Ivacaftor / Lumacaftor Side Effects

Medically reviewed by Drugs.com. Last updated on Jun 22, 2024.

Applies to ivacaftor / lumacaftor: oral granule, oral tablet.

Precautions

It is very important that your doctor check your or your child's progress at regular visits to make sure that this medicine is working properly. Blood tests will be needed to check for unwanted effects. You may also need to monitor your blood pressure at home. If you notice any changes to your normal blood pressure, call your doctor right away.

Birth control pills and other forms of hormone-based contraceptives (eg, patches, rings, implants, and injections) may not work while you are using this medicine. To keep from getting pregnant, use another form of birth control (eg, condoms, diaphragm, or a contraceptive foam or jelly) while using Orkambi®.

Liver problems may be more likely to occur if you already have an existing liver problem (eg, cirrhosis). Check with your doctor right away if you or your child has pain or tenderness in the upper stomach, pale stools, dark urine, loss of appetite, nausea, vomiting, or yellow eyes or skin.

This medicine may cause serious allergic reactions, including anaphylaxis and angioedema, which can be life-threatening and require immediate medical attention. Check with your doctor right away if you have chest tightness, cough, difficulty swallowing, dizziness, fast heartbeat, hives, itching, skin rash, large, hive-like swelling on the face, eyelids, lips, tongue, throat, hands, legs, feet, or sex organs, puffiness or swelling of the eyelids or around the eyes, face, lips, or tongue, trouble breathing, or unusual tiredness or weakness.

Check with your doctor right away if you or your child has cough, trouble breathing, chest tightness, or any type of breathing problem when starting treatment with this medicine.

This medicine may cause eye problem, including cataracts. Check with your doctor right away if you or your child has blindness, blurred vision, or decreased vision.

Do not take other medicines unless they have been discussed with your doctor. This includes prescription or nonprescription (over-the-counter [OTC]) medicines and herbal (eg, St. John's wort) or vitamin supplements.

Serious side effects

Along with its needed effects, ivacaftor/lumacaftor may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

Check with your doctor immediately if any of the following side effects occur while taking ivacaftor / lumacaftor:

Other side effects

Some side effects of ivacaftor / lumacaftor may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects.

Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:

For healthcare professionals

Applies to ivacaftor / lumacaftor: oral granule for reconstitution, oral tablet.

General adverse events

The most frequently reported side effects were dyspnea, nasopharyngitis, nausea, diarrhea, upper respiratory tract infection, fatigue, respiration abnormal, blood creatine phosphokinase increased, rash, flatulence, rhinorrhea, and influenza.

Adverse effects in pediatric patients are generally consistent with those occurring in adult patients.^[Ref]

Cardiovascular

• Common (1% to 10%): Blood pressure increased/hypertension^[Ref]

Dermatologic

• Common (1% to 10%): Rash^[Ref]

Endocrine

Ivacaftor monotherapy only:

• Common (1% to 10%): Breast mass
• Uncommon (0.1% to 1%): Breast inflammation, gynecomastia, nipple disorder, nipple pain^[Ref]

Gastrointestinal

• Very common (10% or more): Nausea (up to 13%), diarrhea (up to 12%), upper abdominal pain
• Common (1% to 10%): Flatulence, oropharyngeal pain, vomiting^[Ref]

Genitourinary

• Common (1% to 10%): Menstruation abnormal/irregular, dysmenorrhea, metrorrhagia, amenorrhea, menorrhagia
• Uncommon (0.1% to 1%): Oligomenorrhea, polymenorrhea^[Ref]

Hepatic

• Common (1% to 10%): Increased AST increased, increased ALT
• Uncommon (0.1% to 1%): Hepatic encephalopathy, cholestatic hepatitis
• Frequency not reported: Total bilirubin increased
• Postmarketing reports: Liver failure leading to death, liver function decompensation^[Ref]

Hypersensitivity

• Postmarketing reports: Anaphylaxis, angioedema

Nervous system

• Very common (10% or more): Headache (up to 15.7%), dizziness^[Ref]

Ocular

• Frequency not reported: Non-congenital lens opacities^[Ref]

Other

• Common (1% to 10%): Blood creatine phosphokinase increased, fatigue, influenza

Ivacaftor monotherapy only:

• Very common (10% or more): Bacteria in sputum
• Common (1% to 10%): Ear discomfort, ear pain, tinnitus, tympanic membrane hyperemia, vestibular disorder, chest discomfort
• Uncommon (0.1% to 1%): Ear congestion^[Ref]

Respiratory

• Very common (10% or more): Infective pulmonary exacerbation of cystic fibrosis (37.5%), cough (30.5%), sputum increased (14.8 %), dyspnea (up to 14%), hemoptysis (13.8%), nasopharyngitis (up to 13%), upper respiratory tract infection (up to 10%)
• Common (1% to 10%): Rhinitis, pharyngeal erythema, sinus congestion, abnormal respiration, rhinorrhea, nasal congestion^[Ref]

See also:

Frequently asked questions

• How do you take Orkambi?
• What is the difference between Symdeko and Orkambi?

Further information

Ivacaftor/lumacaftor side effects can vary depending on the individual. Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.

Note: Medication side effects may be underreported. If you are experiencing side effects that are not listed, submit a report to the FDA by following this guide.

Medical Disclaimer