ivacaftor + lumacaftor at a glance
Medicine class
CFTR corrector plus CFTR potentiator
Licensed use
Cystic fibrosis in people with F508del mutations on both CFTR gene copies
Food
Take with a fat-containing meal or snack
Important checks
Liver function, blood pressure and eye examinations in children
What is ivacaftor + lumacaftor used for?
Licensed uses represented by these products:
Granules: cystic fibrosis from age 1 year in patients with F508del on both copies of the CFTR gene.
Tablets: cystic fibrosis from age 6 years in patients with F508del on both copies of the CFTR gene.
How does ivacaftor + lumacaftor work?
Lumacaftor helps the altered CFTR protein reach the cell surface. Ivacaftor helps channels already there open more effectively, increasing chloride movement.
ivacaftor + lumacaftor preparations and strengths
Sachet
Route: Oral
Strengths: 75 mg + 94 mg, 100 mg + 125 mg, 150 mg + 188 mg
Tablet
Route: Oral
Strengths: 100 mg + 125 mg, 200 mg + 125 mg
How to take ivacaftor + lumacaftor
Follow the specialist CF team's instructions and take with fat-containing food.
Swallow tablets whole; do not chew, break or dissolve them.
Mix the full contents of a granule sachet with one teaspoon of room-temperature or cooler soft food or liquid.
Consume the complete granule mixture within one hour. Suitable options include yoghurt, fruit purée, milk, breast milk, infant food, water or juice.
ivacaftor + lumacaftor side effects
Common or expected effects
- Breathlessness, increased cough or sputum, or nasal congestion
- Diarrhoea, nausea, vomiting, flatulence or abdominal pain
- Headache or dizziness
- Upper respiratory symptoms
- Rash
- Irregular, painful or unexpected menstrual bleeding
- Raised liver enzymes
Get urgent medical advice
- Severe or worsening breathlessness, chest tightness, wheezing or bronchospasm
- Signs of liver injury, including jaundice, dark urine, persistent vomiting or right-sided upper abdominal pain
- Confusion or marked drowsiness associated with worsening liver disease
ivacaftor + lumacaftor in pregnancy
Experience in pregnancy is limited. CFTR modulators may be continued when needed for maternal health, while product information advises avoiding treatment unless the mother's condition requires it. Discuss planning, continuing or stopping treatment with the specialist CF and maternity teams.
ivacaftor + lumacaftor while breastfeeding
It is unknown how much lumacaftor or ivacaftor enters human milk, and risk to a breastfed child cannot be excluded. The specialist team should balance the benefits of breastfeeding and continued treatment rather than either being stopped without review.
ivacaftor + lumacaftor interactions
Lumacaftor strongly induces CYP3A and can substantially alter other treatments. Check prescriptions, over-the-counter products and herbal remedies with the CF team or pharmacist.
Hormonal contraceptives
Pills, injections, patches and implants may become less reliable and should not be relied upon as effective contraception during treatment.
Rifampicin, rifabutin or rifapentine
These can lower ivacaftor exposure and reduce treatment effectiveness; combined use is generally not recommended.
Common questions about ivacaftor + lumacaftor
Answers are fully visible for fast scanning and source review.
Who can take ivacaftor + lumacaftor?
It is licensed for people with cystic fibrosis who have F508del on both copies of the CFTR gene. The licensed formulation depends on age and, for younger children, weight.
Does it cure cystic fibrosis?
No. It improves altered CFTR protein processing and function but does not remove the underlying gene variants or replace wider cystic-fibrosis care.
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.