Orkambi (Lumacaftor And Ivacaftor)
Complete patient guide for uses, dosage, side effects, and safety information
Prescription RequiredQuick Facts
What Is Orkambi Used For?
Orkambi is a prescription medication specifically designed to treat cystic fibrosis (CF) in patients aged 1 year or older who have a specific genetic mutation called F508del.
Primary Uses
Orkambi treats cystic fibrosis in patients who are homozygous for the F508del mutation in the CFTR gene. This means both copies of the gene have this specific mutation.
How It Works
Cystic fibrosis is caused by genetic defects that limit the flow of chloride and water through cell membranes, making mucus thick and sticky. Orkambi contains two active ingredients: lumacaftor (a CFTR corrector) that increases the amount of working CFTR protein at the cell surface, and ivacaftor (a CFTR potentiator) that helps the CFTR protein function better by keeping chloride channels open more often. This combination allows more chloride ions to be transported out of cells, improving lung function and breathing.
Good to know: Orkambi is not addictive and helps improve breathing by enhancing lung function. You may notice you don’t get ill as often or find it easier to maintain a healthy weight.
How to Take Orkambi
Orkambi must be taken with fat-containing food to ensure proper absorption and should be used every day as prescribed by your doctor.
Dosage Instructions
Dosing varies by age and weight:
**Ages 1-2 years:** – 7-9 kg: One 75mg/94mg granule sachet every 12 hours – 9-14 kg: One 100mg/125mg granule sachet every 12 hours – ≥14 kg: One 150mg/188mg granule sachet every 12 hours
**Ages 2-5 years:** – <14 kg: One 100mg/125mg granule sachet every 12 hours - ≥14 kg: One 150mg/188mg granule sachet every 12 hours
**Ages 6-11 years:** – Two 100mg/125mg tablets every 12 hours (4 tablets daily)
**Ages 12+ years:** – Two 200mg/125mg tablets every 12 hours (4 tablets daily)
**For granules:** Mix entire sachet contents with 5mL of room temperature soft food or liquid (pureed fruits/vegetables, yogurt, water, milk, breast milk, formula, or juice). Give immediately or within 1 hour. Provide fat-containing meal or snack just before or after dosing.
**For tablets:** Take with fat-containing meal or snack. Examples include foods prepared with butter or oils, eggs, cheese, whole milk, meats, oily fish, avocados, nuts.
What If You Miss a Dose?
If less than 6 hours have passed since your missed dose, take it as soon as possible and return to your regular schedule. If more than 6 hours have passed, skip the missed dose and take your next dose at the regular time.
Important: Never take a double dose to make up for a missed dose, as this may increase the chance of unwanted side effects.
Available Tablet Strengths
Side Effects
Like all medications, this medicine can cause side effects. Most are mild and temporary, but some require medical attention.
Common Side Effects
These side effects occur relatively frequently and usually don’t require emergency care:
- Shortness of breath
- Diarrhea
- Nausea
- Chest tightness
- Sore throat
- Upper respiratory tract infection
- Gas
- Rash
- Stuffy or runny nose
- Vomiting
- Irregular or painful periods
- Increased blood pressure
- Cough with sputum (in children)
- Headache (in children)
- Stomach pain (in children)
- Increased sputum (in children)
Seek immediate medical attention if you experience:
- Liver problems: significant pain in upper right stomach area, yellowing of skin or eyes, loss of appetite, nausea, vomiting, dark urine, confusion
- Serious allergic reactions: rash or hives, chest or throat tightness, difficulty breathing, swelling of face/lips/tongue, difficulty swallowing, dizziness
Important Warnings
Before taking Orkambi, it’s crucial to discuss your medical history and current medications with your doctor to ensure safe use.
Who Should Not Take Orkambi
Do not use Orkambi if you are allergic to lumacaftor, ivacaftor, or any other ingredients in the medication. Do not give to children under 1 year of age as safety and effectiveness have not been established.
Medical Conditions to Discuss
Tell your doctor if you have liver or kidney disease. Worsening of liver function has been seen in patients with severe liver disease and can be serious or fatal. Your doctor will perform blood tests to check your liver function before starting treatment and regularly during treatment (every 3 months in the first year, then annually). If you have poor lung function, your doctor may monitor you more closely when starting Orkambi due to potential respiratory events like shortness of breath or chest tightness.
