Vertex’s Symdeko ([tezacaftor + ivacaftor]) is a cystic fibrosis transmembrane conductance regulator (CFTR) corrector compound used as an adjunct to Vertex’s other marketed and developmental CFTR-targeting products. The drug’s initial development is as part of a fixed-dose combination with the CFTR potentiator ivacaftor (Vertex), which was approved with the brand name Kalydeco in January 2012. 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 and achieved approval in February 2018.
The combination of a CFTR corrector and potentiator has already been established in Vertex’s other marketed cystic fibrosis therapy, Orkambi ([lumacaftor + ivacaftor]). However, the replacement of lumacaftor with tezacaftor has been shown to be effective in both homozygous and certain heterozygous F508del mutations. Orkambi is currently only approved for homozygous F508del cystic fibrosis patients.
LIST OF FIGURES 9 Figure 1: Symdeko for cystic fibrosis – SWOT analysis
10 Figure 2: Datamonitor Healthcare’s drug assessment summary of Symdeko for cystic fibrosis
11 Figure 3: Datamonitor Healthcare’s drug assessment summary of Symdeko for cystic fibrosis
13 Figure 4: Symdeko sales for cystic fibrosis across the US and five major EU markets, by country, 2016–25
LIST OF TABLES
6 Table 1: Symdeko drug profile
7 Table 2: Symdeko Phase III data in cystic fibrosis
14 Table 3: Symdeko sales for cystic fibrosis across the US and five major EU markets, by country ($m), 2016–25
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