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

results for 1078delT / F508del
See all data on Ivacaftor

Treats

Mutation Link Tested on Impact factor Notes
F508del/F508del 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®.
F508del/F508del 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®.
F508del/F508del Phase 3 study of lumacaftor/ivacaftor in children with CF (Vertex VX15-809-115) humans N/A In combination with lumacaftor as Orkambi®.
F508del/F508del 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®.
F508del/F508del Lumacaftor/ivacaftor in patients with cystic fibrosis and advanced lung disease homozygous for F508del-CFTR. humans 4.29 In combination with lumacaftor as Orkambi®.
F508del/F508del 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®.
F508del/F508del Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR. humans 59.56 In combination with lumacaftor as Orkambi®.
F508del/F508del 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/F508del Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del humans 79.26 In combination with tezacaftor as Symdeko®.
F508del/F508del 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®.
F508del/F508del 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/F508del 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.
F508del/F508del 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/F508del 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.
F508del/F508del 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 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.
F508del 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. 
F508del/F508del 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.
F508del 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/F508del Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR humans 59.56 Tested in combination with lumacaftor.

Does not treat

Mutation Link Tested on Impact factor Notes
F508del/F508del Ivacaftor in subjects with cystic fibrosis who are homozygous for the F508del-CFTR mutation. humans 5.85
F508del/1078delT 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®.