CFTR-based therapies completed or in progress within the Therapeutics Development Network
| CFTR mutation | Proportion of patients with causative mutation (%) | Therapeutic approach | Status |
|---|---|---|---|
| G551D/other | 4 | Ivacaftor | FDA approved, age ≥6 |
| Age 3–5 planned | |||
| Non-G551D gating/other | 1 | Ivacaftor | Phase II/III |
| R117H/other | 5 | Ivacaftor | Phase III |
| F508del/F508del | 49 | Lumacaftor+ivacaftor | Phase II; phase III planned |
| VX-661+ivacaftor | Phase II | ||
| PTC/other | 10 | Ataluren | Phase III (primary endpoint negative) |
CF, cystic fibrosis; CFTR, CF transmembrane conductance regulator; PTC, premature termination codon.