What is a Patient Information Leaflet and why is it useful?

The Patient Information Leaflet (PIL) is the leaflet included in the pack with a medicine. It is written for patients and gives information about taking or using a medicine. It is possible that the leaflet in your medicine pack may differ from this version because it may have been updated since your medicine was packaged.

Below is a text only representation of the Patient Information Leaflet. The original leaflet can be viewed using the link above.

The text only version may be available in large print, Braille or audio CD. For further information call emc accessibility on 0800 198 5000. The product code(s) for this leaflet is: EU/1/12/782/004.


Kalydeco 50mg and 75mg granules sachets

Package leaflet: Information for the patient

Kalydeco 50 mg granules in sachet

Kalydeco 75 mg granules in sachet

ivacaftor

Read all of this leaflet carefully before your child starts taking this medicine because it contains important information for your child.

  • Keep this leaflet. You may need to read it again.
  • If you have any further questions, ask your child’s doctor or pharmacist.
  • This medicine has been prescribed for your child only. Do not pass it on to others. It may harm them, even if their signs of illness are the same as your child’s.
  • If your child gets any side effects, talk to your child’s doctor or pharmacist. This includes any possible side effects not listed in this leaflet. See section 4.

What is in this leaflet

1. What Kalydeco is and what it is used for
2. What you need to know before your child takes Kalydeco
3. How to take Kalydeco
4. Possible side effects
5. How to store Kalydeco
6. Contents of the pack and other information

1. What Kalydeco is and what it is used for

Kalydeco contains the active ingredient ivacaftor. Ivacaftor acts at the level of the cystic fibrosis transmembrane conductance regulator (CFTR), a protein that forms a channel at the cell surface that allows the movement of particles such as chloride in and out of the cell. Due to mutations in the CFTR gene (see below), chloride movement is reduced in those with cystic fibrosis (CF). Ivacaftor helps certain abnormal CFTR proteins open more often to improve chloride movement in and out of the cell.

Kalydeco granules are indicated for the treatment of children with cystic fibrosis (CF) aged 12 months and older and weighing 7 kg to less than 25 kg who have one of the following gating mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.

2. What you need to know before your child takes Kalydeco

Do not take Kalydeco

  • if your child is allergic to ivacaftor or any of the other ingredients of this medicine (listed in section 6).

Warnings and precautions

  • Talk to your child’s doctor if your child has liver problems, or has had them previously. Your childs doctor may need to adjust your childs dose.
  • Increased liver enzymes in the blood have been seen in some people receiving Kalydeco. Tell your child’s doctor right away if your child has any of these symptoms, which may be a sign of liver problems:
    • Pain or discomfort in the upper right stomach (abdominal) area
    • Yellowing of the skin or the white part of the eyes
    • Loss of appetite
    • Nausea or vomiting
    • Dark urine

Your child’s doctor will do some blood tests to check your child’s liver before and during treatment, particularly during the first year and especially blood tests showed high liver enzymes in the past.

  • Talk to your child’s doctor if you have been told your child has kidney problems, or has previously had them.
  • Kalydeco is not recommended for patients who have undergone an organ transplant.
  • Abnormality of the eye lens (cataract) without any effect on vision has been noted in some children and adolescents during treatment.
    Your child’s doctor may perform some eye examinations prior to and during treatment with ivacaftor.

Children

Kalydeco is not to be used in children under 12 months of age with gating mutations as it is not known if ivacaftor is safe and effective in these children.

Other medicines and Kalydeco

Tell your child’s doctor or pharmacist if your child is using, has recently used or might use any other medicines. Some medicines can affect how Kalydeco works, or make side effects more likely. In particular, tell your child’s doctor if you are taking any of the medicines listed below. Your child’s doctor may decide to adjust your dose or that you need extra checkups.

