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 2143delT / G551D
See all data on Ivacaftor

Treats

Mutation Link Tested on Impact factor Notes
G551D 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
G551D Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation. humans 53.48
G551D A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. humans 53.30
G551D Efficacy and safety of ivacaftor in patients aged 6 to 11 years with cystic fibrosis with a G551D mutation. humans 11.99
G551D Potentiation of ΔF508- and G551D-CFTR-Mediated Cl- Current by Novel Hydroxypyrazolines. human cell cultures 2.81
G551D Potentiator Ivacaftor Abrogates Pharmacological Correction of ΔF508 CFTR in Cystic Fibrosis human cell cultures 16.26
G551D A Little CFTR Goes a Long Way: CFTR-Dependent Sweat Secretion from G551D and R117H-5T Cystic Fibrosis Subjects Taking Ivacaftor humans 3.23
G551D Molecular Mechanism of Action of Trimethylangelicin Derivatives as CFTR Modulators human cell cultures 3.85
G551D Ivacaftor potentiation of multiple CFTR channels with gating mutations rat cell cultures 2.87
G551D 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
G551D 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

Does not treat

Mutation Link Tested on Impact factor Notes