Lumacaftor And Ivacaftor
Pronunciation: (loo-MA-ka-for / eye-VA-ka-for).
- Generic name
- lumacaftor and ivacaftor
- Brand names
- Ivafect, Lumivac, Orkambi
- Drug class
- Respiratory System
Availability and supply rules vary by country
Trade names by country: United States
Australia: · Full directory
What lumacaftor and ivacaftor is and how it works
Uses, formulations, monitoring, and supply rules can differ by patient, product, and country. Follow the exact local product information and professional advice.
Mechanism of action: how lumacaftor and ivacaftor works
Lumacaftor is a CFTR corrector that improves the conformational stability of the F508del-CFTR protein, increasing its processing and trafficking to the cell surface. Ivacaftor is a CFTR potentiator that increases the channel open probability (gating) of the CFTR protein at the cell surface. Together, they enhance the quantity and function of CFTR protein in patients homozygous for the F508del mutation, improving chloride transport and reducing disease manifestations.
What lumacaftor and ivacaftor is used for
Cystic fibrosis in patients aged 1 year and older who are homozygous for the F508del mutation in the CFTR gene
Cystic fibrosis in patients aged 1 year and older who are homozygous for the F508del mutation in the CFTR gene (2)
Before taking lumacaftor and ivacaftor
Do not take / not appropriate for
- Contraindicated in patients with known hypersensitivity to lumacaftor, ivacaftor, or any excipients. (Contraindication) (2)
Tell your clinician about
(2)How to take lumacaftor and ivacaftor
- General Instructions: Take Lumacaftor/Ivacaftor exactly as prescribed, usually every 12 hours with fat-containing food to enhance absorption.. Adult and Adolescent Dosing (12 years and older): 2 tablets (each containing 200 mg lumacaftor/125 mg ivacaftor) orally every 12 hours with food.. Pediatric Dosing: Dosing is established from age 1 year and older, based on age and weight, using oral granules (ages 1–5) or tablets (ages 6 and older), taken every 12 hours with fat-containing food. The exact strength and packet/tablet count is set by the prescribing team according to the child's age and weight band.. Administration: Swallow tablets whole; do not crush or chew. For granules, mix the full packet with a teaspoon of age-appropriate soft food or liquid and consume the entire mixture.. When To Stop: Seek medical attention promptly if you notice signs of liver problems (such as yellowing skin/eyes, dark urine, right-upper-abdomen pain) or worsening breathing symptoms; let the clinician you see advise on whether to continue, reduce, or stop Orkambi. Do not stop on your own without medical guidance, even if you feel well.. Storage: Store at room temperature (20–25°C/68–77°F), away from moisture and heat. Keep out of reach of children.
- This page does not provide a dose calculator. Dosing must follow the prescriber, pharmacist, or product label.
Procedures and treatment changes
Tell the treating team before surgery, procedures, or any medicine change. Do not alter treatment without the responsible clinician. (2)
Missed dose
If you miss a dose and it is within 6 hours of your usual time, take it as soon as possible with food. If more than 6 hours have passed, skip the missed dose and resume your regular schedule. Do not double up doses.
Overdose
In case of overdose, seek immediate medical attention or contact a Poison Control Center. Supportive care and monitoring of vital signs and liver function are recommended. (2)
Side effects
Side effects and their urgency vary; report severe, persistent, or concerning symptoms to a healthcare professional.
Dyspnea
Incidence: Reported in the clinician-authored database; frequency not established
Nausea
Incidence: Reported in the clinician-authored database; frequency not established
Diarrhea
Incidence: Reported in the clinician-authored database; frequency not established
Upper respiratory tract infection
Incidence: Reported in the clinician-authored database; frequency not established
Rash
Incidence: Reported in the clinician-authored database; frequency not established
Chest discomfort
Incidence: Reported in the clinician-authored database; frequency not established
Liver enzyme elevations
Incidence: Reported in the clinician-authored database; frequency not established
Cataracts
Incidence: Reported in the clinician-authored database; frequency not established
Serious warnings
Serious, sometimes fatal, liver problems have been reported in people with pre-existing cirrhosis and portal hypertension who took Orkambi, including cases of liver failure — serious
Symptoms: Serious, sometimes fatal, liver problems have been reported in people with pre-existing cirrhosis and portal hypertension who took Orkambi, including cases of liver failure. Anyone with advanced liver disease should only take Orkambi if the prescribing team judges the benefit outweighs this risk, typically with a reduced dose and closer monitoring. Seek prompt medical advice for signs such as yellowing of the skin or eyes, dark urine, or pain in the upper right abdomen.
Action: Follow the urgency stated and seek prompt or emergency care when indicated. (2)
May cause liver enzyme elevations — serious
Symptoms: May cause liver enzyme elevations; routine liver function monitoring is recommended (before starting, every 3 months in year one, and periodically thereafter — exact schedule set by the prescribing team). May worsen respiratory symptoms, especially when starting treatment. Hormonal contraceptives should not be relied upon as an effective method of contraception during Orkambi treatment. Use with caution in patients with severe renal or hepatic impairment. Not recommended for use in patients with unknown CFTR mutation status. Non-congenital lens opacities (cataracts) have been reported in pediatric patients; baseline and follow-up eye examinations are recommended when starting Orkambi in children.