  • Antifungal medicines such as ketoconazole, itraconazole, posaconazole, voriconazole and fluconazole, used for the treatment of fungal infections
  • Antibacterial agents such as telithromycin, clarithromycin, erythromycin, rifampicin and rifabutin, used for the treatment of bacterial infections
  • Anticonvulsant medicines such as phenobarbital, carbamazepine and phenytoin, used for the treatment of epileptic seizures
  • Herbal medicines such as St. John’s wort (Hypericum perforatum)
  • Immunosuppressants such as ciclosporin, tacrolimus, everolimus and sirolimus used after an organ transplantation
  • Cardiac glycosides such asdigoxin, used for the treatment of mild to moderate congestive heart failure and an abnormal heart rhythm called atrial fibrillation
  • Anticoagulant medicines such as warfarin, used to prevent blood clots from forming or growing larger in blood and blood vessels
  • Medicines for diabetes such as glimepiride and glipizide, used to lower sugar blood levels

Kalydeco with food and drink

Avoid giving your child food or drink containing grapefruit or Seville oranges during treatment with Kalydeco as they may increase the side effects of Kalydeco by increasing the amount of ivacaftor in your child’s body.

Driving and using machines

Kalydeco can make your child dizzy. If your child feels dizzy, it is advised that your child does not ride his/her bike or do anything else that needs his/her full attention.

Important information about the contents of Kalydeco

Kalydeco contains lactose. If you have been told by your child’s doctor that your child has an intolerance to some sugars, contact your child’s doctor before your child takes this medicine.

Kalydeco contains less than 1 mmol sodium (23 mg) per dose, that is to say essentially ‘sodium-free’.

3. How to take Kalydeco

Always give your child this medicine exactly as your child’s doctor has told you to. Check with your child’s doctor if you are not sure.

Your child’s doctor will determine the correct dose for your child. Your child must keep using all other medicines, unless your child’s doctor tells him/her to stop using any.

Kalydeco dosing recommendations are provided in Table 1.

Table 1 provides dosing recommendations for patients aged 12 months and older. Details are as follows:

The recommended dose for a patient aged 12 months and older and weighing 7 kg to less than 14 kg is one sachet of 50 mg granules taken orally every 12 hours with fat-containing food. This gives a total daily dose of 100 mg.

The recommended dose for a patient aged 12 months and older and weighing 14 kg to less than 25 kg is one sachet of 75 mg granules taken orally every 12 hours with fat-containing food. This gives a total daily dose of 150 mg.

If a patient is aged 12 months and older and weighs 25 kg or more please refer to the Kalydeco tablets Package Leaflet for dosing recommendations.

If your child has liver problems, either moderate or severe, your child’s doctor may need to reduce the dose of Kalydeco, as your child’s liver will not clear the medicine as fast as in children who have normal liver function.

  • Moderate liver problems: the dose may be reduced to one sachet once daily (50 mg for children weighing less than 14 kg and 75 mg for children weighing 14 kg to less than 25 kg).
  • Severe liver problems: the use is not recommended but your child’s doctor will decide if it is appropriate for your child to use this medical product in which case the dose must be reduced to one sachet every other day (50 mg for children weighing less than 14 kg and 75 mg for children weighing 14 kg to less than 25 kg).

Kalydeco is for oral use.

Each sachet is for single use only.

Giving Kalydeco to your child

  • Hold sachet of granules with cut line on top.
  • Shake sachet gently to settle contents.
  • Tear or cut sachet open along cut line.
  • Mix the entire content of a sachet with 5 mL of age-appropriate soft food or liquid. Food or liquid should be at room temperature or below. Some examples of age-appropriate soft foods or liquids include puréed fruits or vegetables, yogurt, applesauce, water, milk, or juice.
  • Once mixed, give the product to your child immediately. If this is not possible, give it within the following hour after mixing. Ensure that the mixture is completely and immediately consumed.
  • A fat-containing meal or snack should be given to your child just before or just after dosing (some examples are provided below).

Meals or snacks that contain fat include those prepared with butter or oils or those containing eggs. Other fat-containing foods are:

  • Cheese, whole milk, whole-milk dairy products, yogurt, chocolate
  • Meats, oily fish
  • Avocados, hummus, soy-based products (tofu)
  • Nuts, fat-containing nutritional bars or drinks

If your child takes more Kalydeco than he/she should

Your child may experience side effects, including those mentioned in section 4 below. If so, contact your child’s doctor or pharmacist to ask for advice. If possible, have your child’s medicine and this leaflet with you.

