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Overview: Orkambi is used to treat cystic fibrosis in people who have certain genetic mutations. It contains two medicines that reduce symptoms of cystic fibrosis by thinning out the mucus that blocks the lungs and digestive system. Common side effects include breathing problems, nausea, diarrhea, tiredness, infection in the nose or throat, sore throat, runny or stuffy nose, or common cold symptoms. Before using Orkambi, make sure to tell your health care provider about any other medicines you are taking.

Uses

What is Orkambi used for?

Orkambi (lumacaftor/ivacaftor) is commonly used to treat an inherited disorder called cystic fibrosis, which is caused by many different types of genetic changes (mutations). Orkambi should only be used for cystic fibrosis that is caused by certain mutations.

Orkambi may also be used for other conditions as determined by your health care provider.

How does Orkambi work (mechanism of action)?

Cystic fibrosis is caused by changes (mutations) in a specific gene called the cystic fibrosis transmembrane conductance regulator (CFTR), which controls the movement of chloride ions and water through the body. In people with cystic fibrosis, chloride ions and water do not move well through the body, causing thick, sticky mucus that blocks the airways and the digestive system.

Orkambi contains two different medicines that work together to help CFTR move chloride ions and water through the body more easily. This thins out the mucus and reduces the symptoms of cystic fibrosis.

How is Orkambi supplied (dosage forms)?

Brands and other names

  • Orkambi

Dosage forms and strengths

  • 75 mg/94 mg oral granules
  • 100 mg/125 mg oral granules
  • 150 mg/188 mg oral granules
  • 100 mg/125 mg oral tablets
  • 200 mg/125 mg oral tablets

How should I store Orkambi?

Orkambi should be stored at room temperature, between 68 F to 77 F (20 C to 25 C). It can be exposed to temperatures between 59 F to 86 F (15 C to 30 C) for shorter periods of time, such as when transporting it. Store in a cool, dry place.

Side Effects

What are the most common side effects of Orkambi?

The most common side effects of Orkambi are listed below. Tell your health care provider if you have any of these side effects that bother you.

  • Breathing problems (see below)
  • Infection in the nose or throat, sore throat, runny or stuffy nose, or common cold symptoms
  • Nausea
  • Diarrhea
  • Tiredness
  • Rash
  • Gas
  • Influenza (flu)

There may be other side effects of Orkambi that are not listed here. Contact your health care provider if you think you are having a side effect of a medicine. In the U.S., you can report side effects to the FDA at www.fda.gov/medwatch or by calling 800-FDA-1088. In Canada, you can report side effects to Health Canada at www.health.gc.ca/medeffect or by calling 866-234-2345.

What are the serious side effects of Orkambi?

While less common, the most serious side effects of Orkambi are described below, along with what to do if they happen.

Severe Allergic Reactions. Orkambi may cause allergic reactions, which can be serious. Stop taking Orkambi and get help right away if you have any of the following symptoms of a serious allergic reaction.

  • Breathing problems or wheezing
  • Racing heart
  • Fever or general ill feeling
  • Swollen lymph nodes
  • Swelling of the face, lips, mouth, tongue, or throat
  • Trouble swallowing or throat tightness
  • Itching, skin rash, or pale red bumps on the skin called hives
  • Nausea or vomiting
  • Dizziness, feeling lightheaded, or fainting
  • Stomach cramps
  • Joint pain

Liver Damage. Liver damage, also called hepatotoxicity, can happen when taking Orkambi. Keep all appointments for blood work so that your health care provider can monitor your liver function. Call your health care provider right away if you have any of the following symptoms of liver damage.

  • Nausea or vomiting
  • Stomach or belly pain
  • Fever
  • Weakness or unusual tiredness
  • Itching
  • Loss of appetite
  • Light-colored poop
  • Dark-colored urine (pee)
  • Your skin or the whites of your eyes turning yellowish in color (also called jaundice)

Breathing Problems. Some people who take Orkambi may develop new breathing problems, such as chest pain, increased shortness of breath, or changes in breathing patterns. This may cause serious problems in people who already had severe lung disease before starting Orkambi. Call your health care provider right away if you notice any new breathing problems or chest pain.

Increased Blood Pressure (Hypertension). Orkambi may increase your blood pressure or make it worse if you have a history of high blood pressure. Call your health care provider if your blood pressure increases while taking this medicine. If you measure your blood pressure and the top number (systolic) is 180 mm Hg or higher or the bottom number (diastolic) is 120 mm Hg or higher, get medical attention right away. 

Cataracts. Some children have developed changes in the lenses of their eyes (cataracts) while taking Orkambi. It is unclear if Orkambi increases the risk of cataracts. You may need to have your eyes examined while you are taking this medicine. Contact your health care provider if you develop any of the following symptoms of cataracts.

  • Blurry, foggy, or cloudy vision
  • Changes in the way you see color
  • Seeing double

Warnings & Precautions

Who should not use Orkambi?

