Ivacaftor

CFTR potentiator

Phase of research

FDA-/EMA-approved for Cystic Fibrosis

How it helps

Restore CFTR Function

General information

Ivacaftor is a drug developed specifically for cystic fibrosis treatment by Vertex Pharmaceuticals. It is a CFTR potentiator, which helps the channel open probability of CFTR protein in patients with impaired CFTR gating mechanisms. This CFTR modulator was approved by the FDA in 2012. On its own, it is marketed as Kalydeco® and indicated for the following mutations: 2789+5G→A, 3272-26A→G, 3849+10kBc→T, 711+3A→G, A1067T, A455E, D110E, D110H, D1152H, D1270N, D579G, E193K, E56K, E831X, F1052V, F1074L, G1069R, G1244E, G1349D, G178R, G551D, G551S, K1060T, L206W, P67L, R1070Q, R1070W, R117C, R117H, R347H, R352Q, R74W, S1251N, S1255P, S549N, S549R, S945L, and S977F. Ivacaftor is also included in the medicine Orkambi®, Symdeko®/Symkevi® and Trikafta™.

Orkambi® combines ivacaftor with the corrector lumacaftor and is indicated for patients with two copies of F508del. Symdeko® combines ivacaftor and the corrector tezacaftor and is indicated for patients with two copies of F508del, or with at least one responsive mutation from the following list: 711+3A→G, 2789+5G→A, 3272-26A→G, 3849+10kbC→T, A455E, A1067T, D110E, D110H, D579G, D1152H, D1270N, E56K, E193K, E831X, F1052V, F1074L, K1060T, L206W, P67L, R74W, R117C, R347H, R352Q, R1070W, S945L, and S977F. Trikafta™ combines ivacaftor with the correctors tezacaftor and elexacaftor and is indicated for patients with at least one copy of the F508del mutation.

In relation to cystic fibrosis, our AIM tool found the data that ivacaftor can help potentiate multiple CFTR mutations. Results from clinical trials indicated that treatment with ivacaftor results in improved lung function, reduced chance of experiencing a pulmonary exacerbation, reduced sweat chloride, increased weight gain, and improvements in cystic fibrosis symptoms and quality of life.

