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

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Synonyms

VX-770

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Marketed as

• Kalydeco - prescribing information
• Orkambi - prescribing information
• Symdeko - prescribing information
• Trikafta - prescribing information

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Dietary sources

N/A