. 2021 Apr 23;10(5):486. doi: 10.3390/antibiotics10050486

Table 6.

Comparative CFTR modulators.

CFTR Modulator	Country	Mutations	Age	Adverse Events	Expansion Label Studies
Ivacaftor Kalydeco^®	FDA	711 + 3A→G, F311del, I148T, R75Q, S589N, 2789 + 5G→A, F311L, I175V, R117C, S737F, 3272-26A→G, F508C, I807M, R117G, S945L, 3849 + 10kbC→T, F508C, I1027T, S977F, A120T, F1052V, I1139V, R117L, S1159F, A234D, F1074L, K1060T, R117P, S1159P, A349V, G178E, L206W, R170H, A455E, L320V, R347H, S1255P, A1067T, G194R, L967S, R347L, T338I, D110E, G314E, L997F, R352Q, T1053I, D110H, L1480P, R553Q, V232D, D192G, M152V, R668C, V562I, D579G, G576A, M952I, R792G, V754M, D924N, G970D, M952T, R933G, V1293G, D1152H, G1069R, P67L, R1070Q, W1282, D1270N, Q237E, R1070W, Y1014C, E56K, G1249R, Q237H, R1162L, Y1032C, E193K, Q359R, R1283M, E822K, H939R, Q1291R, E831X, H1375P, R74W	>12 months	Sore throat Increases in transaminase levels
	FDA	G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D, R117H.	4 Months
	EMA	R117H, G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R.	6 months
Lumacaftor/Ivacaftor Orkambi^®	FDA and EMA	F508del homozygous	>2 years old	Elevated alanine or aspartate aminotransferase levels. Chest pain Dyspnea	1–2 years old (On going)
Tezacaftor/Ivacaftor Symdeko^® Symkevi^®	FDA	F508del homozygous Have a single copy: A455E, E56K, R74W, A1067T, E193K, R117C, D110E, F1052V, R347H, D110H, F1074L, R352Q, D579G, K1060T, R1070W, D1152H, L206W, S945L, D1270N, P67L, S977F, E831X, 711 + 3A→G, 3272-26A→G, 2789 + 5G→A, 3849 + 10kbC→T	>6 years old	Headache Nasopharyngitis Elevated alanine or aspartate aminotransferase levels.
Tezacaftor/Ivacaftor Symdeko^® Symkevi^®	EMA	F508del homozygous F508del heterozygous with one of this: P67L, R117C, L206W, R352Q, A455E, D579G, 711þ3A > G, S945L, S977F, R1070W, D1152H, 2789þ5G > A, 3272 26A > G, 3849þ10kbC > T	>12 years old
Elexacaftor/Tezacaftor/Ivacaftor Trikafta^® Kaftrio^®	FDA	F508del homozygous All F508del heterozygous	>12 years old	Rash Elevated alanine or aspartate aminotransferase levels. Headache Diarrhea	2–5 years old (On going) 6–11 years old (Completed)
Elexacaftor/Tezacaftor/Ivacaftor Trikafta^® Kaftrio^®	EMA	F508del homozygous F508del heterozygous with minimal function: G542X, W1282X, R553X, R1162X, 621 + 1G→T, 1717-1G→A, 1898 + 1G→A, 3659delC, 394delTT, CFTRdele2,3, N1303K, I507del, G85E, R347P, R560T	>12 years old		2–5 years old (On going) 6–11 years old (Completed)