Kalydeco Clinical Development
This article was originally published in Pharmaceutical Approvals Monthly
Executive Summary
A timeline chronicling the development history of Vertex’s ivacaftor.
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Kalydeco Reviewers
FDA staff that participated in the review of Vertex’s cystic fibrosis drug ivacaftor.
Secondary Endpoints Backed Kalydeco’s Efficacy, Despite Lack Of Correlation With Pulmonary Function
Although a decrease in sweat chloride levels was suggestive of benefit, this did not correlate with improvements in FEV1 function, FDA said. The agency also noted that the patient-reported outcomes instrument used in the pivotal clinical trials, though not fully validated, measured respiratory symptoms that are clinically relevant to patients.
FDA Liked What It Saw In Kalydeco’s Development Program
Agency reviewers looked favorably upon the pivotal trials’ primary efficacy endpoint and low drop-out rate, as well as the overall quality of the NDA submission.