If you forget to give your child Kalydeco

Give the missed dose if less than 6 hours have passed since the time your child missed the dose. Otherwise, wait until your child’s next scheduled dose as you normally would. Do not give your child a double dose to make up for a forgotten dose.

If you stop giving your child Kalydeco

Give Kalydeco to your child for as long as your child’s doctor recommends. Do not stop unless your child’s doctor advises you to. If you have any further questions on the use of this medicine, ask your child’s doctor or pharmacist.

4. Possible side effects

Like all medicines, this medicine can cause side effects, although not everybody gets them.

Serious side effects include stomach (abdominal) ache and increased liver enzymes in the blood. Contact your child’s doctor straight away if he/she gets any of these.

Very common side effects (may affect more than 1 in 10 people)

  • Upper respiratory tract infection (the common cold), including sore throat and nasal congestion
  • Headache
  • Dizziness
  • Diarrhoea
  • Rash
  • Changes in the type of bacteria in mucus

Common side effects (may affect up to 1 in 10 people)

  • Runny nose
  • Ear pain, ear discomfort
  • Ringing in the ears
  • Redness inside the ear
  • Inner ear disorder (feeling dizzy or spinning)
  • Sinus congestion
  • Redness in the throat
  • Breast mass

Uncommon side effects (may affect up to 1 in 100 people)

  • Ear congestion
  • Breast inflammation
  • Enlargement of the breast
  • Nipple changes or pain

Additional side effects in children and adolescents

Side effects seen in children and adolescents are similar to those observed in adults. However, increased liver enzymes in the blood are more frequently seen in young children.

Reporting of side effects

If your child gets any side effects, talk to your child’s doctor or pharmacist. This includes any possible side effects not listed in this leaflet. You can also report side effects directly via the

Yellow Card Scheme
Website: www.mhra.gov.uk/yellowcard

or search for MHRA Yellow Card in the Google Play or Apple App Store

By reporting side effects you can help provide more information on the safety of this medicine.

5. How to store Kalydeco

Keep this medicine out of the sight and reach of children.

Do not use this medicine after the expiry date which is stated on the package after EXP. The expiry date refers to the last day of that month.

This medicine does not require any special storage conditions.

Once mixed, the mixture has been shown to be stable for one hour.

Do not throw away any medicines via wastewater or household waste. Ask your pharmacist how to throw away medicines you no longer use. These measures will help to protect the environment.

6. Contents of the pack and other information

What Kalydeco contains

  • Kalydeco 50 mg granules in sachet: The active substance is ivacaftor. Each sachet contains 50 mg of ivacaftor.
  • Kalydeco 75 mg granules in sachet: The active substance is ivacaftor. Each sachet contains 75 mg of ivacaftor.
  • The other ingredients are: colloidal silicon dioxide, croscarmellose sodium, hypromellose acetate succinate, lactose monohydrate, magnesium stearate, mannitol, sucralose and sodium laurilsulfate (E487).

See the end of section 2 - Important information about the contents of Kalydeco.

What Kalydeco looks like and contents of the pack

Kalydeco 50 mg granules in sachet are white to off-white granules

Kalydeco 75 mg granules in sachet are white to off-white granules

The granules are supplied in sachets.

  • Pack size of 56 sachets (contains 4 individual wallets with 14 sachets per wallet)

Marketing Authorisation Holder and Manufacturer

Marketing Authorisation Holder:

Vertex Pharmaceuticals (Ireland) Limited
28-32 Pembroke Street Upper
Dublin 2
D02 EK84
Ireland
Tel: +353 (0)1 761 7299

Manufacturer:

Almac Pharma Services (Ireland) Limited
Finnabair Industrial Estate
Dundalk
Co. Louth
A91 P9KD
Ireland

Almac Pharma Services Limited
Seagoe Industrial Estate
Craigavon
County Armagh
BT63 5UA
United Kingdom

This leaflet was last revised in February 2019.

Other sources of information

Detailed information on this medicine is available on the European Medicines Agency website: http://www.ema.europa.eu. There are also links to other websites about rare diseases and treatments.