Answer
It is licensed for people with cystic fibrosis who have F508del on both copies of the CFTR gene. The licensed formulation depends on age and, for younger children, weight.
Sources for this answer
- Orkambi 150 mg/188 mg granules in sachetemc · SmPC Current selected SmPC · Version 10
- Orkambi 200 mg/125 mg film coated tabletsemc · SmPC Current selected SmPC · Version 19