Action: Follow the urgency stated and seek prompt or emergency care when indicated. (2)
The FDA label lists no formal contraindications for Orkambi — serious
Symptoms: The FDA label lists no formal contraindications for Orkambi. Avoid use if you have had a serious allergic reaction to lumacaftor, ivacaftor, or Orkambi's other ingredients. Dose is reduced (not withheld outright) for moderate-to-severe hepatic impairment (Child-Pugh B or C), with liver function tests monitored before and during treatment. Use with caution in patients with advanced renal impairment or organ transplantation.
Action: Follow the urgency stated and seek prompt or emergency care when indicated. (2)
Interactions
| Medicine or test | Effect | Action |
|---|---|---|
| Recorded interactions | Lumacaftor/Ivacaftor can interact with several medications, including hormonal contraceptives, antifungals, antibiotics (such as rifampin), anticonvulsants, and certain immunosuppressants. Always inform your healthcare provider about all medications and supplements you are taking. | |
| Strong CYP3A inducers (e.g., rifampin, St. John's wort) | Taking Orkambi together with strong CYP3A-inducing medicines or supplements is not recommended, because they can substantially lower the level of ivacaftor in the body and reduce how well Orkambi works. | (2) |
| Strong CYP3A inhibitors (certain antifungals, some antibiotics) | When starting Orkambi in someone already taking a strong CYP3A-inhibiting medicine, the prescribing team typically uses a temporarily reduced Orkambi dose for the first week before returning to the usual dose. | (2) |
| Hormonal contraceptives | Hormonal contraceptives (pills, patches, rings, implants) should not be relied upon as an effective method of birth control while taking Orkambi; combined use has also been linked to more menstruation-related side effects. Discuss alternative or backup contraception with your healthcare provider. | (2) |
Pregnancy, breastfeeding, kidney/liver function, age
Pregnancy
Human data on Orkambi in pregnancy are limited and incomplete. Animal studies at several multiples of the human dose did not show harm to the fetus, but this does not guarantee human safety. Use during pregnancy should be a shared decision with the prescribing team, weighing the mother's need for CF treatment against any potential risk. (2)
Breastfeeding
It is not known whether lumacaftor or ivacaftor pass into human breast milk (both have been found in the milk of nursing animals). The developmental and health benefits of breastfeeding should be weighed against the mother's clinical need for Orkambi and any potential effect on the infant — discuss this with the prescribing team. (2)
Children
Pediatric use is established from age 1 year and older, homozygous for the F508del mutation, using age/weight-based dosing: oral granules for ages 1–5 years, and tablets for ages 6 years and older, both given every 12 hours with fat-containing food. The exact packet or tablet strength for a given age/weight band is set by the prescribing team — this page does not provide a pediatric dose calculator. (2)
Renal_impairment
Hepatic impairment: no dose adjustment for mild impairment (Child-Pugh A); reduced dosing for moderate impairment (Child-Pugh B); use with caution at a further-reduced dose (or less frequent dosing) for severe impairment (Child-Pugh C), only if the benefit is expected to outweigh the risk, with closer monitoring — see the professional information for the exact regimen. Renal impairment: no adjustment needed for mild-to-moderate impairment; use with caution in severe impairment (creatinine clearance <=30 mL/min) or end-stage renal disease, where data are limited. (2)
Hepatic_impairment
Hepatic impairment: no dose adjustment for mild impairment (Child-Pugh A); reduced dosing for moderate impairment (Child-Pugh B); use with caution at a further-reduced dose (or less frequent dosing) for severe impairment (Child-Pugh C), only if the benefit is expected to outweigh the risk, with closer monitoring — see the professional information for the exact regimen. Renal impairment: no adjustment needed for mild-to-moderate impairment; use with caution in severe impairment (creatinine clearance <=30 mL/min) or end-stage renal disease, where data are limited. (2)
Monitoring
(2)Storage
Store Orkambi tablets and oral granules at controlled room temperature, 20-25C (68-77F), with brief excursions to 15-30C permitted, away from moisture and heat, and out of reach of children.
Professional information
Clinician-facing context only. This section does not replace current product labeling.
General
Indication: Cystic fibrosis in patients aged 1 year and older who are homozygous for the F508del mutation in the CFTR gene
Dose summary: Ages 12+ and adults: lumacaftor 200 mg/ivacaftor 125 mg (2 tablets) every 12 hours with fat-containing food. Ages 6-11: lumacaftor 100 mg/ivacaftor 125 mg per tablet, 2 tablets every 12 hours. Ages 2-5 and 1-2: weight-based oral granule packets every 12 hours per the FDA label's age/weight dosing table. Hepatic impairment: dose reduced per Child-Pugh class (see organ_impairment). Strong CYP3A inhibitor co-administration: reduce dose for the first week of initiation.
