Lumacaftor and Ivacaftor (Oral)

Lumacaftor and ivacaftor is a combination drug that is used to treat patients over six years of age who suffer from cystic fibrosis.

Overview

In the US, lumacaftor and ivacaftor in combination is known by the brand name, Orkambi. It is a prescription only drug that comes in the form of tablets.

Lumacaftor and ivacaftor is a combination of two different medicines that are used to treat cystic fibrosis in people who are aged over six years. The medication is only suitable for use in patients whose genetic make-up has two copies of the F508del mutation in their CFTR gene (transmembrane conductance regulator).

Before being prescribed lumacaftor and ivacaftor, your health care professional will carry out tests to see whether you have this genetic mutation to ensure that it is suitable.

Conditions treated

Type of medicine

  • Cystic fibrosis transmembrane conductance regulator (CFTR) correctors
  • Cystic fibrosis transmembrane conductance regulator (CFTR) potentiators

Side effects

In addition to its desired effects, lumacaftor and ivacaftor may cause a few unwanted side effects. Not everyone taking this medicine will notice any untoward effects, but if they do occur, you may need to seek further medical attention.

If you notice any of the effects listed below while taking this drug, check with your health care professional right away:

  • Yellow eyes or skin
  • Unusual tiredness
  • Tightness in the chest
  • Slow or rapid heartbeat
  • Pounding in the ears
  • Discomfort or pain in the upper right stomach area
  • Nervousness
  • Nausea and vomiting
  • Loss of appetite
  • Headaches
  • Dizziness
  • Difficult or labored breathing
  • Dark urine
  • Blurred vision

There are some side effects caused by lumacaftor and ivacaftor that will sort themselves out without the need for any further treatment as your body gets used to the drug. In addition, your treating physician may be able to recommend ways of preventing or reducing some of these effects. Ask your doctor if any of the effects listed below become persistent or are particularly troublesome:

  • Feelings of unusual weakness
  • Stuffy or runny nose
  • Cessation of menstrual bleeding
  • Sore throat
  • Sneezing
  • Rashes
  • Passing more gas than usual
  • Muscle aches
  • Loss of voice
  • Longer or heavier menstrual periods
  • Full feeling
  • Fever
  • Excess air or gas in the intestines or stomach
  • Ear congestion
  • Diarrhea
  • Cramps
  • Cough
  • Chills
  • Body aches or pains
  • Feeling bloated
  • Irregular, missed, or absent menstrual periods

There may be other side effects that affect some people who are taking this drug, which are not mentioned in this guide. If you experience anything else untoward, check with your treating physician right away.

Dosage

You must use lumacaftor and ivacaftor precisely as instructed by your health care professional. Do not increase your dose, use it more frequently, or take it for a longer time than your health care professional has told you to. Misusing this medicine can increase the likelihood of side effects.

Lumacaftor and ivacaftor will come with a medication guide. Be sure to read the guide thoroughly and follow the instructions. If you have any questions about using this drug, check with your health care professional.

You should try to take this medication with foods that contain fat, such as butter, cheese, avocado, eggs, nuts, whole milk dairy products (yogurt and whole milk), and peanut butter.

You must inform your health care professional if you do not take lumacaftor and ivacaftor for more than one week. It may be necessary for your doctor to adjust the dose of lumacaftor and ivacaftor or some of the other medication that you are taking.

The dose of lumacaftor and ivacaftor that you are prescribed will vary between patients. You must stick to your doctor's instructions or follow those on the product label. The information in this guide is based on the average recommended dosage for this drug. If your dose is not the same as this, do not change it unless your health care professional tells you to.

The dose of lumacaftor and ivacaftor that you take will depend on the potency of the tablets. In addition, the daily dose you take, the time you leave between doses, and the total duration of your course of treatment will depend on your health condition.

Tablets for the treatment of cystic fibrosis:

  • Adults and children aged over 12 years: Take two tablets at 12 hourly intervals with food that contains fat. Each tablet comprises 125 mg of ivacaftor and 200 mg of lumacaftor.
  • Children aged six to 11 years and older: Take two tablets at 12 hourly intervals with food that contains fat. Each tablet comprises 125 mg of ivacaftor and 100 mg of lumacaftor.
  • Children aged under six years: The use and dose rate of this medicine will be determined by your child's GP.

If you forget to take a dose of your medication, you should try to take it right away. If your next dose is almost due, leave out the one you missed and start again from your usual schedule. If your next scheduled dose is less than six hours away, take it at the usual time.

Do not take a double dose.

Interactions

Drug interactions

Some medicines must never be taken together as to do so could cause an interaction to take place. In some cases, your health care professional may decide to use both drugs together, even though an interaction may take place. When you are using lumacaftor and ivacaftor in combination, you must tell your health care professional if you are using any other medicines, especially those listed below.

