Table 1.
Approved CFTR modulators and their indications.
| Modulators | Commercial Name | Approval Year | Responsive Mutations | Approved Age |
|---|---|---|---|---|
| Ivacaftor | Kalydeco® (EU/USA) | 2012 | G551D, S549N, G1244E, G178R, S1251N, G551S, G1349D, S1255P, R117H, E56K, K1060T, P67L, E193K, A1067T, R74W, L206W, G1069R, D110E, R347H, D579G, R1070Q, D1270N, D110H, R352Q, S945L, R1070W, R117C, A455E, S977F, F1074L, F1052V, D115H; 3849 + 10 kb C > T, 2789 + 5G > A, 327326A > G, 711 + 3A > G, E831X | ≥4 months |
| Lumacaftor-ivacaftor | Orkambi® (EU/USA) | 2015 | Two copy of F508del | ≥2 years |
| Tezacaftor-ivacaftor | Symkevi® (EU) Symdeko® (USA) |
2018 | Two copy of F508del One copy of F508del in association with E56K, K1060T, P67L, E193K, A1067T, R74W, L206W, D110E, D110H, R347H, D579G, R1070Q, D1270N, R352Q, S945L, R1070W, R117C, A455E, S977F, F1074L, F1052V, D1152H, 3849 + 10 kb C > T, 2789 + 5G > A, 327326A > G, 711 + 3A > G |
≥6 years |
| Elexacaftor-tezacaftor-ivacaftor | Kaftrio® (EU) Trikafta® (USA) |
2020 (EU) 2019 (USA) |
One copy of F508del | ≥6 years |