What is Kalydeco?

Ivacaftor is used to treat cystic fibrosis (CF) in patients 12 months of age and older. It is only used for patients who have the following mutations in the CF transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, R117H, S1251N, S1255P, S549N, or S549R mutations. Your doctor will use a test to check for the mutation before you receive the medicine. This medicine is a CFTR potentiator, and works by moving more chloride into the cells of your body.

This medicine is available only with your doctor's prescription.

Take this medicine exactly as directed by your doctor. Do not take more of it, do not take it more often, and do not take it for a longer time than your doctor ordered. To do so may increase the chance of side effects.

This medicine comes with a patient information insert. Read and follow the instructions carefully. Ask your doctor if you have any questions.

Take this medicine with fat-containing foods such as butter, cheese pizza, eggs, peanut butter, or whole milk dairy products (eg, whole milk, cheese, yogurt).

To use the oral granules:

• Shake the packet gently before opening it.
• Open one packet and pour the contents into 1 teaspoon of cold soft food or liquid (eg, applesauce, pureed fruits or vegetables, yogurt, water, milk, or juice).
• Mix the granules with the soft food or liquid.
• After mixing, give this medicine to your child within 1 hour. Make sure all medicine is taken.

Do not eat grapefruit or Seville oranges or drink grapefruit juice or Seville orange juice while you are using this medicine. These may cause the amount of medicine absorbed in the blood to be too high.

The dose of this medicine will be different for different patients. Follow your doctor's orders or the directions on the label. The following information includes only the average doses of this medicine. If your dose is different, do not change it unless your doctor tells you to do so.

The amount of medicine that you take depends on the strength of the medicine. Also, the number of doses you take each day, the time allowed between doses, and the length of time you take the medicine depend on the medical problem for which you are using the medicine.

• For oral dosage forms (granules or tablets):
  • For cystic fibrosis:
    • Adults and children 6 years of age and older—150 milligrams (mg) every 12 hours, taken with fat-containing food.
    • Children 12 months of age to 5 years of age and weighing 14 kilograms (kg) or more—Dose is based on body weight and must be determined by your doctor. The dose is usually one 75 mg packet every 12 hours, taken with fat-containing food.
    • Children 12 months of age to 5 years of age and weighing 7 to 13 kg—Dose is based on body weight and must be determined by your doctor. The dose is usually one 50 mg packet every 12 hours, taken with fat-containing food.
    • Children 12 months of age to 5 years of age and weighing less than 7 kg—Use and dose must be determined by your doctor.
    • Children younger than 12 months of age—Use is not recommended.

If you miss a dose of this medicine, take it as soon as possible. However, if it is almost time for your next dose, skip the missed dose and go back to your regular dosing schedule. Do not double doses.

If you miss a dose and it is 6 hours or less from your regular schedule, take it as soon as you can. If you miss a dose and it is more than 6 hours away from your next regular dose, skip the missed dose and go back to your regular schedule.

Store the medicine in a closed container at room temperature, away from heat, moisture, and direct light. Keep from freezing.

Keep out of the reach of children.

Do not keep outdated medicine or medicine no longer needed.

Ask your healthcare professional how you should dispose of any medicine you do not use.

In deciding to use a medicine, the risks of taking the medicine must be weighed against the good it will do. This is a decision you and your doctor will make. For this medicine, the following should be considered:

Tell your doctor if you have ever had any unusual or allergic reaction to this medicine or any other medicines. Also tell your health care professional if you have any other types of allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, read the label or package ingredients carefully.

Appropriate studies performed to date have not demonstrated pediatric-specific problems that would limit the usefulness of ivacaftor in children. However, safety and efficacy have not been established in children younger than 12 months of age.

Although appropriate studies on the relationship of age to the effects of ivacaftor have not been performed in the geriatric population, no geriatric-specific problems have been documented to date.

There are no adequate studies in women for determining infant risk when using this medication during breastfeeding. Weigh the potential benefits against the potential risks before taking this medication while breastfeeding.

Although certain medicines should not be used together at all, in other cases two different medicines may be used together even if an interaction might occur. In these cases, your doctor may want to change the dose, or other precautions may be necessary. When you are taking this medicine, it is especially important that your healthcare professional know if you are taking any of the medicines listed below. The following interactions have been selected on the basis of their potential significance and are not necessarily all-inclusive.

Using this medicine with any of the following medicines is usually not recommended, but may be required in some cases. If both medicines are prescribed together, your doctor may change the dose or how often you use one or both of the medicines.

• Alfentanil
• Aprepitant
• Atazanavir
• Benzhydrocodone
• Boceprevir
• Buprenorphine
• Carbamazepine
• Ciprofloxacin
• Clarithromycin
• Cobicistat
• Codeine
• Conivaptan
• Crizotinib
• Cyclosporine
• Dihydrocodeine
• Diltiazem
• Dronedarone
• Duvelisib
• Eliglustat
• Enzalutamide
• Erythromycin
• Fentanyl
• Fluconazole
• Fluvoxamine
• Fosnetupitant
• Fosphenytoin
• Hydrocodone
• Idelalisib
• Imatinib
• Indinavir
• Itraconazole
• Ivosidenib
• Ketoconazole
• Letermovir
• Lopinavir
• Meperidine
• Methadone
• Mitotane
• Morphine
• Morphine Sulfate Liposome
• Nefazodone
• Nelfinavir
• Netupitant
• Nilotinib
• Oxycodone
• Pentazocine
• Phenytoin
• Posaconazole
• Rifampin
• Ritonavir
• Saquinavir
• St John's Wort
• Sufentanil
• Tacrolimus
• Telaprevir
• Telithromycin
• Tramadol
• Verapamil
• Voriconazole

Certain medicines should not be used at or around the time of eating food or eating certain types of food since interactions may occur. Using alcohol or tobacco with certain medicines may also cause interactions to occur. The following interactions have been selected on the basis of their potential significance and are not necessarily all-inclusive.

Using this medicine with any of the following is usually not recommended, but may be unavoidable in some cases. If used together, your doctor may change the dose or how often you use this medicine, or give you special instructions about the use of food, alcohol, or tobacco.

• Grapefruit Juice

The presence of other medical problems may affect the use of this medicine. Make sure you tell your doctor if you have any other medical problems, especially:

• Cataracts—Use with caution. May make this condition worse.

• Kidney disease, severe (eg, end-stage kidney disease) or
• Liver disease, moderate or severe—Use with caution. The effects may be increased because of slower removal of the medicine from the body.

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.

Check with your doctor right away if you or your child have pain or tenderness in the upper stomach, pale stools, dark urine, loss of appetite, nausea, unusual tiredness or weakness, or yellow eyes or skin. These could be symptoms of a serious liver problem.

This medicine may make you dizzy. Do not drive or do anything else that could be dangerous until you know how this medicine affects you.

Check with your doctor immediately if blurred vision, difficulty in reading, or any other change in vision occurs during or after treatment. Your doctor may want your eyes be checked by an ophthalmologist (eye doctor).

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.