7 The tezacaftor/ivacaftor combination has been evaluated in a large Phase III program enrolling cystic fibrosis patients with a wide range of genotypes, including at least one copy of the F508del mutation. Having come through these trials successfully, Vertex filed for regulatory approval in the US in August 2017, with expected first approval in February 2018 (Vertex, 2017a).
The combination of a CFTR corrector and potentiator has already been established in Vertex’s other marketed cystic fibrosis therapy, Orkambi. However, the replacement of lumacaftor with tezacaftor has been shown to be effective in both homozygous and certain heterozygous F508del mutations (Vertex, 2017b). Orkambi is currently only approved for homozygous F508del cystic fibrosis patients (Orkambi prescribing information, 2016).
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