Patient leaflet - Kalydeco 13.4 mg granules in sachet
• 1 What Kalydeco is and what it is used for
Kalydeco contains the active substance 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:
- As monotherapy for the treatment of babies and children aged 1 month and older and weighing 3 kg to less than 25 kg with cystic fibrosis (CF) who have an R117H CFTR mutation or one of the following gating mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.
- In combination with ivacaftor/tezacaftor/elexacaftor granules for patients aged 2 to 6 years who have CF, with at least one F508del mutation in the CFTR gene. If you have been prescribed Kalydeco to be taken with ivacaftor/tezacaftor/ elexacaftor, read the package leaflet of the latter. It contains important information about how to take these two medicines.
• 2 What you need to know before your child takes Kalydeco
Do not give your child 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 before your child takes Kalydeco.
- Talk to your child’s doctor if your child has liver problems or has had them previously. Your child’s doctor may need to adjust your child’s 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 if 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
Do not give this medicine to children under 1 month of age as it is not known if ivacaftor is safe and effective in these children.
Do not give this medicine in combination with ivacaftor/tezacaftor/elexacaftor to children under 2 years of age as it is not known if they are safe and effective for them.
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 your child is taking any of the medicines listed below. Your child’s doctor may decide to adjust your child’s dose or if extra check-ups are needed.
- Antifungal medicines (used for the treatment of fungal infections). These include fluconazole, itraconazole, ketoconazole, posaconazole, and voriconazole.
- Antibiotic medicines (used for the treatment of bacterial infections). These include clarithromycin, erythromycin, rifabutin, rifampicin, and telithromycin.
- Epilepsy medicines (used for the treatment of epileptic seizures or fits). These include carbamazepine, phenobarbital, and phenytoin.
- Herbal medicines. These include St. John’s wort (Hypericum perforatum).
- Immunosuppressants (used after an organ transplantation). These include ciclosporin, everolimus, sirolimus, and tacrolimus.
- Cardiac glycosides (used for the treatment of some heart conditions). These include digoxin.
- Anticoagulant medicines (used to prevent blood clots). These include warfarin.
- Medicines for diabetes. These include glimepiride and glipizide.
- Medicines for lowering blood pressure. These include verapamil.
Kalydeco with food and drink
Avoid giving your child food or drink containing grapefruit 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.
Kalydeco contains lactose and sodium.
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: Dosing recommendations
Age Weight Morning dose Evening dose Kalydeco as monotherapy
1 month to less
than 3 months
≥ 3 kg One sachet of
ivacaftor 13.4 mg granules
One sachet of ivacaftor 13.4 mg granules
3 months to less
than 6 months
≥ 3 kg One Kalydeco sachet of
25 mg granules
One Kalydeco sachet of 25 mg granules 6 months and
older
≥ 5 kg to
< 7 kg
One Kalydeco sachet of
25 mg granules
One Kalydeco sachet of 25 mg granules 7 kg to
< 14 kg
One Kalydeco sachet of
50 mg granules
One Kalydeco sachet of 50 mg granules 14 kg to
< 25 kg
One Kalydeco sachet of
75 mg granules
One Kalydeco sachet of 75 mg granules ≥ 25 kg Please refer to Kalydeco tablets Package Leaflet Kalydeco in combination with ivacaftor/tezacaftor/elexacaftor
2 years to less
than 6 years,
< 14 kg One sachet of ivacaftor 60 mg/ tezacaftor 40 mg/
elexacaftor 80 mg granules
One Kalydeco sachet of 59.5 mg granules
≥ 14 kg One sachet of ivacaftor 75 mg/ tezacaftor 50 mg/
elexacaftor 100 mg granules
One Kalydeco sachet of 75 mg granules
Give your child the morning and evening granules about 12 hours apart.
If your child has liver problems, 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 in children 6 months of age or older: the dose may be reduced to one half of the indicated dose in the table above, that is one sachet once daily.
