October 19, 2011
NDA Submitted for Kalydeco for the Treatment of Cystic FibrosisVertex announced that it has submitted a New Drug Application (NDA) for priority review for Kalydeco (VX-770, ivacaftor), a drug being evaluated to target the defective protein that causes cystic fibrosis (CF). This submission was based on two Phase 3 studies, STRIVE and ENVISION, that evaluated Kalydeco in patients ≥6 years of age with CF who have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
Kalydeco is an oral tablet that directly targets the defective CFTR protein known to cause CF. Kalydeco increases the function of defective CFTR protein by increasing the gating activity, or ability to transport chloride ions across the cell membrane, of CFTR once it reaches the cell surface.
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