Patient Leaflet Updated 11-May-2020 | Alexion Pharma UK Ltd
KANUMA 2mg/ml concentrate solution
Package leaflet: Information for the user
KANUMA 2 mg/ml concentrate for solution for infusion
sebelipase alfa
▼This medicine is subject to additional monitoring. This will allow quick identification of new safety information. You can help by reporting any side effects you or your child may get. See the end of section 4 for how to report side effects.
Read all of this leaflet carefully before you are given this medicine because it contains important information for you.
What is in this leaflet
1. What KANUMA is and what it is used for
2. What you need to know before you are given KANUMA
3. How KANUMA is given
4. Possible side effects
5. How to store KANUMA
6. Contents of the pack and other information
1. What KANUMA is and what it is used for
KANUMA contains the active substance sebelipase alfa. Sebelipase alfa is similar to the naturally occurring enzyme lysosomal acid lipase (LAL), which the body uses to breakdown fats. It is used to treat patients of all ages with lysosomal acid lipase deficiency (LAL deficiency).
LAL deficiency is a genetic disease that leads to liver damage, high blood cholesterol, and other complications due to a build-up of certain types of fats (cholesteryl esters and triglycerides).
How KANUMA works
This medicine is an enzyme replacement therapy. This means that it replaces the missing or defective LAL enzyme in patients with LAL deficiency. This medicine works by lowering the build-up of fat that causes medical complications, including impaired growth, liver damage and heart complications. It also improves blood levels of fats, including elevated LDL (bad cholesterol) and triglycerides.
2. What you need to know before KANUMA is given
You must not be given KANUMA
Warnings and precautions
Other medicines and KANUMA
Tell your doctor if you or your child are using, have recently used or might use any other medicines.
Pregnancy
There are no data from the use of sebelipase alfa in pregnant women. As a precautionary measure, you should not be given KANUMA if you are pregnant.
Breast-feeding
It is not known whether sebelipase alfa passes into breast milk. Tell your doctor if you are breast-feeding or plan to do so. Your doctor will then help you decide whether to stop breast-feeding, or whether to stop taking KANUMA, considering the benefit of breast-feeding to the baby and the benefit of KANUMA to the mother.
If you are pregnant or breast-feeding, think you may be pregnant or are planning to have a baby, ask your doctor for advice before taking this medicine.
Driving and using machines
KANUMA has no or negligible influence on the ability to drive and use machines.
KANUMA contains sodium
This medicine, when diluted with sodium chloride 9 mg/mL (0.9%) solution for injection for intravenous administration contains 33 mg sodium (main component of cooking/table salt) at the recommended dose. This is equivalent to 1.7% of the recommended maximum daily dietary intake of sodium for an adult. Tell your doctor if you or your child is on a controlled sodium diet.
3. How KANUMA is given
The dose you or your child receives is based on your or your child’s body weight.
Infants (< 6 months of age)
For patients who have signs and symptoms of the disease when they are infants, the recommended starting dose is 1 mg/kg once weekly. Dose adjustments may be considered based on how well you or your child responds to treatment.
Children and adults
The recommended dose is 1 mg per kg body weight once every other week through a drip into a vein.
Each infusion will take approximately 1 to 2 hours. You or your child may be monitored by your doctor or nurse for an additional hour after the infusion. KANUMA should be started at as young an age as possible and is intended for long-term use.
Your doctor or nurse will give KANUMA to you or your child by an infusion (drip) into a vein. The medicine will be diluted before being given to you or your child.
4. Possible side effects
Like all medicines, this medicine can cause side effects, although not everybody gets them.
Side effects were seen while patients were being given the medicine or shortly after (infusion reactions). The most serious side effects may include an allergic reaction (seen very commonly [may affect more than 1 in 10 people] in infants younger than 6 months old, or commonly [may affect up to 1 in 10 people] in children and adults) with symptoms including difficulty breathing, swelling of the throat, rapid breathing, fast heartbeat, chest discomfort, mild swelling of eyelids, red eyes, runny nose, flushing, and hives. If you or your child experiences symptoms like these, seek immediate medical attention. If you or your child has an infusion reaction you or your child may be given additional medicines to treat or help prevent future reactions. If the infusion reaction is severe, your doctor may stop the infusion of KANUMA in the vein and start giving appropriate medical treatment.
Very common
eyelid swelling
decreased muscle tone
paleness
cough
diarrhoea
vomiting
red swollen skin
chills
fast heartbeat
agitation
difficulty breathing
stuffy or swollen nose
heartburn (reflux diseases)
hives
itching
fever
rapid breathing
irritability
high blood pressure
wheezing
sneezing
dry heaving
rash
raised rash
swelling
decreased oxygen in the blood
Common
severe allergic reaction (anaphylactic reaction)
temporary increased cholesterol or triglyceride (fats) levels in the blood
Sleeplessness
redness in face
Diarrhoea
Nausea
Itching
Shivers
Tiredness
infection of the urinary system
fast heartbeat
Dizziness
shortness of breath
stomach ache
Hives
red swollen skin
chest discomfort
hardened area around the infusion site
swelling of the eyelids
anxiety
low blood pressure
swelling of the throat
stomach bloating
rash
increased menstrual bleeding
swelling
fever
Frequency, type and severity of adverse reactions in children are the same as in adults.
Reporting of side effects
If you get any side effects, talk to your doctor or pharmacist or nurse. This includes any possible side effects not listed in this leaflet. You can also report side effects directly via the national reporting system listed below:
United Kingdom:
via the
or search for MHRA Yellow Card in the Google Play or Apple App Store.
Ireland:
5. How to store KANUMA
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 label and carton after EXP. The expiry date refers to the last day of that month.
Store in a refrigerator (2 °C to 8 °C). Do not freeze. Do not shake. Store in the original package in order to protect from light.
For diluted solutions, immediate use is recommended. If not used immediately, the diluted solution may be stored up to 24 hours at 2 °C to 8 °C or up to 12 hours below 25 °C.
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 protect the environment.
6. Contents of the pack and other information
What KANUMA contains
What KANUMA looks like and contents of the pack
KANUMA is supplied as a concentrate for solution for infusion (sterile concentrate). It is a solution that is clear to slightly opalescent, and colourless to slightly coloured.
Pack size: 1 vial containing 10 ml of concentrate.
Marketing Authorisation Holder and Manufacturer
Marketing Authorisation Holder
Manufacturer:
This leaflet was last revised in April 2020.
Other sources of information
Detailed information on this medicine is available on the European Medicines Agency web site: http://www.ema.europa.eu. There are also links to other websites about rare diseases and treatments.
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