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Ivacaftor improves lung function in CF patients

  • In a 24-patient two-part proof-of-concept study, Vertex Pharmaceuticals' (VRTX) Kalydeco (ivacaftor) demonstrated a statistically significant improvement in mean absolute lung function (percent predicted forced expiratory volume in one second (PPFEV1) after two weeks of ivacaftor treatments versus placebo (part 1) and improvements in lung function after eight weeks of treatment (part 2).
  • The proof-of-concept study was the first to evaluate the use of ivacaftor in multiple residual mutations and is supported by in vitro observations that showed ivacaftor enhanced the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein in cells with residual function mutations.
  • Based on these data, Vertex plans to initiate a larger Phase 3 trial that will evaluate longer-term ivacaftor treatment.
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