Ivacaftor on PubChem
Ivacaftor on DrugBank
Ivacaftor on Wikipedia

Synonyms

VX-770

Marketed as

Dietary sources

N/A

Structure not available

C24H28N2O3

Drug-Mutation Relation

Treats

Mutation Number of sources Average impact factor
F508del/A455E
1 79.26
Link Tested on Impact factor Notes
Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis humans 79.26 In combination with tezacaftor as Symdeko®. Better than ivacaftor alone.
F508del/R352Q
2 41.78
Link Tested on Impact factor Notes
Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis humans 79.26 In combination with tezacaftor as Symdeko®. Better than ivacaftor alone.
A phase 3 study of tezacaftor in combination with ivacaftor in children aged 6 through 11 years with cystic fibrosis humans 4.29 In combination with tezacaftor as Symdeko®.
F508del/F1052V
1 79.26
Link Tested on Impact factor Notes
Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis humans 79.26 In combination with tezacaftor as Symdeko®. Better than ivacaftor alone.
F508del/D1270N
1 79.26
Link Tested on Impact factor Notes
Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis humans 79.26 In combination with tezacaftor as Symdeko®. Better than ivacaftor alone.
F508del/Q493X
4 70.67
Link Tested on Impact factor Notes
VX-445–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles humans 70.67 In combination with elexacaftor and tezacaftor.
Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis humans 70.67 In combination with tezacaftor and VX-659.
Phase 2 study of VX-440 combination drug in people with cystic fibrosis (Vertex VX-440-101) humans N/A In combination with tezacaftor and VX-440.
Phase 2 study of VX-152 combination drug in people with cystic fibrosis (Vertex VX-152-102) humans N/A In combination with tezacaftor and VX-152.
F508del/S945L
1 79.26
Link Tested on Impact factor Notes
Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis humans 79.26 In combination with tezacaftor as Symdeko®. Better than ivacaftor alone.
S1251N
2 3.18
Link Tested on Impact factor Notes
Ivacaftor potentiation of multiple CFTR channels with gating mutations rat cell cultures 2.87
Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation humans 3.48
S549N
2 3.18
Link Tested on Impact factor Notes
Ivacaftor potentiation of multiple CFTR channels with gating mutations rat cell cultures 2.87
Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation humans 3.48
F508del/R117C
1 79.26
Link Tested on Impact factor Notes
Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis humans 79.26 In combination with tezacaftor as Symdeko®. Better than ivacaftor alone.
F508del/G85E
4 70.67
Link Tested on Impact factor Notes
VX-445–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles humans 70.67 In combination with elexacaftor and tezacaftor.
VX-659–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles humans 70.67 In combination with tezacaftor and VX-659.
Phase 2 study of VX-440 combination drug in people with cystic fibrosis (Vertex VX-440-101) humans N/A In combination with tezacaftor and VX-440.
Phase 2 study of VX-152 combination drug in people with cystic fibrosis (Vertex VX-152-102) humans N/A In combination with tezacaftor and VX-152.
F508del/N1303K
4 70.67
Link Tested on Impact factor Notes
VX-445–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles humans 70.67 In combination with elexacaftor and tezacaftor.
VX-659–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles humans 70.67 In combination with tezacaftor and VX-659.
Phase 2 study of VX-440 combination drug in people with cystic fibrosis (Vertex VX-440-101) humans N/A In combination with tezacaftor and VX-440.
Phase 2 study of VX-152 combination drug in people with cystic fibrosis (Vertex VX-152-102) humans N/A In combination with tezacaftor and VX-152.
F508del/R347H
1 79.26
Link Tested on Impact factor Notes
Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis humans 79.26 In combination with tezacaftor as Symdeko®. Better than ivacaftor alone.
F508del/1154insTC
1 3.16
Link Tested on Impact factor Notes
In vivo and in vitro ivacaftor response in cystic fibrosis patients with residual CFTR function: N-of-1 studies. humans 3.16
F508del/E56K
1 79.26
Link Tested on Impact factor Notes
Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis humans 79.26 In combination with tezacaftor as Symdeko®. Better than ivacaftor alone.
R117H
2 9.39
Link Tested on Impact factor Notes
