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

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.