Orkambi
Orkambi (lumacaftor / ivacaftor) is a combination medication used to treat cystic fibrosis (CF) in people aged 2 years and older. It's only for people who have two copies of the F508del mutation (change in DNA) in the CFTR (cystic fibrosis transmembrane conductance regulator) gene. This medication is typically only available at certain pharmacies.
What is Orkambi (lumacaftor / ivacaftor)?
What is Orkambi (lumacaftor / ivacaftor) used for?
Cystic fibrosis
How Orkambi (lumacaftor / ivacaftor) works
Orkambi (lumacaftor / ivacaftor) is a combination of two medications that work together to help improve cystic fibrosis and its related symptoms.
Ivacaftor is a CFTR potentiator. It works by keeping chloride "gates" on the CFTR protein open so that salt and water can move freely in the lungs. This helps prevent mucus buildup in the lungs.
Lumacaftor is a CFTR corrector. It works by correcting the shape of the CFTR protein, which is often misshaped in people with CF. Once the CFTR protein forms the correct 3-D shape, it's able to move to the surface of cells in the body and work properly.
Drug Facts
What are the side effects of Orkambi (lumacaftor / ivacaftor)?
Common Side Effects
- Shortness of breath and chest tightness (13%)
- Upper respiratory tract infections (e.g., common cold, sore throat, stuffy nose, runny nose; up to 13%)
- Nausea (13%)
- Diarrhea (12%)
- Tiredness (9%)
- Rash (7%)
- Gas (7%)
- Flu or flu-like symptoms (5%)
Other Side Effects
- Irregular periods (menses) or more menstrual bleeding
- Higher blood pressure
Serious Side Effects
- Liver damage: upper right stomach pain or discomfort, dark-colored urine, pale-colored stools, yellowing of the skin or eyes
Source: DailyMed
The following side effects have also been reported
Side effects that you should report to your care team as soon as possible:
- Allergic reactions—skin rash, itching, hives, swelling of the face, lips, tongue, or throat
- Change in vision
- Increase in blood pressure
- Liver injury—right upper belly pain, loss of appetite, nausea, light-colored stool, dark yellow or brown urine, yellowing skin or eyes, unusual weakness or fatigue
- Shortness of breath or chest pain
Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):
Pros and cons of Orkambi (lumacaftor / ivacaftor)
Pros
Available in an oral granule form which can be mixed in soft foods or liquids for children who cannot swallow tablets
Combination of two medications that work differently to treat CF
Dose can be adjusted for people with moderate-to-severe liver disease
Cons
Only works for people with two copies of the F508del mutation in their CFTR gene
Cannot be used in children younger than 2 years of age
Might not be safe for those who have had an organ transplant (hasn't been studied)
Pharmacist tips for Orkambi (lumacaftor / ivacaftor)
Keep in mind that taking Orkambi (lumacaftor / ivacaftor) every 12 hours is not the same as taking it twice a day. Set alarms or reminders for yourself to help remember to take it on time every 12 hours.
Always take Orkambi (lumacaftor / ivacaftor) with foods high in fat to help your body absorb the medication better.
Avoid foods or drinks with grapefruit, because these might raise the amount of Orkambi (lumacaftor / ivacaftor) in your body.
If you're taking the Orkambi (lumacaftor / ivacaftor) oral granules, pour and mix all of the granules into 1 teaspoon (about 5 mL) of soft food or liquid that's at or below room temperature. Some examples of soft foods include puréed fruits, yogurt, pudding, or juice. Make sure your child takes the mixture within an hour of mixing it, right before or right after eating a high-fat meal.
Talk to your provider before starting, changing, or stopping any medications while taking Orkambi (lumacaftor / ivacaftor), including prescription and non-prescription medications, vitamins, and supplements. Orkambi (lumacaftor / ivacaftor) can interact with certain medications, so it's important that your provider knows about all the medications you take or plan to take to make sure they're safe for you.
Frequently asked questions about Orkambi (lumacaftor / ivacaftor)
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What are the risks and warnings for Orkambi (lumacaftor / ivacaftor)?
Orkambi (lumacaftor / ivacaftor) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.
Liver damage, including high liver test results
Risk factors: History of liver problems
Orkambi (lumacaftor / ivacaftor) can sometimes cause abnormally high liver test results. Your provider will check your liver before you start Orkambi (lumacaftor / ivacaftor), then every 3 months for the next year, and then every year after that. If you have a history of liver problems, your provider might check your liver labs more often.
If you have severe liver disease, discuss with your provider about the risks and benefits of taking Orkambi (lumacaftor / ivacaftor), as this medication might cause worsening liver health. This can lead to life-threatening liver failure.
While taking Orkambi (lumacaftor / ivacaftor), if you experience stomach pain, dark-colored urine, pale-colored stools, or yellowing of the skin or eyes, get medical help right away.
Respiratory (breathing) problems
Risk factors: Advanced lung disease (e.g., FEV1 less than 40)
In clinical studies, some people had chest discomfort and shortness of breath after taking Orkambi (lumacaftor / ivacaftor). These symptoms were bothersome enough for some people that they ended up stopping Orkambi (lumacaftor / ivacaftor) treatment altogether. Talk to your provider about your symptoms while taking this medication. They'll want to monitor you closely, especially if you have advanced lung disease (forced expiratory volume or FEV1 less than 40).
Higher blood pressure
Orkambi (lumacaftor / ivacaftor) can cause your blood pressure to rise. Make sure that you're checking your blood pressure regularly while taking this medication.
Interactions with other medications
Taking Orkambi (lumacaftor / ivacaftor) along with certain medications can affect the amount of Orkambi (lumacaftor / ivacaftor) in your body, how well it works, and the risk of side effects.
Some medications, like the antibiotic, rifampin, and a common supplement, St. John's wort, can lower the amount of Orkambi (lumacaftor / ivacaftor) in your body. This can make Orkambi (lumacaftor / ivacaftor) not work as well.
Other medications can raise the levels of Orkambi (lumacaftor / ivacaftor) in your body. You'll also want to avoid grapefruit and grapefruit juice while taking this medication, because grapefruit might cause you to have too much medication in your body as well. This can lead to a higher risk of side effects.
Orkambi (lumacaftor / ivacaftor) can also affect how other medications work, so tell your provider and pharmacist about all the medications and supplements you're taking before starting Orkambi (lumacaftor / ivacaftor). They will make sure all your medications are safe for you to take.
Cataracts
Risk factors: Taking steroid medications | Exposure to radiation
Orkambi (lumacaftor / ivacaftor) can cause cataracts in children. Your child's provider will likely give your child an eye exam before starting the medication and throughout treatment.
Orkambi (lumacaftor / ivacaftor) dosage forms
Typical dosing for Orkambi (lumacaftor / ivacaftor)
Adults and children age 12 years and older: The typical dose is 2 tablets by mouth every 12 hours with a high-fat meal. Each tablet for this age group contains 200 mg/125 mg (lumacaftor/ivacaftor).
Children age 6 to 11 years old: The typical dose is 2 tablets by mouth every 12 hours with a high-fat meal. Each tablet for this age group contains 100 mg/125 mg (lumacaftor/ivacaftor).
Children younger than 6 years of age: The dose and strength will depend on the child’s age and weight. The typical dose is 1 packet of oral granules by mouth every 12 hours with a high-fat meal. The oral granules come in two strengths: 100 mg/125 mg or 150 mg/188 mg (lumacaftor/ivacaftor).
Your dose might differ if you or your child has liver problems.
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