- Severe liver problems in children 6 months of age or older: the use is not recommended but your child’s doctor will decide if it is appropriate for your child to use this medicine in which case the dose (as indicated in the table above) must be reduced to one sachet every other day.
- Liver problems in children between 4 months and 6 months of age: the use is not recommended but your child’s doctor will decide if it is appropriate for your child to use and what dose your child should have.
- Liver problems in children between 1 month and 4 months of age: the use is not recommended.
Kalydeco is for oral use.
Each sachet is for single use only.
➤ PX3724/1
PX3724/1
Packaging Site: Almac
LFT-0007-02
Overall dimensions
560 x 280 mm
Folded dimensions (if needed)
70 x 35 mm
CMO Drawing
CDS-PX000078
CMO Code
10345000
Revision Date
01 Sep 2023
27 Jun 2024
Leaflet 01
PX3724/1 11 pt
37241 820350
N/A
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-appropriat liquid. Food or liquid should be at room temperature or below.
e soft food or Some examples
Possible signs of liver problems
Increased liver enzymes in the blood are be signs of liver problems:
- Pain or discomfort in the upper right a
- Yellowing of the skin or white part of t
- Loss of appetite
of age-appropriate soft foods or liquids include puréed fruits o yogurt, applesauce, water, milk, breast milk, infant formula, or j
- Once mixed, give the product to your child immediately. If this give it within the following hour after mixing. Ensure that the m completely and immediately consumed.
A fat-containing meal or snack should be given to your child ju after dosing (some examples are provided below).
r vegetables,
uice.
is not possible,
ixture is
st before or just
- Nausea or vomiting
- Dark urine
Tell your child’s doctor straight away if he
Very common side effects
(may affect more than 1 in 10 people)
Meals or snacks that contain fat include those prepared with butt those containing eggs. Other fat-containing foods are:
- Cheese, whole milk, whole-milk dairy products, yogurt, breast formula, chocolate
- Meats, oily fish
Avocados, hummus, soy-based products (tofu)
er or oils or
milk, infant
- Upper respiratory tract infection (the c nasal congestion
- Headache
- Dizziness
- Diarrhoea
- Stomach or abdominal pain
- 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 mentione below. If so, contact your child’s doctor or pharmacist to ask for a have your child’s medicine and this leaflet with you.
d in section 4 dvice. If possible,
- Changes in the type of bacteria in muc
- Increased liver enzymes (signs of stres
- Rash
Common side effects
(may affect up to 1 in 10 people)
- Runny nose
If you forget to give your child Kalydeco
Give the missed dose if less than 6 hours have passed since the ti child missed the dose. Otherwise, wait until your child’s next sche as you normally would. Do not give your child a double dose to m forgotten dose.
If you stop giving your child Kalydeco
Give Kalydeco to your child for as long as your child’s doctor reco
me your
duled dose
ake up for a
mmends. Do not
- Ear pain, ear discomfort
- Ringing in the ears
- Redness inside the ear
- Inner ear disorder (feeling dizzy or spi
- Sinus problems (Sinus congestion)
- Redness in the throat
stop unless your child’s doctor advises you to. If you have any furt 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 gets them.
her questions
not everybody
- Breast mass
- Flu• Low blood sugar (hypoglycaemia)
- Abnormal breathing (shortness of bre
- Wind (flatulence)
- Spots (Acne)
•
Serious side effects
Stomach (abdominal) ache and increased liver enzymes in the blo
od.
Itchy skin• Increased creatine phosphokinase (sig blood tests
Uncommon side effects
(may affect up to 1 in 100 people)
Ear congestion
common in patients with CF. These may rea of the stomach (abdominal) area he eyes
/she gets any of these.
ommon cold), including sore throat and us s on the liver)
nning)
ath or difficulty breathing)
n of muscle breakdown) seen in
- Breast inflammation
- Enlargement of the breast in males
- Nipple changes or pain
- Wheezing
Increased blood pressure
Additional side effects in children a
Side effects seen in children and adolesc adults. However, increased liver enzymes in young children.