It is not recommended that you use lumacaftor and ivacaftor with any of the following drugs, but this may be necessary in some circumstance. If you are prescribed both drugs together, your GP may adjust the dose or frequency of use of one or both of them:

  • Voriconazole
  • Verapamil
  • Vardenafil
  • Triazolam
  • Topotecan
  • Tolvaptan
  • Tipranavir
  • Temsirolimus
  • Telithromycin
  • Telaprevir
  • Tacrolimus
  • St John's Wort
  • Sirolimus
  • Simvastatin
  • Sildenafil
  • Saquinavir
  • Ritonavir
  • Rifampin
  • Regorafenib
  • Quinidine
  • Quetiapine
  • Posaconazole
  • Pimozide
  • Phenytoin
  • Oxycodone
  • Osimertinib
  • Norgestrel
  • Norgestimate
  • Norethindrone
  • Nelfinavir
  • Nefazodone
  • Morphine Sulfate Liposome
  • Morphine
  • Midazolam
  • Mestranol
  • Maraviroc
  • Lurasidone
  • Lovastatin
  • Lopinavir
  • Levonorgestrel
  • Ketoconazole
  • Ivabradine
  • Itraconazole
  • Isavuconazonium
  • Indinavir
  • Imatinib
  • Idelalisib
  • Gestodene
  • Fosphenytoin
  • Fosaprepitant
  • Fluticasone
  • Fluconazole
  • Fentanyl
  • Felodipine
  • Everolimus
  • Etonogestrel
  • Ethynodiol
  • Ethinyl Estradiol
  • Estradiol
  • Erythromycin
  • Erlotinib
  • Ergotamine
  • Eplerenone
  • Eliglustat
  • Eletriptan
  • Drospirenone
  • Dronedarone
  • Diltiazem
  • Dihydroergotamine
  • Dienogest
  • Desogestrel
  • Dasatinib
  • Darunavir
  • Darifenacin
  • Cyclosporine
  • Crizotinib
  • Conivaptan
  • Cobicistat
  • Clarithromycin
  • Carbamazepine
  • Buspirone
  • Budesonide
  • Boceprevir
  • Axitinib
  • Atazanavir
  • Aprepitant
  • Alfentanil

Other interactions

Some medicines must not be used when you are eating or eating certain foodstuffs, as this could cause interactions to occur. Using tobacco or alcohol with some drugs can also trigger interactions. Check with your health care professional before using tobacco, alcohol, or particular food groups with lumacaftor and ivacaftor.

Medical interactions

Some existing or historical medical problems can affect how this medication works. Be sure to tell your health care professional if you have a history of any other medical problems especially those mentioned below.

If you have a history of cataracts or organ transplantation, you should use lumacaftor and ivacaftor with caution as the drug could make these conditions worse.

Patients with severe or moderate liver or kidney disease should use lumacaftor and ivacaftor with caution, as the effects of the drug could be increased due to its slower removal from your body.

Warnings

Before you decide to use any form of medication, you should consider the risks of using it as well as the benefits. This decision should be taken following discussions with your health care professional. In the case of lumacaftor and ivacaftor in combination, consider the points mentioned below.

Be sure to tell your treating physician if you have ever noted an allergic reaction to lumacaftor and ivacaftor or any form of prescription or non-prescription drug. You should also mention any allergies that you have to food colors, preservatives, animal derivatives or specific food groups.

Although it is safe and effective to use lumacaftor and ivacaftor in children over six years of age, there have been no studies to show that this is the case in children younger than this. Discuss any concerns about treating your child with this medication with your child's health care professional.

There have been no specific studies into the effect of using lumacaftor and ivacaftor in elderly patients. However, geriatrics tend to be more likely to have age-related kidney and liver disease, meaning that the effects of the drug could be increased due to its slower removal from the body.

It is not known whether lumacaftor and ivacaftor passes into breast milk. If you are breastfeeding, you should discuss the risks to your nursing infant with your doctor or midwife and perhaps consider using an alternative feeding solution until you have finished your course of this drug.

You must attend your health care professional regularly for progress checks and blood test while you are using lumacaftor and ivacaftor. You may also be asked to monitor your blood pressure in your own home. If you notice any change in your blood pressure, check with your health care professional right away.

Patients should note that some hormone-based contraceptives, including birth control pills, implants, rings, and injections might not be effective while you are taking lumacaftor and ivacaftor in combination. To avoid becoming pregnant, you should use an alternative form of contraception such as a diaphragm, condoms, or contraceptive jelly or foam while you are using this medication.

You must not use any other form of medicine while you are taking lumacaftor and ivacaftor, unless you have discussed its use with your health care professional first. This includes over the counter preparations, herbal and vitamin supplements.

Storage

Your lumacaftor and ivacaftor tablets must be stored in a closed container and at room temperature. Do not expose the tablets to direct sunlight or sources of heat or moisture. Do not freeze the tablets.

Be careful that children and pets do not have access to your medicine. If a pet does eat any of your lumacaftor and ivacaftor tablets, seek emergency veterinary advice right away.

Do not retain any out-of-date or unwanted lumacaftor and ivacaftor tablets. Ask your treating physician or pharmacist for instructions on how to dispose of this medicine safely.

Summary

Lumacaftor and ivacaftor is a combination drug that is used to treat people who are aged over six years who suffer from cystic fibrosis. This medication is only suitable for use in people whose genetic make-up features two copies of the F508del mutation in their CFTR gene (transmembrane conductance regulator).

This medication interacts with a wide range of other drugs and can cause complications in certain existing health conditions. Be sure to discuss your medical history fully with your health care professional before you begin using this medicine.

You will need to attend your doctor for regular checks and blood tests while you are taking lumacaftor and ivacaftor. These visits are necessary to check that the drug is working properly and to address any side effects that it may be causing.

Patients should note that lumacaftor and ivacaftor can negate the effect of hormone-based contraceptives. To avoid becoming pregnant, use a barrier form of birth control while you are using this drug.