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Vertex’s Latest Bid For Kalydeco Label Expansion Heads To FDA Panel

This article was originally published in The Pink Sheet Daily

Executive Summary

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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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.

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