Secondary Endpoints Backed Kalydeco’s Efficacy, Despite Lack Of Correlation With Pulmonary Function
This article was originally published in Pharmaceutical Approvals Monthly
Executive Summary
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.
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Regulatory Round-Up: Drug Review Profiles From 2012
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