Ivacaftor
Ivacaftor is a CFTR potentiator medication designed to treat certain forms of cystic fibrosis in patients with specific genetic mutations. It works by improving the function of defective chloride channels at the surface of epithelial cells, facilitating increased chloride transport. This targeted approach addresses the underlying defect in cystic fibrosis rather than just managing symptoms.
Ivacaftor is a cystic fibrosis medication that improves CFTR protein function to enhance chloride transport in patients with specific responsive mutations.
Key facts at a glance
- ATC
- R07AX02
- RxNorm
- 1243041
- Generic
- Ivacaftor
- Class
- atc-r07
- Brands
- Kalydeco, ORKAMBI
- Availability
- rx
- Lactation
- unknown
- Routes
- oral
- Forms
- tablet
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Treatment of cystic fibrosis in patients with specific CFTR gene mutations that result in defective channel gating. Treatment eligibility requires confirmed presence of these mutations through genetic testing.
Ivacaftor is a potentiator that binds to the CFTR protein and increases the channel open probability (gating), allowing increased chloride transport across epithelial cell membranes in multiple organs including the lungs, pancreas, and sweat glands.
Effectiveness is limited to patients with specific CFTR mutations responsive to potentiation; not all cystic fibrosis mutations respond to this mechanism. Requires genetic testing to confirm eligible mutations before treatment initiation.
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy