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news.Vertex Pharmaceuticals has submitted a new drug application (NDA) for priority review to the US Food and Drug Administration (FDA) for approval of Kalydeco to treat cystic fibrosis (CF).
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Kalydeco was studied among people with CF ages 6 and older who have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
The NDA includes Phase 3 pivotal studies results which showed sustained improvements in lung function and other measures of disease in people with CF.
The company said if the NDA is granted, the FDA’s review time would get shortened from 10 to 6 months.
Vertex also plans to submit a marketing authorization application (MAA) for Kalydeco with the European Medicines Agency (EMA) and if granted will shorten the review time from 210 days to 150 days following the start of the review.
Vertex has also submitted requests to the FDA and EMA to use the trade name KALYDECO (ivacaftor) for VX-770.
Vertex Global Research and Development CSO and executive vice president Peter Mueller said they look forward to working with US and European regulatory agencies to make Kalydeco available as quickly as possible.