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Kalydeco Passed FDA’s Approval Test With The Help Of A Failed Study

This article was originally published in Pharmaceutical Approvals Monthly

Executive Summary

FDA’s speedy approval of Vertex’s disease-modifying, genetically targeted cystic fibrosis treatment ivacaftor appears to have been made easier by the company’s failure to show efficacy in a population with a different genetic mutation. The failed study not only had important implications for preventing off-label use, it added considerably to the safety database in an orphan indication.

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