Vertex’s Combo Drug Orkambi Faces FDA Doubts About Lumacaftor’s Benefit
This article was originally published in The Pink Sheet Daily
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.
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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.
Lengthy withdrawal process creates an opening for the FDA to better convey its concerns about a medicine’s benefit-risk profile in a way that impacts prescribing guidelines, Johns Hopkins’ Joshua Sharfstein says; CDER’s Jacqueline Corrigan-Curay says new expedited withdrawal procedures under omnibus law appear more ‘streamlined’ than those followed for Makena.