Renal context: No dosage adjustment necessary for mild-to-moderate renal impairment. Use with caution in severe renal impairment (creatinine clearance <=30 mL/min) or end-stage renal disease; data in these patients are limited. (2)
Frequently asked questions
What is Lumacaftor/Ivacaftor (Orkambi) used for?
It is used to treat cystic fibrosis in patients aged 1 year and older who have two copies of the F508del mutation in the CFTR gene.
How does Orkambi work?
Orkambi combines two medicines: lumacaftor helps correct the misfolded CFTR protein, and ivacaftor improves its function at the cell surface, helping to reduce symptoms of cystic fibrosis.
What are the common side effects of Orkambi?
Common side effects include headache, nausea, diarrhea, upper respiratory tract infection, and increased sputum.
Can Orkambi be taken with other cystic fibrosis medications?
Yes, Orkambi is often used alongside other CF treatments, but always consult your healthcare provider for guidance.
What should I do if I miss a dose?
Take the missed dose within 6 hours if possible. If more than 6 hours have passed, skip the dose and continue your regular schedule. Do not double up.
Are there any foods or drugs to avoid while taking Orkambi?
Avoid grapefruit, Seville oranges, and strong CYP3A inducers like rifampin, as these can affect drug levels.
Is Orkambi safe during pregnancy or breastfeeding?
There is limited data on safety in pregnancy and breastfeeding. Discuss potential risks and benefits with your healthcare provider.
What happens if I take too much Lumacaftor/Ivacaftor (Orkambi)?
Taking too much Lumacaftor/Ivacaftor (Orkambi) can lead to an exacerbation of side effects, such as respiratory issues, liver problems, or gastrointestinal upset. If you suspect an overdose, it is crucial to seek emergency medical attention immediately. Contact your local poison control center or emergency services for guidance and prompt treatment.
Can I drink alcohol while taking Lumacaftor/Ivacaftor (Orkambi)?
It is generally advisable to limit or avoid alcohol consumption while taking Lumacaftor/Ivacaftor (Orkambi). Alcohol can potentially increase the risk of liver-related side effects, which are already a concern with this medication. Discuss your alcohol intake with your healthcare provider to understand the specific risks and recommendations for your individual health situation.
Is Lumacaftor/Ivacaftor (Orkambi) safe during pregnancy?
The safety of Lumacaftor/Ivacaftor (Orkambi) during pregnancy has not been definitively established. It is categorized as 'Consult doctor' regarding pregnancy. If you are pregnant, planning to become pregnant, or breastfeeding, it is essential to consult your healthcare provider. They can assess the potential risks and benefits and help you make an informed decision about your treatment.
What is the mechanism of action of lumacaftor and ivacaftor?
Lumacaftor is a CFTR corrector that improves the conformational stability of the F508del-CFTR protein, increasing its processing and trafficking to the cell surface. Ivacaftor is a CFTR potentiator that increases the channel open probability (gating) of the CFTR protein at the cell surface. Together, they enhance the quantity and function of CFTR protein in patients homozygous for the F508del mutation, improving chloride transport and reducing disease manifestations.
What is the generic name of Lumacaftor And Ivacaftor?
The generic name is lumacaftor and ivacaftor. It is sold under brand names including Ivafect, Lumivac, Orkambi.
What class of drug is lumacaftor and ivacaftor?
Lumacaftor And Ivacaftor is classed as: Respiratory System.
References
- Lumacaftor/Ivacaftor (Orkambi) — clinician-authored medicine recordMedGuideGlobal · Global · accessed 2026-08-06 · source 1
- ORKAMBI (lumacaftor and ivacaftor) tablets and oral granules — Full Prescribing InformationU.S. National Library of Medicine DailyMed · United States · accessed 2026-08-06 · source 2
- ORKAMBI (LUMACAFTOR AND IVACAFTOR) TABLET, FILM COATED ORKAMBI (LUMACAFTOR AND IVACAFTOR) GRANULE [VERTEX PHARMACEUTICALS INCORPORATED] — US prescribing information (DailyMed SPL)U.S. National Library of Medicine (DailyMed) · US · accessed 2026-08-14 · source 3
- Lumacaftor and Ivacaftor — patient drug information (MedlinePlus)U.S. National Library of Medicine (MedlinePlus) · US · accessed 2026-08-14 · source 4
- ORKAMBI — FDA approval record (Drugs@FDA NDA211358)U.S. Food and Drug Administration · US · accessed 2026-08-14 · source 5
- lumacaftor and ivacaftor — RxNorm normalized drug concept (RXCUI 1655927)U.S. National Library of Medicine (RxNorm) · US · accessed 2026-08-14 · source 6
Brand names by country
Single-ingredient lumacaftor and ivacaftor names are shown separately by country. Product availability, strengths, and supply rules can change; combination products are not included here.
Priority countries
United States
- ORKAMBI