Folding–function relationship of the most common cystic fibrosis–causing CFTR conductance mutants human cell cultures 3.45
Efficacy and safety of ivacaftor in patients with cystic fibrosis who have an Arg117His-CFTR mutation: a double-blind, randomised controlled trial. humans 15.33
G542X/3849+10kbC->T
1 3.16
Link Tested on Impact factor Notes
In Vivo and In Vitro Ivacaftor Response in Cystic Fibrosis Patients With Residual CFTR Function: N-of-1 Studies humans 3.16
F508del/R1070W
1 79.26
Link Tested on Impact factor Notes
Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis humans 79.26 In combination with tezacaftor as Symdeko®. Better than ivacaftor alone.
G551S
2 3.18
Link Tested on Impact factor Notes
Ivacaftor potentiation of multiple CFTR channels with gating mutations rat cell cultures 2.87
Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation humans 3.48
S1255P
2 3.18
Link Tested on Impact factor Notes
Ivacaftor potentiation of multiple CFTR channels with gating mutations rat cell cultures 2.87
Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation humans 3.48
W1282X
1 2.81
Link Tested on Impact factor Notes
Robust Stimulation of W1282X-CFTR Channel Activity by a Combination of Allosteric Modulators human cell cultures 2.81 Better in combination with curcumin.
F508del/E831X
1 79.26
Link Tested on Impact factor Notes
Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis humans 79.26 In combination with tezacaftor as Symdeko®. Better than ivacaftor alone.
F508del
3 7.77
Link Tested on Impact factor Notes
MicroRNA-145 Antagonism Reverses TGF-β Inhibition of F508del CFTR Correction in Airway Epithelia human cell cultures 16.49 In combination with lumacaftor. Better in combination with lumacaftor and a miR-145 antagonist.
Two Small Molecules Restore Stability to a Subpopulation of the Cystic Fibrosis Transmembrane Conductance Regulator with the Predominant Disease-causing Mutation. yeast cell cultures 4.01 In combination with lumacaftor restores only a subpopulation of F508del-CFTR Cl− channels. 
Mechanistic Approaches to Improve Correction of the Most Common Disease-Causing Mutation in Cystic Fibrosis human cell cultures 2.81 In combination with lumacaftor and Corr-4a.
F508del/3120+1G->A
4 70.67
Link Tested on Impact factor Notes
VX-445–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles humans 70.67 In combination with elexacaftor and tezacaftor.
VX-659–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles humans 70.67 In combination with tezacaftor and VX-659.
Phase 2 study of VX-440 combination drug in people with cystic fibrosis (Vertex VX-440-101) humans N/A In combination with tezacaftor and VX-440.
Phase 2 study of VX-152 combination drug in people with cystic fibrosis (Vertex VX-152-102) humans N/A In combination with tezacaftor and VX-152.
F508del/2184delA
4 70.67
Link Tested on Impact factor Notes
VX-659–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles humans 70.67 In combination with tezacaftor and VX-659.
Phase 2 study of VX-440 combination drug in people with cystic fibrosis (Vertex VX-440-101) humans N/A In combination with tezacaftor and VX-440.
Phase 2 study of VX-152 combination drug in people with cystic fibrosis (Vertex VX-152-102) humans N/A In combination with tezacaftor and VX-152.
VX-445–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles humans 70.67 In combination with elexacaftor and tezacaftor.
E831X
1 5.22
Link Tested on Impact factor Notes
Capitalizing on the heterogeneous effects of CFTR nonsense and frameshift variants to inform therapeutic strategy for cystic fibrosis human cell cultures 5.22 Only after a treatment with lumacaftor and tezacaftor.
F508del/3659delC
4 70.67
Link Tested on Impact factor Notes
VX-445–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles humans 70.67 In combination with elexacaftor and tezacaftor.
VX-659–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles humans 70.67 In combination with tezacaftor and VX-659.
Phase 2 study of VX-440 combination drug in people with cystic fibrosis (Vertex VX-440-101) humans N/A In combination with tezacaftor and VX-440.
Phase 2 study of VX-152 combination drug in people with cystic fibrosis (Vertex VX-152-102) humans N/A In combination with tezacaftor and VX-152.
F508del/E193K
1 79.26
Link Tested on Impact factor Notes
Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis humans 79.26 In combination with tezacaftor as Symdeko®. Better than ivacaftor alone.
