Orkambi contains the combination of lumacaftor and ivacaftor, which has the dual mechanism of correcting and potentiating the function of CFTR protein, normalizing chloride conductance in patients with cystic fibrosis. The drug was first approved in the US in July 2015 for the treatment of cystic fibrosis in patients aged 12 years and older with two copies of the F508del mutation, and corresponding EU approval was granted in November 2015. It is marketed by Vertex alongside Kalydeco, which contains the single CFTR potentiator ivacaftor.
LIST OF FIGURES
9 Figure 1: Orkambi for cystic fibrosis – SWOT analysis
10 Figure 2: Datamonitor Healthcare’s drug assessment summary of Orkambi for cystic fibrosis
11 Figure 3: Datamonitor Healthcare’s drug assessment summary of Orkambi for cystic fibrosis
13 Figure 4: Orkambi sales for cystic fibrosis across the US and five major EU markets, by country, 2016–25
LIST OF TABLES
6 Table 1: Orkambi drug profile
7 Table 2: Orkambi Phase III data in cystic fibrosis
14 Table 3: Orkambi sales for cystic fibrosis across the US and five major EU markets, by country ($m), 2016–25
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