Lumacaftor and Ivacaftor
Orkambi is a prescription medication combining lumacaftor and ivacaftor, designed to treat cystic fibrosis in patients who have two copies of the F508del mutation in the CFTR gene. It is specifically approved for use in children ages 2 years and older with this genetic form of the disease. Orkambi works by targeting the underlying cause of CF rather than just managing symptoms.
Orkambi is a CFTR modulator used to treat cystic fibrosis in patients with two copies of the F508del mutation.
Key facts at a glance
- RxNorm
- 1655929
- Generic
- Orkambi
- Class
- brand-name
- Brands
- —
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- —
- 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.
Orkambi is indicated for the treatment of cystic fibrosis in patients ages 2 years and older who have two copies of the F508del mutation in the CFTR gene. Genetic testing is required to confirm eligibility before starting treatment.
Orkambi contains two active ingredients: lumacaftor and ivacaftor. Lumacaftor helps the F508del-CFTR protein fold into its proper shape and reach the cell surface, while ivacaftor helps the protein function more effectively once at the surface, improving chloride transport across cell membranes.
Orkambi may cause side effects including respiratory events, liver enzyme elevations, and menstrual irregularities. Blood pressure should be monitored regularly. Patients should avoid taking with strong CYP3A inducers as they can decrease the effectiveness of the medication. Always take exactly as prescribed and do not adjust dose without consulting a healthcare provider.
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