F508del/I507del
4 70.67
Link Tested on Impact factor Notes
VX-445–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles humans 70.67 In combination with elexacaftor and tezacaftor.
VX-659–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles humans 70.67 In combination with tezacaftor and VX-659.
Phase 2 study of VX-440 combination drug in people with cystic fibrosis (Vertex VX-440-101) humans N/A In combination with tezacaftor and VX-440.
Phase 2 study of VX-152 combination drug in people with cystic fibrosis (Vertex VX-152-102) humans N/A In combination with tezacaftor and VX-152.
F508del/G542X
4 70.67
Link Tested on Impact factor Notes
VX-445–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles humans 70.67 In combination with elexacaftor and tezacaftor.
VX-659–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles humans 70.67 In combination with tezacaftor and VX-659.
Phase 2 study of VX-440 combination drug in people with cystic fibrosis (Vertex VX-440-101) humans N/A In combination with tezacaftor and VX-440.
Phase 2 study of VX-152 combination drug in people with cystic fibrosis (Vertex VX-152-102) humans N/A In combination with tezacaftor and VX-152.
G970R
2 3.18
Link Tested on Impact factor Notes
Ivacaftor potentiation of multiple CFTR channels with gating mutations rat cell cultures 2.87
Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation humans 3.48
F508del/R553X
4 70.67
Link Tested on Impact factor Notes
VX-445–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles humans 70.67 In combination with elexacaftor and tezacaftor.
VX-659–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles humans 70.67 In combination with tezacaftor and VX-659.
Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis humans N/A In combination with tezacaftor and VX-440.
Phase 2 study of VX-152 combination drug in people with cystic fibrosis (Vertex VX-152-102) humans N/A In combination with tezacaftor and VX-152.
G551D/F508del
2 21.14
Link Tested on Impact factor Notes
Safety, pharmacokinetics, and pharmacodynamics of ivacaftor in patients aged 2-5 years with cystic fibrosis and a CFTR gating mutation (KIWI): an open-label, single-arm study. humans 19.29
Ivacaftor treatment of cystic fibrosis in children aged 12 to <24 months and with a CFTR gating mutation (ARRIVAL): a phase 3 single-arm study. humans 22.99
F508del/F1074L
1 79.26
Link Tested on Impact factor Notes
Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis humans 79.26 In combination with tezacaftor as Symdeko®. Better than ivacaftor alone.
R117H-5T/F508del
1 2.77
Link Tested on Impact factor Notes
The magnitude of ivacaftor effects on fluid secretion via R117H-CFTR channels: Human in vivo measurements humans 2.77 Only minimal effect.
F508del/K1060T
1 79.26
Link Tested on Impact factor Notes
Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis humans 79.26 In combination with tezacaftor as Symdeko®. Better than ivacaftor alone.
G1244E
2 3.18
Link Tested on Impact factor Notes
Ivacaftor potentiation of multiple CFTR channels with gating mutations rat cell cultures 2.87
Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation humans 3.48
F508del/R347P
4 70.67
Link Tested on Impact factor Notes
VX-445–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles humans 70.67 In combination with elexacaftor and tezacaftor.
VX-659–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles humans 70.67 In combination with tezacaftor and VX-659.
Phase 2 study of VX-440 combination drug in people with cystic fibrosis (Vertex VX-440-101) humans N/A In combination with tezacaftor and VX-440.
Phase 2 study of VX-152 combination drug in people with cystic fibrosis (Vertex VX-152-102) humans N/A In combination with tezacaftor and VX-152.
F508del/3272-26A->G
2 41.78
Link Tested on Impact factor Notes
Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis humans 79.26 In combination with tezacaftor as Symdeko®. Better than ivacaftor alone.
A phase 3 study of tezacaftor in combination with ivacaftor in children aged 6 through 11 years with cystic fibrosis humans 4.29 In combination with tezacaftor as Symdeko®.
S549R
2 3.18
Link Tested on Impact factor Notes
Ivacaftor potentiation of multiple CFTR channels with gating mutations rat cell cultures 2.87
Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation humans 3.48
