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Kalydeco Reviewers

This article was originally published in Pharmaceutical Approvals Monthly

Executive Summary

FDA staff that participated in the review of Vertex’s cystic fibrosis drug ivacaftor.

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Kalydeco Clinical Development

A timeline chronicling the development history of Vertex’s 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.

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