Vertex’s Latest Bid For Kalydeco Label Expansion Heads To FDA Panel
This article was originally published in The Pink Sheet Daily
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.
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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.
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.
Kalydeco, an oral CFTR potentiator, becomes the first approved drug to address the underlying cause of cystic fibrosis, rather than just treating the disease’s symptoms.