F508del/D1152H
2 41.78
Link Tested on Impact factor Notes
Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis humans 79.26 In combination with tezacaftor as Symdeko®. Better than ivacaftor alone.
A phase 3 study of tezacaftor in combination with ivacaftor in children aged 6 through 11 years with cystic fibrosis humans 4.29 In combination with tezacaftor as Symdeko®.
G1349D
2 3.18
Link Tested on Impact factor Notes
Ivacaftor potentiation of multiple CFTR channels with gating mutations rat cell cultures 2.87
Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation humans 3.48
F508del/711+3A->G
1 79.26
Link Tested on Impact factor Notes
Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis humans 79.26 In combination with tezacaftor as Symdeko®. Better than ivacaftor alone.
F508del/2184insA
4 70.67
Link Tested on Impact factor Notes
VX-445–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles humans 70.67 In combination with elexacaftor and tezacaftor.
VX-659–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles humans 70.67 In combination with tezacaftor and VX-659.
Phase 2 study of VX-440 combination drug in people with cystic fibrosis (Vertex VX-440-101) humans N/A In combination with tezacaftor and VX-440.
Phase 2 study of VX-152 combination drug in people with cystic fibrosis (Vertex VX-152-102) humans N/A In combination with tezacaftor and VX-152.
F508del/R1162X
4 70.67
Link Tested on Impact factor Notes
VX-445–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles humans 70.67 In combination with elexacaftor and tezacaftor.
VX-659–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles humans 70.67 In combination with tezacaftor and VX-659.
Phase 2 study of VX-440 combination drug in people with cystic fibrosis (Vertex VX-440-101) humans N/A In combination with tezacaftor and VX-440.
Phase 2 study of VX-152 combination drug in people with cystic fibrosis (Vertex VX-152-102) humans N/A In combination with tezacaftor and VX-152.
G178R
3 3.18
Link Tested on Impact factor Notes
Ivacaftor potentiation of multiple CFTR channels with gating mutations rat cell cultures 2.87
Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation humans 3.48
Open-label study of ivacaftor in people with CF who have a non-G551D CFTR gating mutation (Vertex VX-770-112) humans N/A
F508del/R560T
4 70.67
Link Tested on Impact factor Notes
VX-445–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles humans 70.67 In combination with elexacaftor and tezacaftor.
VX-659–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles humans 70.67 In combination with tezacaftor and VX-659.
Phase 2 study of VX-440 combination drug in people with cystic fibrosis (Vertex VX-440-101) humans N/A In combination with tezacaftor and VX-440.
Phase 2 study of VX-152 combination drug in people with cystic fibrosis (Vertex VX-152-102) humans N/A In combination with tezacaftor and VX-152.
F508del/1898+1G->A
4 70.67
Link Tested on Impact factor Notes
Phase 2 study of VX-440 combination drug in people with cystic fibrosis (Vertex VX-440-101) humans N/A In combination with tezacaftor and VX-440.
Phase 2 study of VX-152 combination drug in people with cystic fibrosis (Vertex VX-152-102) humans N/A In combination with tezacaftor and VX-152.
VX-445–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles humans 70.67 In combination with elexacaftor and tezacaftor.
VX-659–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles humans 70.67 In combination with tezacaftor and VX-659.
F508del/3849+10kbC->T
2 41.78
Link Tested on Impact factor Notes
Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis humans 79.26 In combination with tezacaftor as Symdeko®. Better than ivacaftor alone.
A phase 3 study of tezacaftor in combination with ivacaftor in children aged 6 through 11 years with cystic fibrosis humans 4.29 In combination with tezacaftor as Symdeko®.
F508del/R74W
1 79.26
Link Tested on Impact factor Notes
Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis humans 79.26 In combination with tezacaftor as Symdeko®. Better than ivacaftor alone.
E1418X
1 5.22
Link Tested on Impact factor Notes
Capitalizing on the heterogeneous effects of CFTR nonsense and frameshift variants to inform therapeutic strategy for cystic fibrosis human cell cultures 5.22 Only minimal effect on its own. Better in combination with lumacaftor and tezacaftor.
G551D
11 16.22
Link Tested on Impact factor Notes
