Orkambi is a medicine used to treat cystic fibrosis in patients aged 1 year and above. Cystic fibrosis is an inherited disease that has severe effects on the lungs, the digestive system (gut) and other organs. Orkambi is used in patients who have a genetic mutation (change) called the F508del mutation. This mutation affects the gene for a protein called cystic fibrosis transmembrane conductance regulator (CFTR). Orkambi is used in patients who have inherited the mutation from both parents. Orkambi contains the active substances lumacaftor and ivacaftor.
Therapeutic Indication
### Therapeutic indication Orkambi tablets are indicated for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who are homozygous for the F508del mutation in the CFTR gene. Orkambi granules are indicated for the treatment of cystic fibrosis (CF) in children aged 1 year and older who are homozygous for the F508del mutation in the CFTR gene.
Therapeutic Area (MeSH)
ATC Code
R07AX30
ATC Item
ivacaftor and lumacaftor
Pharmacotherapeutic Group
Other respiratory system products
Active Substance (Summary)
INN / Common Names
| Substance | CAS | Monograph |
|---|---|---|
| Lumacaftor | N/A | Lumacaftor |
| ivacaftor | N/A | 依伐卡托 |
EMA Name
Orkambi
Medicine Name
Orkambi
Aliases
N/A
