The US FDA has scheduled a Pulmonary-Allergy Drugs Advisory Committee (PADAC) for Tuesday, October 21, 2014 to discuss supplemental new drug application (sNDA) 203188, Kalydeco (ivacaftor) oral tablets, submitted by Vertex Pharmaceuticals Inc. (Vertex), for the treatment of cystic fibrosis in adult patients with an R117H mutation in the cystic fibrosis transmembrane conductance regulator gene.
The sNDA submission is based on data from a Phase 3 study of ivacaftor (ClinicalTrials.gov identifier NCT01614457) in patients with CF aged 6 years and older (N=69). For the overall population, the study did not meet its primary endpoint of the mean absolute change from baseline in ppFEV1 (percent predicted forced expiratory volume in one second) for ivacaftor compared to placebo. However Vertex says that a pre-specified subset analysis in patients aged 18 years and older (N=50) with a mean baseline absolute FEV1 of 65% predicted showed statistically significant improvements in ppFEV1. In particular, a statistically significant mean absolute treatment difference of 5.0 percentage points (p=0.01) in ppFEV1 was observed through 24 weeks of treatment. This corresponds to a mean relative treatment difference of 9.1 percent (p=0.008).