Table 4.
Summary of KalydecoTM and OrkambiTM approval for CF patients’ treatment.
| Pharmaceutical treatment | CFTR mutations | Jurisdiction approved | Age group licensed |
|---|---|---|---|
| KalydecoTM | G551D∗ | United States, Europe and Canada | >2 years |
| Australia and New Zealand | >6 years | ||
| G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D∗ | United States, Europe and Canada | >2 years | |
| Australia | >6 years | ||
| R117H∗ | United States | >2 years | |
| Europe and Canada | >18 years | ||
| OrkambiTM | ΔF508-homozygous | United States, Europe and Canada | >12 years |
∗Mutation in at least one allele.