Ivacaftor (Oral)

Ivacaftor treats individuals with cystic fibrosis and attacks the root causes of the issue rather than the outward symptoms, like most cystic fibrosis medications.


Ivacaftor is a prescription medication that treats cystic fibrosis (CF) for patients who are over age two. It is unique because ivacaftor remedies the cause of the disease rather than only the symptoms on the surface. This medication is only used for individuals with specific CF transmembrane regulator mutation, these mutations are: S549R, S549N, S1255P, S1251N, R117H, G551S, G178R, G1349D, G1244E, G551D. Approximately 4 to 5% of patients with cystic fibrosis can be treated with ivacaftor.

Your physician will test for the mutation prior to you having the medication prescribed. Ivacaftor is a CFTR potentiator, which moves more chloride into body cells.

This medication can only be obtained with a valid prescription from your physician. It can only be obtained in tablet form. It is currently being manufactured under the US brand name Kalydeco.

The Cystic Fibrosis Foundation and Vertex Pharmaceuticals initially developed ivacaftor. When the FDA approved ivacaftor in 2012, it was described as wonderful and the most important new medication of the year. Ivacaftor is extremely expensive, as the total cost per year is over $300,000 USD.

Condition treated

  • Cystic fibrosis

Type of medicine

  • CFTR activator

Side Effects

In addition to positive effects medications can provide, prescription drugs can also trigger side effects that may be undesirable. While it is not typical for every one of the following side effects to take place, they could require medical care if they do occur.

Consult with your medical care professional right away if you experience any of the following side effects:

More Common Side Effects (Medical Care Required)

  • Voice loss
  • Unusual weakness or tiredness
  • Trouble with or difficulty breathing
  • Sore throat
  • Sneezing
  • Runny nose
  • Nasal congestion
  • Headache
  • Fever
  • Ear congestion
  • Chills
  • Chest pains
  • Body pains or aches

Less Common Side Effects (Medical Care Required)

  • Yellow skin or eyes
  • Vomiting and nausea
  • Stools that are light in color
  • Stomach pain
  • Fever (chills may or may not occur)
  • Dark urine
  • Appetite loss

Additional side effects could take place but these typically do not require medical care. These specific side effects may disappear on their own throughout the course of treatment as your body becomes more adjusted to ivacaftor. In addition, your medical care professional can likely inform you of additional methods of preventing or reducing certain side effects. Consult with your doctor if the side effects become prolonged, troublesome, or simply if you have questions regarding them.

More Common Side Effects (Medical Care Not Required)

  • Skin blemishes
  • Rash
  • Pimples
  • Muscle stiffness or pain
  • Muscle aches
  • Moving difficulty
  • Joint pain
  • Diarrhea

Side effects not included here could also take place for certain individuals. If other side effects are observed, consult with your medical care professional right away.

Contact your physician for healthcare advice regarding side effects. The FDA also accepts reports of side effects, and they can be reached by calling 1-800-FDA-1088.


Always take this medication exactly as your physician has instructed. Never take an amount of this medication that is greater than what was prescribed. Do not take a dose of this medication more frequently than instructed. Do not take this medication for a longer duration been ordered by your physician. This could raise the likelihood of certain side effects taking place.

This medication is it distributed with a patient informational pamphlet. Always follow and carefully read the directions in the overview. Consult with your physician if you have questions regarding dosage.

Take this medication along with foods that contain fat like cheese pizza, eggs, butter, whole milk products, hold dairy products such as yogurt and cheese, or peanut butter.

For oral use, take note of the following

  • Gently shake the package prior to opening it. Proceed to open one package and empty the contents into a teaspoon of the liquid (such as yogurt, milk, water, juice, puréed vegetables or fruit, or applesauce).
  • Mix the medication into the liquid or soft food completely. After thoroughly mixed together, be sure the patient takes this medication within a period of one hour. Ensure the entire amount of medication is taken.
  • It is important to be aware that grapefruit juice or grapefruit by itself should not be consumed while taking this medication. Grapefruit has a possibility of interacting with this medication by allowing there to be too much medication within the bloodstream.
  • Seville oranges (oranges that are sour or bitter) and their juice should be avoided while on this medication. These fruits also cause the medication level in the blood to exceed the recommended amount.

Different patients will be prescribed a unique dose of this medication. Always follow the instructions given by your physician or on the label of the medication container. The information included here will summarize only typical doses of this medication. If you were given a dose that differs from the information here, do not adjust your dose unless order to buy your physician.

The quantity of medications you are prescribed will depend on how strong the medication is. In addition, the number of doses you are prescribed to take per day can impact how much medication you are prescribed to take in a single dose. The allotted time between each dose and the total duration for which you take the medication will also impact your dose.


Oral form


For Treatment of Cystic Fibrosis:

  • Adults and kids over age 6: Take 150 mg (milligrams) once for each 12-hour period, along with food that contains fat.
  • Children between the ages of 2 and 5 who weigh at least 14 kg (kilograms): Physician will need to calculate individual dosage information due to the various body needs of patients. However, the typical dose is one package (75 MG) for each period of 12 hours, and the medication needs to be taken how are with a food that contains fat.
  • Children between the ages of 2 and 5 who weigh between 0 and 14 kg: physician will prescribe a unique dose according to patient's weight. Usually, children who are very light should not take more then one package (50 milligrams) for each period of 12 hours along with a food that contains fat.
  • Children under age 2 should not take this medication.