Pregnancy and Breastfeeding
Check with your doctor if you are pregnant or planning to become pregnant. Tell your doctor if you are breastfeeding or plan to breastfeed, as it’s unknown whether Orkambi passes into breast milk. Hormonal birth control (pills, injections, patches, implants) may not work effectively when taking Orkambi – discuss alternative contraception methods with your doctor.
Liver Function Monitoring: Regular blood tests are required to monitor liver function, especially in the first year of treatment. Contact your doctor immediately if you experience liver-related symptoms.
Drug Interactions
Many medications can interact with Orkambi, affecting how well it works or increasing side effects. Always tell your doctor about all medications, vitamins, and supplements you’re taking.
- Anti-allergic medicines: montelukast, fexofenadine
- Antifungal medicines: ketoconazole, itraconazole, posaconazole, voriconazole, fluconazole
- Antibiotics: telithromycin, clarithromycin, erythromycin, rifampicin, rifabutin, rifapentine
- Anticonvulsants: phenobarbital, carbamazepine, phenytoin
- Anti-inflammatory medicines: ibuprofen
- Herbal medicine: St. John’s wort
- Benzodiazepines: midazolam, triazolam
- Immunosuppressants: ciclosporin, everolimus, sirolimus, tacrolimus
- Cardiac glycosides: digoxin
- Anticoagulants: warfarin, dabigatran
- Hormonal contraceptives: oral, injectable, patches, implants
- Glucocorticoids: prednisone, methylprednisolone
- Antidepressants: citalopram, escitalopram, sertraline, bupropion
- Diabetes medications: repaglinide
- Acid reflux medications: omeprazole, esomeprazole, lansoprazole, ranitidine
Alcohol & Driving
Exercise caution when driving or operating machinery until you know how Orkambi affects you.
Alcohol
No specific alcohol warnings mentioned in the source material. Consult your doctor about alcohol consumption while taking Orkambi.
Driving and Operating Machinery
Be careful before driving or using machines or tools until you know how Orkambi affects you, as the medication may cause side effects that could impair your ability to perform these activities safely.
Storage
Store Orkambi below 30°C in a cool, dry place away from moisture, heat, and sunlight. Do not store in the bathroom, near a sink, in the car, or on window sills. Keep this medicine where young children cannot reach it. If your doctor tells you to stop using Orkambi or it expires, take it to any pharmacy for safe disposal.
Overdose Information
If you think you have taken too much Orkambi, seek urgent medical attention immediately. Contact the Poisons Information Centre (13 11 26), your doctor, or go to the nearest hospital emergency department, even if you feel fine or have no symptoms of poisoning.
Frequently Asked Questions
The source doesn’t specify exact timing, but Orkambi helps improve lung function and breathing over time. You may notice that you don’t get ill as often or find it easier to maintain a healthy weight as the medication works.
Yes, you must take Orkambi with fat-containing food. This is essential for proper absorption. Examples include foods prepared with butter or oils, eggs, cheese, whole milk, meats, oily fish, avocados, or nuts.
No, Orkambi is not addictive according to the medication information.
Do not stop using Orkambi or change the dose without first checking with your doctor. Your doctor will determine if your treatment should be stopped and will guide you through the process safely.
If less than 6 hours have passed since your missed dose, take it as soon as possible and return to your regular schedule. If more than 6 hours have passed, skip the missed dose and take your next dose at the regular time. Never double dose.
Yes, your doctor will perform blood tests to check your liver function before starting treatment and regularly during treatment – every 3 months in the first year, then once yearly thereafter.
Hormonal contraceptives (oral, injectable, patches, implants) may not work effectively when taking Orkambi. Discuss alternative contraception methods with your doctor if you’re using hormonal birth control.
Important Disclaimer
This information is intended for general educational purposes only and should not be considered medical advice. Always consult your healthcare provider or pharmacist before starting, stopping, or changing any medication.
If you think you may have a medical emergency, call your doctor or emergency services immediately.