Long-term safety and efficacy of ivacaftor in patients with cystic fibrosis who have the Gly551Asp-CFTR mutation: a phase 3, open-label extension study (PERSIST). humans 9.63
Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation. humans 53.48
A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. humans 53.30
Efficacy and safety of ivacaftor in patients aged 6 to 11 years with cystic fibrosis with a G551D mutation. humans 11.99
Potentiation of ΔF508- and G551D-CFTR-Mediated Cl- Current by Novel Hydroxypyrazolines. human cell cultures 2.81
Potentiator Ivacaftor Abrogates Pharmacological Correction of ΔF508 CFTR in Cystic Fibrosis human cell cultures 16.26
A Little CFTR Goes a Long Way: CFTR-Dependent Sweat Secretion from G551D and R117H-5T Cystic Fibrosis Subjects Taking Ivacaftor humans 3.23
Molecular Mechanism of Action of Trimethylangelicin Derivatives as CFTR Modulators human cell cultures 3.85
Ivacaftor potentiation of multiple CFTR channels with gating mutations rat cell cultures 2.87
Effect of ivacaftor in patients with advanced cystic fibrosis and a G551D-CFTR mutation: Safety and efficacy in an expanded access program in the United States. humans 4.73
Assessment of clinical response to ivacaftor with lung clearance index in cystic fibrosis patients with a G551D-CFTR mutation and preserved spirometry: a randomised controlled trial. humans N/A
R334W
1 3.45
Link Tested on Impact factor Notes
Folding–function relationship of the most common cystic fibrosis–causing CFTR conductance mutants human cell cultures 3.45
R117H-7T/R117H-7T
1 2.77
Link Tested on Impact factor Notes
The magnitude of ivacaftor effects on fluid secretion via R117H-CFTR channels: Human in vivo measurements humans 2.77
F508del/W1282X
4 70.67
Link Tested on Impact factor Notes
VX-445–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles humans 70.67 In combination with elexacaftor and tezacaftor.
VX-659–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles humans 70.67 In combination with tezacaftor and VX-659.
Phase 2 study of VX-440 combination drug in people with cystic fibrosis (Vertex VX-440-101) humans N/A In combination with tezacaftor and VX-440.
Phase 2 study of VX-152 combination drug in people with cystic fibrosis (Vertex VX-152-102) humans N/A In combination with tezacaftor and VX-152.
F508del/D579G
2 41.78
Link Tested on Impact factor Notes
Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis humans 79.26 In combination with tezacaftor as Symdeko®. Better than ivacaftor alone.
A phase 3 study of tezacaftor in combination with ivacaftor in children aged 6 through 11 years with cystic fibrosis humans 4.29 In combination with tezacaftor as Symdeko®.
F508del/S977F
1 79.26
Link Tested on Impact factor Notes
Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis humans 79.26 In combination with tezacaftor as Symdeko®. Better than ivacaftor alone.
R117H-5T
1 3.23
Link Tested on Impact factor Notes
A Little CFTR Goes a Long Way: CFTR-Dependent Sweat Secretion from G551D and R117H-5T Cystic Fibrosis Subjects Taking Ivacaftor humans 3.23
F508del/F508del
17 34.76
Link Tested on Impact factor Notes
Assessment of safety and efficacy of long-term treatment with combination lumacaftor and ivacaftor therapy in patients with cystic fibrosis homozygous for the F508del-CFTR mutation (PROGRESS): a phase 3, extension study. humans 21.47 In combination with lumacaftor as Orkambi®.
Vertex Lumacaftor (VX-809) and Ivacaftor in Children with CF aged 6 to 11 years and have two copies of the delF508 CFTR mutation (Vertex VX13-809-011b) humans 15.24 In combination with lumacaftor as Orkambi®.
Phase 3 study of lumacaftor/ivacaftor in children with CF (Vertex VX15-809-115) humans N/A In combination with lumacaftor as Orkambi®.
Efficacy and safety of lumacaftor and ivacaftor in patients aged 6-11 years with cystic fibrosis homozygous for F508del-CFTR: a randomised, placebo-controlled phase 3 trial. humans 21.47 In combination with lumacaftor as Orkambi®.
Lumacaftor/ivacaftor in patients with cystic fibrosis and advanced lung disease homozygous for F508del-CFTR. humans 4.29 In combination with lumacaftor as Orkambi®.
A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2 randomised controlled trial. humans 9.63 In combination with lumacaftor as Orkambi®.
Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR. humans 59.56 In combination with lumacaftor as Orkambi®.
