CFTR potentiator

Ivacaftor

Brand names: Kalydeco

Ivacaftor is a CFTR potentiator used in the treatment of cystic fibrosis in patients with specified responsive CFTR gene mutations.

Dosing — being independently re-sourced

ClinCalc Pro is rebuilding its dose data from primary open sources — the manufacturer SmPC (eMC), the WHO Model Formulary and other official references — under clinician review. This drug's structured dose is not yet published here. Confirm all doses against the product SmPC and your local formulary before prescribing.

Clinical monograph

How it works

It increases the channel-open probability (gating) of CFTR protein at the cell surface, enhancing chloride ion transport and improving epithelial fluid balance.

Prescribing in practice

Monitor liver function, as elevated transaminases and hepatobiliary events have been reported with CFTR modulator therapy.
Exposure is altered by CYP3A inducers and inhibitors, so review interacting drugs and avoid potent inducers; advise avoiding grapefruit.
Cases of cataracts have been reported in paediatric patients, warranting baseline and follow-up eye examinations.

Monitoring

Monitor liver transaminases before and periodically during treatment, with ophthalmological examinations in children.

Counselling the patient

Take with fat-containing food to help absorption.
Report jaundice, dark urine or abdominal pain promptly.
Avoid grapefruit and Seville orange products.

Evidence & guidelines

Ivacaftor is supported by randomised trials and NICE access arrangements showing improved lung function and reduced sweat chloride in eligible patients.

Reference: NICE TA398; SmPC; Drug verified in RxNorm (NLM); confirm dosing against the manufacturer SPC (eMC). Verify against your local formulary and current prescribing references before prescribing. Monograph status: clinician-reviewed (2026-07-04).

Curated clinical cross-links plus same-class fallbacks.

Pathways