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...
Saved in:
| Online Access: |
Full text |
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| Format: | Electronic eBook |
| Language: | English |
| Published: |
Ottawa (ON) :
Canadian Agency for Drugs and Technologies in Health,
2018
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| Edition: | Version: Final (with redactions). |
| Series: | Common drug review clinical review report.
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| Subjects: |
| 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 online resource (1 PDF file (184 pages)) : illustrations. |
| Bibliography: | Includes bibliographical references. |
| Source of Description, Etc. Note: | Description based on online resource; title from PDF title page (viewed June 19, 2019). |
| Funding Information Note: | Funding: CADTH receives funding from Canada's federal, provincial, and territorial governments, with the exception of Quebec. |
