Return Of Vertex’s Ivacaftor To FDA Panel May Signal Efficacy Concerns
This article was originally published in The Pink Sheet Daily
Kalydeco raced to market for cystic fibrosis, but effort to get ‘breakthrough’ approval with lumacaftor for much larger patient population may have FDA worrying about the meaningfulness of pivotal efficacy results.
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In addition to a spate of FDA approvals closing out the year, Roche contended with trial failures and Actavis received a “complete response” letter for its nebivolol/valsartan combination.
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.
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.