FDA’s First Breakthrough Designations Continue Vertex Kalydeco’s Regulatory Successes
This article was originally published in The Pink Sheet Daily
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.
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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.