A phase 3 study of tezacaftor in combination with ivacaftor in children aged 6 through 11 years with cystic fibrosis humans 4.29 In combination with tezacaftor as Symdeko®.
Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del humans 79.26 In combination with tezacaftor as Symdeko®.
Study of VX-661 combined with ivacaftor in adults with cystic fibrosis (Vertex VX-661-103) humans N/A In combination with tezacaftor as Symdeko®.
Tezacaftor/Ivacaftor in Subjects with Cystic Fibrosis and F508del/F508del-CFTR or F508del/G551D-CFTR humans 16.49 Better in combination with tezacaftor as Symdeko®.
VX-445–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles humans 70.67 In combination with tezacaftor. Better in combination with tezacaftor plus elexacaftor.
VX-659–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles humans 70.67 In combination with tezacaftor and VX-659.
Phase 2 study of VX-440 combination drug in people with cystic fibrosis (Vertex VX-440-101) humans N/A In combination with tezacaftor and VX-440.
Phase 2 study of VX-152 combination drug in people with cystic fibrosis (Vertex VX-152-102) humans N/A In combination with tezacaftor and VX-152.
Efficacy and safety of lumacaftor/ivacaftor combination therapy in patients with cystic fibrosis homozygous for Phe508del CFTR by pulmonary function subgroup humans 19.29 In combination with lumacaftor.
Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR humans 59.56 Tested in combination with lumacaftor.
F508del/D110H
1 79.26
Link Tested on Impact factor Notes
Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis humans 79.26 In combination with tezacaftor as Symdeko®. Better than ivacaftor alone.
F508del/621+1G->T
4 70.67
Link Tested on Impact factor Notes
VX-445–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles humans 70.67 In combination with elexacaftor and tezacaftor.
Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis humans 70.67 In combination with tezacaftor and VX-659.
Phase 2 study of VX-440 combination drug in people with cystic fibrosis (Vertex VX-440-101) humans N/A In combination with tezacaftor and VX-440.
Phase 2 study of VX-152 combination drug in people with cystic fibrosis (Vertex VX-152-102) humans N/A In combination with tezacaftor and VX-152.
F508del/2789+5G->A
2 41.78
Link Tested on Impact factor Notes
Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis humans 79.26 In combination with tezacaftor as Symdeko®. Better than ivacaftor alone.
A phase 3 study of tezacaftor in combination with ivacaftor in children aged 6 through 11 years with cystic fibrosis humans 4.29 In combination with tezacaftor as Symdeko®.
F508del/P67L
1 79.26
Link Tested on Impact factor Notes
Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis humans 79.26 In combination with tezacaftor as Symdeko®. Better than ivacaftor alone.
F508del/D110E
1 79.26
Link Tested on Impact factor Notes
Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis humans 79.26 In combination with tezacaftor as Symdeko®. Better than ivacaftor alone.
F508del/L206W
2 41.78
Link Tested on Impact factor Notes
Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis humans 79.26 In combination with tezacaftor as Symdeko®. Better than ivacaftor alone.
A phase 3 study of tezacaftor in combination with ivacaftor in children aged 6 through 11 years with cystic fibrosis humans 4.29 In combination with tezacaftor as Symdeko®.
F508del/G551D
1 16.49
Link Tested on Impact factor Notes
Tezacaftor/Ivacaftor in Subjects with Cystic Fibrosis and F508del/F508del-CFTR or F508del/G551D-CFTR humans 16.49 Better in combination with tezacaftor as Symdeko®.
F508del/Y563N
1 3.16
Link Tested on Impact factor Notes
In Vivo and In Vitro Ivacaftor Response in Cystic Fibrosis Patients With Residual CFTR Function: N-of-1 Studies humans 3.16
F508del/1717-1G->A
4 70.67
Link Tested on Impact factor Notes
VX-659–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles humans 70.67 In combination with tezacaftor and VX-659.
Phase 2 study of VX-440 combination drug in people with cystic fibrosis (Vertex VX-440-101) humans N/A In combination with tezacaftor and VX-440.
Phase 2 study of VX-152 combination drug in people with cystic fibrosis (Vertex VX-152-102) humans N/A In combination with tezacaftor and VX-152.
VX-445–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles humans 70.67 In combination with elexacaftor and tezacaftor.

