Medical uses
Ivacaftor is used for the treatment of cystic fibrosis in people having one of several specific mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) protein: E56K, G178R, S549R, K1060T, G1244E, P67L, E193K, G551D, A1067T, S1251N, R74W, L206W, G551S, G1069R, S1255P, D110E, R347H, D579G, R1070Q, D1270N, D110H, R352Q, S945L, R1070W, G1349D, R117C, A455E, S977F, F1074L, R117H, S549N, F1052V, D1152H.Ivacaftor is also included in a combination product, lumacaftor/ivacaftor, in a single pill, which is used to treat people with cystic fibrosis who have the F508del mutation in CFTR.Ivacaftor is also included in the combination product tezacaftor/ivacaftor with ivacaftor sold as Symdeko and as Symkevi.Symdeko is indicated to treat people aged six and older who have two copies of the F508del mutation in CFTR.Symkevi is indicated in a combination regimen with ivacaftor 150 mg tablets for the treatment of people with cystic fibrosis (CF) aged twelve years and older who are homozygous for the F508del mutation or who are heterozygous for the F508del mutation and have one of the following mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272 26A→G, and 3849+10kbC→T.Ivacaftor is available in a combination product with elexacaftor and tezacaftor called Trikafta for the treatment of people with cystic fibrosis who have the F508del mutation or other mutations.
UK agencies estimated the cost per quality adjusted life year (QALY) at between £335,000 and £1,274,000 —well above the National Institute for Health and Care Excellence thresholds.The medication was not covered under the Ontario Drug Benefit plan until June 2014, when the Government of Ontario and the manufacturer negotiated for what "Ontario Health Minister Deb Matthews had called a "fair price" for taxpayers". The negotiations took 16 months and it was estimated that around 20 Ontarians required the medication at the time.The province of Alberta began covering the medication in July 2014, and in September the province of Saskatchewan became the third province to include it in its provincial medication plan.Delay in agreement on a price for Vertex to charge national health plans led to patient group protests in Wales, England, and Australia.As of March 2016, the combination medication cost $259,000 a year in the United States. Research
The clinical trials used in the regulatory approval of ivacaftor are described here. G551D mutation
Of the approximately 70,000 cases of cystic fibrosis worldwide, 4% (~3,000) are due to a mutation called G551D. The safety and efficacy of ivacaftor for the treatment of cystic fibrosis in patients with this mutation was examined in two clinical trials.The first trial was performed in adults having baseline respiratory function (FEV1) between 32% and 98% of normal for persons of similar age, height, and weight. The baseline average was 64%. Improvement in FEV1 was rapid and sustained.