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FDA Panel To Weigh Adequacy Of Efficacy Evidence For Kalydeco Label Expansion

This article was originally published in The Pink Sheet Daily

Executive Summary

Vertex is seeking approval for cystic fibrosis patients ages six years and older with the R117H mutation despite the pivotal trial’s primary endpoint failure and unfavorable pediatric subgroup results. Company believes totality of evidence supports approval and pediatric efficacy can be extrapolated from positive adult subgroup findings.

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