drug Administration (FDA) for the combination product, lumacaftor and ivacaftor for people with cystic fibrosis (CF) ages ≥12 who have two copies of the F508del mutation in the cystic fibrosis transmembrance conductance regulator (CFTR) gene.
The NDA submission is based on previously announced data from two global Phase 3 studies, TRAFFIC and TRANSPORT, and the first interim data from the subsequent rollover study in people ages ≥12 who have two copies of the F508del mutation treated with standard-of-care medicines. The TRAFFIC and TRANSPORT studies showed improvements in lung function and other measures of disease, such as pulmonary exacerbations, through 24 weeks of treatment with lumacaftor in combination with ivacaftor. Initial interim data from the rollover study showed that lung function improvements were sustained for 48 total weeks of treatment (24 weeks in TRAFFIC/TRANSPORT + 24 weeks in rollover study).
In 2012, the combination of lumacaftor and ivacaftor was granted Breakthrough Therapy designation by the FDA.
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