Ivacaftor / Lumacaftor Side Effects

Medically reviewed by Drugs.com. Last updated on Jul 15, 2024.

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

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:

More common side effects

• body aches or pain
• chest tightness
• chills
• cough
• difficult or labored breathing
• ear congestion
• fever
• headache
• loss of voice
• muscle aches
• sneezing
• sore throat
• stuffy or runny nose
• unusual tiredness or weakness
• upper abdominal or stomach pain

Incidence not known

• blurred vision
• dark urine
• difficulty swallowing
• discomfort in the upper right stomach area
• dizziness
• fast heartbeat
• general feeling of tiredness or weakness
• hives, itching, skin rash
• large, hive-like swelling on the face, eyelids, lips, tongue, throat, hands, legs, feet, or sex organs
• light-colored stools
• loss of appetite
• nausea
• nervousness
• pounding in the ears
• puffiness or swelling of the eyelids or around the eyes, face, lips, or tongue
• slow or fast heartbeat
• stomach pain, continuing
• vomiting
• yellow eyes or skin

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:

More common side effects

• absent, missed, or irregular menstrual periods
• bloated feeling
• cough producing mucus
• cramps
• diarrhea
• excess air or gas in the stomach or bowels
• feeling of fullness
• heavy bleeding
• increased sputum production
• longer or heavier menstrual periods
• loss of voice
• passing gas
• rash
• stopping of menstrual bleeding

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]

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