SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) is indicated for the treatment of patients with cystic fibrosis (CF) age 6 years and older who are homozygous for the F508del mutation or who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to tezacaftor/ivacaftor based on in vitro data and/or clinical evidence.

CFTR mutations with
clinical data

CFTR mutations with
in vitro data only

CFTR Mutations That Produce CFTR Protein Responsive to SYMDEKO Based on Clinical or In Vitro Data1-3*

F508del/F508del*
c.1521_1523delCTT
E56K
c.166G>A
R347H
c.1040G>A
A1067T
c.3199G>A
E831X
c.2491G>T
R352Q
c.1055G>A
A455E
c.1364C>A
F1052V
c.3154T>G
R74W
c.220C>T
D110E
c.330C>A
F1074L
c.3222T>A
S945L
c.2834C>T
D110H
c.328G>C
K1060T
c.3179A>C
S977F
c.2930C>T
D1152H
c.3454G>C
L206W
c.617T>G
2789+5GA
c.2657+5G>A
D1270N
c.3808G>A
P67L
c.200C>T
3272-26AG
c.3140-26A>G
D579G
c.1736A>G
R1070W
c.3208C>T
3849+10kbCT
c.3718-2477C>T
E193K
c.577G>A
R117C
c.349C>T
711+3AG
c.579+3A>G

A patient must have 2 copies of the F508del mutation or at least one copy of a responsive mutation listed above in this table to be indicated.

Barbara, a CF patient age 28, with other mutations predicted to be responsive to
                        SYMDEKO

See EXPAND (Trial 2) Results

aPatients who have one responsive mutation do not also need to have an F508del mutation.