Table 2

CFTR-based therapies completed or in progress within the Therapeutics Development Network

CFTR mutationProportion of patients with causative mutation (%)Therapeutic approachStatus
G551D/other4IvacaftorFDA approved, age ≥6
Age 3–5 planned
Non-G551D gating/other1IvacaftorPhase II/III
R117H/other5IvacaftorPhase III
F508del/F508del49Lumacaftor+ivacaftorPhase II; phase III planned
VX-661+ivacaftorPhase II
PTC/other10AtalurenPhase III (primary endpoint negative)
  • CF, cystic fibrosis; CFTR, CF transmembrane conductance regulator; PTC, premature termination codon.