| Summary: | Orkambi is a fixed-dose combination tablet containing 200 mg lumacaftor and 125 mg ivacaftor (LUM/IVA). It is indicated for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who are homozygous for the F508del mutation in the cystic fibrosis transmembrane regulator (CFTR) gene. This is most common CF-causing mutation worldwide and approximately half of all Canadian patients with CF are homozygous for the F508del mutation. LUM/IVA is the first treatment specifically indicated for the treatment of patients who are homozygous for the F508del mutation in the CFTR gene. The manufacturer has requested that LUM/IVA be listed in accordance with the Health Canada-approved indication. The recommended dose is lumacaftor 400 mg/ivacaftor 250 mg every 12 hours. This represents the lower of the dosages considered in both the TRANSPORT AND TRAFFIC trials. At the current marketed price of $170.54 per tablet, the daily cost of treatment per patient with LUM/IVA is $682, or $248,982 annually. CADTH reviewed LUM/IVA in 2015 for the treatment of CF in patients aged 12 years and older who are homozygous for the F508del mutation in the CFTR gene. CDEC recommended that LUM/IVA not be reimbursed, based on the clinical findings. The price submitted for LUM/IVA in the original submission is the same as the current submission. The manufacturer submitted a cost-utility analysis to assess the cost-effectiveness of LUM/IVA + standard of care (SoC) compared with SoC alone in patients with CF who are 6 years of age or older and homozygous for the F508del-CFTR mutation
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