Reporting of side effects
nd adolescents ents are similar to those observed in in the blood are more frequently seen
If your child gets any side effects, talk to y This includes any possible side effects no side effects directly via:
Yellow Card Scheme
Website: www.mhra.gov.uk/yellowcard o Google Play or Apple App Store.
our child’s doctor or pharmacist. t listed in this leaflet. You can also report r search for MHRA Yellow Card in the
By reporting side effects you can help pr this medicine.
• 5 How to store Kalydeco
Keep this medicine out of the sight and r
ovide more information on the safety of each of children.
Do not use this medicine after the expiry wallet and sachet after EXP. The expiry da
This medicine does not require any speci
Once mixed, the mixture has been shown
Do not throw away any medicines via wa
date which is stated on the carton, te refers to the last day of that month.
al storage conditions.
to be stable for one hour.
stewater or household waste. Ask your pharmacist how to throw away medicine help to protect the environment.
• 6 Contents of the pack and othe
What Kalydeco contains
Kalydeco 13.4 mg granules in sachet:
s you no longer use. These measures will r information
The active substance is ivacaftor. Each sa
Kalydeco 25 mg granules in sachet: The active substance is ivacaftor. Each sa
Kalydeco 50 mg granules in sachet: The active substance is ivacaftor. Each sa
chet contains 13.4 mg of ivacaftor.
chet contains 25 mg of ivacaftor.
chet contains 50 mg of ivacaftor.
Kalydeco 59.5 mg granules in sachet: The active substance is ivacaftor. Each sa
Kalydeco 75 mg granules in sachet: The active substance is ivacaftor. Each sa
The other ingredients are: silica, colloidal
chet contains 59.5 mg of ivacaftor.
chet contains 75 mg of ivacaftor.
anhydrous, croscarmellose sodium, hypromellose acetate succinate, lactose mannitol, sucralose and sodium laurilsulf
See the end of section 2 - Kalydeco conta
monohydrate, magnesium stearate, ate (E487).
ins lactose and sodium.
What Kalydeco looks like and conte
Kalydeco 13.4 mg granules in sachet are Kalydeco 25 mg granules in sachet are w Kalydeco 50 mg granules in sachet are w Kalydeco 59.5 mg granules in sachet are Kalydeco 75 mg granules in sachet are w
nts of the pack white to off-white granules. hite to off-white granules. hite to off-white granules. white to off-white granules. hite to off-white granules.
The granules are supplied in sachets.
Kalydeco 13.4 mg granules in sachet, Kaly Kalydeco 50 mg granules in sachet, and K
- Pack size of 56 sachets (contains 4 indi
Kalydeco 59.5 mg granules in sachet and
deco 25 mg granules in sachet, alydeco 75 mg granules in sachet: vidual wallets with 14 sachets per wallet)
Kalydeco 75 mg granules in sachet:
- Pack size of 28 sachets (contains 4 indi
Marketing Authorisation Holder
Vertex Pharmaceuticals (Europe) Limited 2 Kingdom Street
London, W2 6BD
United Kingdom
vidual wallets with 7 sachets per wallet)
Tel: +44 (0)203 204 5100
Manufacturer
Almac Pharma Services Limited Seagoe Industrial Estate Craigavon
Northern Ireland
BT63 5UA
United Kingdom
This leaflet was last revised in March 2
Other sources of information
Detailed information on this medicine is a Healthcare products Regulatory Agency
024 vailable on the Medicines and website: http://www.mhra.gov.uk.
Packaging Site: Almac
LFT-0007-02
Overall dimensions
560 x 280 mm
Folded dimensions (if needed) 70 x 35 mm
CMO Drawing
CDS-PX000078
CMO Code
10345000
Revision Date
01 Sep 2023
2 of 2 27 Jun 2024
Leaflet 01
PX3724/1 11 pt
37241 820350
N/A