Clinical review report: Lumacaftor/Ivacaftor (Orkambi) (Vertex Pharmaceuticals (Canada) Incorporated)
Orkambi is a fixed-dose combination tablet containing lumacaftor and ivacaftor (LUM/IVA). It is indicated for the treatment of cystic fibrosis (CF) in patients aged six years and older who are homozygous for the F508del mutation in the cystic fibrosis transmembrane regulator (CFTR) gene. This is the...
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| Format: | eBook |
| Language: | English |
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Ottawa (ON)
Canadian Agency for Drugs and Technologies in Health
2018, October 2018
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| Edition: | Version: Final (with redactions) |
| Series: | CADTH common drug review
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| Online Access: | |
| Collection: | National Center for Biotechnology Information - Collection details see MPG.ReNa |
| Summary: | Orkambi is a fixed-dose combination tablet containing lumacaftor and ivacaftor (LUM/IVA). It is indicated for the treatment of cystic fibrosis (CF) in patients aged six years and older who are homozygous for the F508del mutation in the cystic fibrosis transmembrane regulator (CFTR) gene. This is the 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 receive a recommendation to reimburse in accordance with the Health Canada-approved indication. CADTH previously reviewed LUM/IVA for treatment of patients aged 12 years and older. The indication was subsequently expanded to include patients who are least six years of age. The current CADTH Common Drug Review submission is for the full Health Canada-approved indication |
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| Physical Description: | 1 PDF file (184 pages) illustrations |