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Why is this medication prescribed?

Ivacaftor is a medication that is prescribed to treat cystic fibrosis (CF) in patients who have specific gene mutations. CF is a genetic disorder that affects the production and function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, leading to the buildup of thick, sticky mucus in various organs, particularly the lungs and digestive system.

Ivacaftor is specifically indicated for CF patients who have a specific mutation in their CFTR gene known as a gating mutation. This mutation affects the function of the CFTR protein by reducing the ability of the protein channels to open and allow the movement of ions across cell membranes. Ivacaftor works by helping to open these channels, which improves the transport of ions, such as chloride, across cell membranes and helps restore more normal function to the CFTR protein.

How should this medicine be used?

Ivacaftor is a prescription medication that is used to treat cystic fibrosis (CF) in patients who have specific gene mutations. The specific dosage and usage instructions for Ivacaftor should be provided by a healthcare professional, as they can vary depending on the patient’s age, weight, and individual circumstances. However, here is a general guideline on how Ivacaftor is commonly used:

  • Dosage: The recommended dosage of Ivacaftor may vary depending on the specific mutation and the patient’s age. The healthcare provider will determine the appropriate dose for each individual.
  • Administration: Ivacaftor is usually taken orally as tablets or oral granules. The medication should be swallowed whole with water and can be taken with or without food. However, it is generally recommended to take Ivacaftor with a fat-containing meal or snack or with a nutritional supplement that contains fat. This is because the presence of fat can enhance the absorption of the medication.
  • Timing: Ivacaftor is typically taken once or twice daily, as prescribed by the healthcare provider. It is important to follow the recommended dosing schedule and not to skip or double doses unless specifically instructed by a healthcare professional.
  • Compliance: It is important to take Ivacaftor regularly and consistently to achieve the desired therapeutic effects. Missing doses or discontinuing the medication without medical guidance may reduce its effectiveness.

Other uses for this medicine

Ivacaftor is primarily used for the treatment of cystic fibrosis (CF) in patients who have specific gene mutations. It is not commonly prescribed for other conditions or purposes. Its effectiveness and safety for other uses have not been extensively studied or established.

What special precautions should I follow?

When using Ivacaftor, it is important to follow certain precautions:

  • Inform your healthcare provider: Before starting Ivacaftor, inform your healthcare provider about any other medications you are taking, including prescription drugs, over-the-counter medications, and herbal supplements. Some medications may interact with Ivacaftor and affect its effectiveness or increase the risk of side effects.
  • Pregnancy and breastfeeding: If you are pregnant, planning to become pregnant, or breastfeeding, it is crucial to discuss the potential risks and benefits of using Ivacaftor with your healthcare provider. They can provide guidance based on your specific situation.
  • Liver function: Ivacaftor may affect liver function, and periodic monitoring of liver enzymes may be required. If you experience symptoms such as abdominal pain, jaundice (yellowing of the skin or eyes), dark urine, or unexplained fatigue, contact your healthcare provider immediately.
  • Eye disorders: Some patients taking Ivacaftor have reported the development of cataracts or lens opacities. If you notice any changes in your vision or experience eye-related symptoms, consult your healthcare provider.

What special dietary instructions should I follow?

For Ivacaftor, there are no specific dietary restrictions that need to be followed. However, it’s generally recommended to take Ivacaftor with a fat-containing meal or snack, as this helps with the absorption of the medication. This means that you can take Ivacaftor with or without food, but if you choose to take it without food, it may reduce its effectiveness. It’s important to follow the instructions provided by your healthcare provider or the medication’s label regarding the timing of doses with respect to meals.

What should I do if I forget a dose?

If you forget to take a dose of any medication, including Ivacaftor, it is generally recommended to follow these steps:

  • Check the medication instructions: Refer to the prescribing information or the patient information leaflet that comes with Ivacaftor for specific guidance on missed doses. It may provide instructions on what to do in case of a missed dose.
  • Contact your healthcare provider: If you’re unsure about what to do or need further guidance, it’s best to reach out to your healthcare provider or pharmacist. They can provide you with specific advice based on your individual circumstances and medical history.
  • Follow the instructions given: Your healthcare provider may advise you to take the missed dose as soon as you remember, or they may suggest skipping the missed dose and continuing with the regular dosing schedule. It’s important not to take a double dose to make up for the missed one unless explicitly instructed to do so.

What side effects can this medication cause?

Regarding the potential side effects of Ivacaftor, it’s essential to note that individual experiences can vary. Here are some commonly reported side effects associated with Ivacaftor:

  • Gastrointestinal issues: These may include stomach pain, diarrhea, nausea, vomiting, and increased liver enzymes.
  • Respiratory symptoms: Some individuals may experience respiratory issues such as upper respiratory tract infections, nasal congestion, or sinusitis.
  • Headache: Headaches have been reported as a potential side effect of Ivacaftor.
  • Dizziness or lightheadedness: Some individuals may experience these symptoms while taking the medication.
  • Skin rash: In rare cases, Ivacaftor has been associated with skin reactions such as rashes or itching.

It’s important to remember that this is not an exhaustive list, and other side effects may occur. If you experience any concerning or persistent side effects while taking Ivacaftor, it’s crucial to inform your healthcare provider promptly. They can evaluate your symptoms and provide appropriate guidance or adjustments to your treatment if necessary.

What should I know about storage and disposal of this medication?


  • Keep Ivacaftor in its original container or packaging, tightly closed.
  • Store Ivacaftor at room temperature, away from excessive heat, moisture, and light.
  • Do not freeze Ivacaftor.


  • If you no longer need Ivacaftor or it has expired, consult with a pharmacist or healthcare professional regarding proper disposal.
  • Do not dispose of Ivacaftor in household trash or flush it down the toilet, as this can potentially harm the environment. Follow local guidelines or drug take-back programs for safe disposal.

In case of emergency/overdose

  • In case of emergency, such as a severe allergic reaction or life-threatening symptoms, call your local emergency number immediately.
  • If you suspect an overdose of Ivacaftor, contact a poison control center or go to the nearest emergency room. It may be helpful to bring the medication container or packaging with you to provide information to healthcare professionals.

What other information should I know?

  • Ivacaftor is a prescription medication, so always take it exactly as prescribed by your healthcare provider.
  • Inform your doctor about any other medications, vitamins, or supplements you are taking, as they may interact with Ivacaftor.
  • Inform your doctor about any medical conditions you have, especially liver or kidney problems, as these may affect the use of Ivacaftor.
  • Regularly attend follow-up appointments with your doctor to monitor your progress and discuss any concerns or side effects.

Remember, the information provided here is a general overview, and it’s essential to consult your healthcare provider or pharmacist for specific instructions and guidance regarding the storage, disposal, and usage of Ivacaftor.

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