Allergies to Ingredients. People who are allergic to any of the following should not take Orkambi.

  • Ivacaftor
  • Lumacaftor
  • Orkambi
  • Any of the ingredients in the specific product dispensed

Your pharmacist can tell you all of the ingredients in Orkambi.

What should I know about Orkambi before using it?

Do not take Orkambi unless it has been prescribed to you by a health care provider. Take it as prescribed.

Do not share Orkambi with other people, even if they have the same condition as you. It may harm them.

Keep Orkambi out of the reach of children.

Orkambi can affect your alertness or coordination. Do not drive or do other activities that require alertness or coordination until you know how Orkambi affects you.

Orkambi oral granules should be mixed with a teaspoon of room temperature or cooler liquid or soft food. Once it is mixed, the dose should be taken within one hour. Examples of soft foods include pureed fruits or vegetables, yogurt, or applesauce. Examples of liquids include milk, infant formula, juice, or water. 

Orkambi may reduce the effects of certain types of birth control. If you use a birth control pill, patch, vaginal ring, implant, injection, or intrauterine device (IUD), you may need to use a second form of birth control while taking Orkambi. Examples of other forms of birth control include condoms, a diaphragm, or spermicide. Talk to your health care provider about your birth control methods and options.

What should I tell my health care provider before using Orkambi?

Tell your health care provider about all of your health conditions and any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using. This will help them determine if Orkambi is right for you.

In particular, make sure that you discuss any of the following.

Current and Past Health Conditions. Tell your health care provider if you have any of the following.

  • A transplanted organ
  • Kidney problems
  • Liver problems
  • Lung and breathing problems
  • Using a birth control pill, patch, vaginal ring, implant, injection, or intrauterine device (IUD)

Other Medicines and Supplements. Orkambi may interact with other medicines and supplements. Before taking Orkambi, tell your health care provider about any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using. See the Interactions section for more details.

Pregnancy. It is not known if or how Orkambi could affect pregnancy or harm an unborn baby. Tell your health care provider if you are or plan to become pregnant. Your health care provider will advise you if you should take Orkambi while you are pregnant or trying to get pregnant.

Breastfeeding. It is not known if Orkambi passes into breast milk. Tell your health care provider if you are breastfeeding or plan to breastfeed. Your health care provider will advise you if you should take Orkambi while breastfeeding.

How can I get more information about Orkambi?

For more information about Orkambi, you can visit the manufacturer’s website at www.orkambi.com or call them at 877-752-5933.

Interactions

Does Orkambi interact with foods or drinks?

Orkambi should be taken with a meal that contains a fatty food, such as whole milk, yogurt, cheese, eggs, butter, peanut butter, avocado, or cheese pizza. 

You should not eat grapefruit or drink grapefruit juice while taking Orkambi. Grapefruit or grapefruit juice can cause a dangerous increase in the blood levels of Orkambi.

It is unknown if drinking alcohol will affect Orkambi. The risk of dizziness may be increased if you drink alcohol while taking Orkambi.

Does Orkambi interact with other medicines (drug interactions)?

Always tell your health care provider about any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using.

In particular, make sure that you discuss if you are using any of the following before using Orkambi.

  • Clarithromycin or erythromycin, which are medicines used for bacterial infections
  • Ketoconazole, itraconazole (Sporanox, Tolsura), posaconazole (Noxafil), fluconazole (Diflucan), or voriconazole (Vfend), which is a medicine used for fungal infections
  • Rifabutin (Mycobutin), which is a medicine used to prevent Mycobacterium avium complex infection
  • Rifampin (Rifadin), which is a medicine commonly used to treat an infection called tuberculosis
  • St. John’s wort, which is a supplement that may be used to treat depressionA benzodiazepine, such as triazolam (Halcion) or midazolam (Versed), which is a medicine used for anxiety, muscle spasms, or seizures
  • A birth control pill, patch, vaginal ring, implant, injection, or intrauterine device (IUD)
  • A medicine to suppress your immune system, such as cyclosporine (Neoral, Sandimmune), everolimus, sirolimus, or tacrolimus
  • Any medicine for seizures, such as carbamazepine, phenytoin, or phenobarbital

Many other medicines may alter the blood levels of Orkambi, and vice versa. Tell your health care provider about all medicines that you take or have recently taken.

This may not be a complete list of medicines that can interact with Orkambi. Always check with your health care provider.

Does interact with other drugs you are taking?

Enter your medication into the WebMD interaction checker

Overdose/Missed Dose

What should I do if I accidentally use too much Orkambi?

If you or someone else has used too much Orkambi, get medical help right away, call 911, or contact a Poison Control center at 800-222-1222.

What should I do if I miss a dose of Orkambi?

If you miss a dose and it has been less than six hours since the missed dose, take it as soon as you remember. If it has been more than six hours since the missed dose, skip the missed dose and only take the next dose at the regularly scheduled time. Do not take double or extra doses.