Vertex Phase III For CF Combo Shows Power Of “Breakthrough” Designation
This article was originally published in The Pink Sheet Daily
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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Agency’s trial design recommendations made it difficult to assess lumacaftor’s contribution to the lumacaftor/ivacaftor fixed-dose combination’s efficacy in cystic fibrosis. The experience may color FDA’s future approach to combination product trial designs and how it advises other ‘breakthrough therapy’ sponsors on the evidence likely needed to support approval.
Advisory committee to consider whether Vertex should conduct new clinical trial comparing ‘breakthrough therapy’-designated, fixed-dose combo of lumacaftor and ivacaftor to ivacaftor alone in subgroup of cystic fibrosis patients.
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.