Innovative Drug Development: Kalydeco Provides A Paradigm, FDA’s Spielberg Says
The deputy commissioner detailed the steps involved in getting Vertex’s cystic fibrosis drug rapidly through the approval process and noted that FDA is working on innovative study designs and assessing how to better use diagnostics.
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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.
Supplemental NDA for use in adult cystic fibrosis patients with R117H mutation is based on a pre-specified subgroup analysis of 50 patients from a 69-patient pivotal trial that failed its primary efficacy endpoint.
Label expansion efforts for Vertex’s cystic fibrosis therapy will be claims taken through FDA’s new review pathway; the initial product could reach more patients more quickly and serve as a guide to how the agency will handle drugs earmarked for streamlined development and approval.