Missed Dose

If a dose of this medication is skipped accidentally, it should be taken shortly after the patient remembers it was missed. Although, if it is nearer to the scheduled next dose in your dosing schedule (less than a period of six hours away), the next dose may be taken at the normal time and the original dosing schedule may be resumed. Never take a double dose of this medication.


Drug Interactions

Some prescription drugs should never be taken simultaneously, however in certain circumstances two medications can be combined, despite the possibility of interactions taking place. Under these circumstances, your physician may be inclined to adjust your dose, or they may choose to take a additional types of precautions while on this medication, it is essential that your physician is aware if you are taking any of the prescription medications included below. The following medications could have a potentially significant interaction with ivacaftor, and extra caution should be taken. The following list is not completely all-inclusive.

It is not typically recommended to combine ivacaftor with any of the medications below, however certain unique circumstances may require the combination. If ivacaftor is prescribed with any of the following medications, your physician may choose to adjust your dose or frequency for either or both medications.

  • Voriconazole
  • Verapamil
  • Topotecan
  • Telithromycin
  • Telaprevir
  • St John's Wort
  • Saquinavir
  • Ritonavir
  • Rifampin
  • Posaconazole
  • Phenytoin
  • Nelfinavir
  • Nefazodone
  • Morphine Sulfate Liposome
  • Morphine
  • Lopinavir
  • Ketoconazole
  • Itraconazole
  • Indinavir
  • Imatinib
  • Idelalisib
  • Fosphenytoin
  • Fosaprepitant
  • Fluconazole
  • Erythromycin
  • Eliglustat
  • Dronedarone
  • Diltiazem
  • Crizotinib
  • Conivaptan
  • Cobicistat
  • Clarithromycin
  • Carbamazepine
  • Boceprevir
  • Atazanavir
  • Aprepitant

Other Interactions

Some medications should not be taken near mealtime because of the likelihood of interactions taking place. Avoid grapefruit and oranges while taking ivacaftor. The use of tobacco and alcohol with specific medications could also trigger onset of interactions.

Medical Interactions

Patients with other medical issues cut experience usefulness in the effectiveness of ivacaftor. Be sure to inform your physician if you have other kinds of medical issues, specifically:

  • Liver issues for liver disease (severe or moderate) patients should take caution, side effects could be heightened due to slower extraction of the medication from the body
  • Kidney issues or kidney disease (severe)
  • Cataracts'patients should take caution, conditions could be made worse


It is essential that your physicians monitor your progress regularly to ensure that this medication is as effective as it should be. The patient may need to have regular blood tests conducted in order to monitor for undesired side effects.

Consult with your physician immediately if you or the patient has tenderness for pain in there upper stomach area, darkened urine, nausea, yellow skin or eyes, pale stools, appetite loss, or unusual weakness or tiredness. These could be sign of a severe liver issue.

This medication could cause you to be more dizzy then usual. Do not operate machinery, drive, or perform any other similar activity that could be highly dangerous for someone who is not completely alert.

Consult with your physician right away if you experience reading difficulties, blurred vision, or a vision change after or while you are taking ivacaftor. Your general care physician may refer you to a specialist such as an (eye doctor) ophthalmologist if these conditions take place.

Other medications should not be taken with ivacaftor if your physician has not previously approved it. This refers to nonprescription and prescription medications, as well as vitamins or herbal supplements (Such as St. John's Wort).

When choosing whether or not a medication is right for you and your unique medical needs, you must compare whether or not the likely benefits of the medication Will outweigh any potential risks that the medication may have. You must make this decision carefully with doctor. Consider the following aspects when deciding whether or not ivacaftor is right for you.


Patients with allergies could encounter issues when taking ivacaftor. Be sure to inform your physician ahead of time if you have allergic or unusual reactions to any this or other medications. Also inform your doctor if you have allergies of any kind including to animals, preservatives, dies, or foods. For products that are non-prescription, always read the entire label on the medication bottle or ingredients on the package carefully.

Specific Demographic Use


Current research has not discovered any issues that are specific to the pediatric population that could potentially inhibit the effectiveness of ivacaftor for children. However, children under age 2 should not take this medication, as it has not yet been proven both safe and effective for them.


Research on the elderly population and the use of ivacaftor has not identified any issues for geriatric patients. Consult with your physician if you are a geriatric patient who is interested in taking this medication.


Research performed on animals has not outlined any potential harm for the unborn baby but similar research has not been performed in women; or, research in pregnant women has identifies no risk to the baby, but animal research has revealed a negative effect.


Current research has not identified any issues for women who breastfeed while taking ivacaftor. Women should compare the potential dangers against possible risks prior to taking ivacaftor while breastfeeding.


Ivacaftor must be kept in a sealed medication container. Always store this drug far from excessively freezing or hot temperatures. Also avoid storing this medication in an area that is exposed to a lot of direct or natural light.

Never keep ivacaftor in an area that is easily accessible for children.

Medication that is expired or no longer required for the original medical condition should not be kept. Consult with your doctor to find the best method of disposing any unused medication.


Ivacaftor is a medication for patients with cystic fibrosis over the age of two. This is a special medication because ivacaftor actually treats the underlying causes of the disease, instead of the surface problems. Only patients with certain CF transmembrane regulator mutations can be treated with ivacaftor, which is approximately 4 to 5% of patients.