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Vertex Phase III For CF Combo Shows Power Of “Breakthrough” Designation

This article was originally published in Pharmaceutical Approvals Monthly

Executive Summary

Pivotal trials for the combination of Kalydeco and VX-809 will measure safety and efficacy over 24 weeks, whereas Kalydeco monotherapy gained FDA approval on the basis of 56 weeks of data; Vertex and FDA developed the protocol together following a recent “breakthrough therapy” designation for the two-drug combo.

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