Table 1

CFTR modulators licenced for use by the European Medicines Agency (EMA)

Modulator	Year of EMA licensing	Age group	Genetics
Ivacaftor	2012	≥6 years	Heterozygous G551D
	2014	≥6 years	Heterozygous for additional gating mutations^#
	2015	≥2 years ≥18 years	As above Heterozygous R117H
	2018	≥12 months	Heterozygous for gating mutations^#
	2019	≥6 months	As above
	2020	≥4 months	Heterozygous R117H Heterozygous for gating mutations^#
Lumacaftor/ivacaftor	2015	≥12 years	Homozygous Phe508del
	2017	≥6 years	As above
	2018	≥2 years	As above
Tezacaftor/ivacaftor	2018	≥12 years	Homozygous Phe508del or Phe508del+residual function mutation^¶
	2020	≥6 years	As above
Elexacaftor/tezacaftor/ivacaftor	2020	≥12 years	Homozygous Phe508del or Phe508del+minimal function mutation
	2021	≥12 years	Heterozygous Phe508del

^#: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1225P, S549N, S549R. ^¶: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A>G, S945L, S977F, R1070W, D1152H, 2789+5G>A, 3272-26A>G, 3849+10kbC>T.