Vertex’s Orkambi Efficacy Data May Make FDA More Cautious With Other ‘Breakthroughs’
Executive Summary
Agency’s trial design recommendations made it difficult to assess lumacaftor’s contribution to the lumacaftor/ivacaftor fixed-dose combination’s efficacy in cystic fibrosis. The experience may color FDA’s future approach to combination product trial designs and how it advises other ‘breakthrough therapy’ sponsors on the evidence likely needed to support approval.
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