Kalydeco Clinical Development
This article was originally published in Pharmaceutical Approvals Monthly
A timeline chronicling the development history of Vertex’s ivacaftor.
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FDA staff that participated in the review of Vertex’s cystic fibrosis drug ivacaftor.
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.
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.