Does not treat

Mutation Number of sources Average impact factor
F508del/4016insT
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/1717-1G->A
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/S341P6
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/2183AA->G
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/A455E
1 3.16
Link Tested on Impact factor Notes
In Vivo and In Vitro Ivacaftor Response in Cystic Fibrosis Patients With Residual CFTR Function: N-of-1 Studies humans 3.16
F508del/2183delAA->G
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/Q290X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/W1089X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/L1065P
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/3850-3T->G
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/1677delTA
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/A46D6
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/C276X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/E730X
1 3.45
Link Tested on Impact factor Notes
Folding–function relationship of the most common cystic fibrosis–causing CFTR conductance mutants human cell cultures 3.45
F508del/E585X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/S1255X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/Q493X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/Q525X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/3876delA
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/T338I8
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/3905insT
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/K710X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/G85E
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/N1303K
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/V520F
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/1154insTC
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/1811+1.6kbA->G
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/574delA
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/1341+1G->A
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/541delC
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/L732X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/R785X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/Q220X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
R347P
1 3.45
Link Tested on Impact factor Notes
Folding–function relationship of the most common cystic fibrosis–causing CFTR conductance mutants human cell cultures 3.45 Not even in combination with lumacaftor.
F508del/3007delG
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/R709X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/2347delG
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/3120+1G->A
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/Y569D6
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/2143delT
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/3737delA
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/2184delA
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/3791delC
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/S466X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/3659delC
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/405+1G->A
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/Y122X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/CFTRdel2,3
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
1717-1G->A/G85E
1 3.16
Link Tested on Impact factor Notes
In Vivo and In Vitro Ivacaftor Response in Cystic Fibrosis Patients With Residual CFTR Function: N-of-1 Studies humans 3.16
F508del/E822X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/S1196X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/R1066C
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/R764X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/I507del
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
1717-1G->A/A455E
1 3.16
Link Tested on Impact factor Notes
In Vivo and In Vitro Ivacaftor Response in Cystic Fibrosis Patients With Residual CFTR Function: N-of-1 Studies humans 3.16
F508del/G542X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/R553X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/R1158X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/G330X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/R560S
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/R347P
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/2307insA
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/E92X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/A561E
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/1078delT
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/E60X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/A559T6
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/4040delA
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/W57X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/711+5G->A
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/3120G->A
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/L218X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/Q98X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/E1104X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/3600+2insT
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/2184insA
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/H1085R6
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/W1204X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/711+1G->T
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/1812-1G->A
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/406-1G->A
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/2711delT
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/G673X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/1248+1G->A
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/Q414X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/R1162X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/S489X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/R560T
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
LXX8
1 5.22
Link Tested on Impact factor Notes
Capitalizing on the heterogeneous effects of CFTR nonsense and frameshift variants to inform therapeutic strategy for cystic fibrosis human cell cultures 5.22 Alone or in combination with lumacaftor.
F508del/Q1313X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/4005+1G->A
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/712-1G->T
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/1898+1G->A
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/394delTT
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/Q39X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/457TAT->G
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/663delT
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/R792X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/4374+1G->T
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/W496X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/R851X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/W1282X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/L1077P6
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/S434X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/405+3A->C
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/4382delA7
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/2622+1G->A
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/2869insG
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/1811+1G->C
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/3850-1G->A
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/R75X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/F508del
1 5.85
Link Tested on Impact factor Notes
Ivacaftor in subjects with cystic fibrosis who are homozygous for the F508del-CFTR mutation. humans 5.85
F508del/R1066M
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/L927P
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/M1101K
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/621+1G->T
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/Q552X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/2043delG
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/S912X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/W846X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/W401X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/Y1092X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/Q890X
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/CFTRdele22-23
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.
F508del/L467P6
1 0.00
Link Tested on Impact factor Notes
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107) humans N/A In combination with tezacaftor as Symdeko®.