KANUMA 2 mg /Ml Concentrate For Infusion Solution

Зміст інструкції

1. KANUMA 2 mg/mL concentrate for solution for infusion
   1. What is KANUMA and what is it used for
   2. What you need to know before you receive KANUMA
   3. How KANUMA is administered
   4. Possible side effects
   5. How to store KANUMA
   6. Contents of the pack and other information

KANUMA 2 mg/mL concentrate for solution for infusion

sebelipase alfa

Medicinal product subject to additional monitoring. This will allow the rapid identification of
new safety information. You can contribute by reporting any adverse effects
experienced during the use of this medicine by you or your child. See the
end of section 4 for information on how to report adverse effects.

Read this leaflet carefully before receiving this medicine as it contains
important information for you.

• Keep this leaflet. You may need to read it again.
• If you have any questions, ask your doctor.
• If you experience any side effects, including those not listed in this leaflet, tell your doctor. See section 4.

Contents of this leaflet:

• 1. What KANUMA is and what it is used for
• 2. What you need to know before you receive KANUMA
• 3. How KANUMA is administered
• 4. Possible side effects
• 5. How to store KANUMA
• 6. Contents of the pack and other information

1. What is KANUMA and what is it used for

KANUMA contains the active substance sebelipase alfa. Sebelipase alfa is similar to the lysosomal acid lipase (LAL) enzyme naturally present in the body and that the body uses to break down fats. It is
used to treat patients of all ages affected by lysosomal acid lipase deficiency (LAL deficiency).
LAL deficiency is a genetic disease that causes damage to the liver, high levels of cholesterol
in the blood and other complications due to the accumulation 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 LAL enzyme
missing or defective in patients affected by LAL deficiency. This medicine works by reducing
the accumulation of fat that causes medical complications, including growth impairment, liver
damage and cardiac complications. It also improves fat levels in the blood, including levels
increased LDL cholesterol (bad cholesterol) and triglycerides.

2. What you need to know before you receive KANUMA

You must not receive KANUMA

• If you or the child have experienced potentially life-threatening allergic reactions to sebelipase alfa that cannot be managed by re-administering the medicine, or allergic reactions to egg or any of the other ingredients of this medicine (listed in section 6).

Warnings and precautions

• During treatment with KANUMA, you or the child may experience undesirable effects during the administration of the medicine or during the hours following the infusion (see section 4). This so-called “infusion reaction” can sometimes be severe and may include an allergic reaction that could be life-threatening and require medical treatment. When you or the child receive KANUMA for the first time, you or the child must be monitored by a healthcare professional for 1 hour, in order to detect any signs of an infusion reaction. If you or the child experience a severeinfusion reaction of this type, seek immediate medical attention. If you or the child experience an infusion reaction, you may be given further medicines to treat or prevent future reactions. These medicines may include antihistamines, medicines to reduce fever and/or corticosteroids (a type of anti-inflammatory medicine). If the infusion reaction is severe, the doctor may decide to stop the KANUMA infusion and start administering appropriate medical treatment to you or the child.
• During the course of treatment, proteins directed against KANUMA, called “anti-drug antibodies”, may develop in the blood. If you notice a decrease in the effectiveness of KANUMA, talk to your doctor.
• This medicine may contain egg proteins. If you or the child suffer from an egg allergy or have a history of egg allergy, inform your doctor or nurse (see section You must not receive KANUMA).

Other medicines and KANUMA
Tell your doctor if you or the child are taking, have recently taken or might take
any other medicine.

Pregnancy
There are no data regarding the use of sebelipase alfa in pregnant women. As a precaution, if
you are pregnant, KANUMA should not be administered to you.

Breast-feeding
It is not known whether sebelipase alfa passes into breast milk. Inform your doctor if you are breast-feeding or if you intend to do so. The doctor will help you decide whether to stop breast-feeding or the
treatment with KANUMA, taking into account the benefit of breast-feeding for the baby and
the benefit of KANUMA for the mother.
If you are pregnant, suspect you are pregnant, are planning to become pregnant or are breast-feeding,
ask your doctor for advice before taking this medicine.

Driving and using machines
KANUMA may slightly impair the ability to drive or operate machines. Among the undesirable effects of sebelipase alfa are dizziness, which may affect the ability to drive
vehicles or operate machines.

KANUMA contains sodium
When diluted with sodium chloride 9 mg/mL (0.9%) solution for
intravenous infusion, this medicine contains 33 mg of sodium (the main component of
table salt) at the recommended dose. This is equivalent to 1.7% of the maximum recommended daily
intake in the diet of an adult. Inform your doctor if you or the child are following a
low-sodium diet.

3. How KANUMA is administered

The dose that you or your child will receive depends on your body weight.

Newborns (less than 6 months of age)
For newborn patients with signs and symptoms of the disease, the recommended initial dose is 1 mg/kg or
3 mg/kg once a week. Depending on the child's response to treatment, dose adjustments may
be considered.

Children and adults
The recommended dose is 1 mg per kg of body weight every other week by intravenous
infusion (into a vein). Dose adjustments may be considered based on how you or your child
respond to treatment.
Each infusion will take approximately 1-2 hours. The doctor or nurse may monitor you or your child for an additional hour after the infusion. KANUMA should be started as soon as possible and is
designed for long-term use.
The doctor or nurse will administer KANUMA to you or your child by infusion
into a vein. The medicine must be diluted before being administered 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 have been observed during or immediately after administration of the medicine
(infusion reactions). The most serious side effects included an allergic reaction
(observed very commonly [may affect more than 1 in 10 people] in infants under 6 months of
age; and observed commonly [may affect up to 1 in 10 people] in children and adults), with
symptoms including difficulty breathing, rapid breathing, fast heartbeat, chest discomfort, mild
swelling of the eyelids, red eyes, runny nose, flushing, hives, itching, diarrhea,
paleness, wheezing, low blood oxygen levels, skin redness and irritability. If
you or your child develop symptoms like these, seek immediate medical attention.If you or your child
develop an infusion reaction, you may be given further medicines to treat or prevent future
reactions. If the infusion reaction is severe, your doctor may decide to stop the KANUMA infusion
and start appropriate medical treatment.

Very common side effects (may affect more than 1 in 10 people) reported in
infants (from 1 to 6 months of age) are:
Hypersensitivity (irritability, agitation, vomiting, hives, eczema, itching, paleness and drug
hypersensitivity), severe allergic reactions (anaphylactic reactions)
Swelling of the eyelids
Fast heartbeat
Difficulty breathing
Diarrhea, vomiting
Skin rash, raised rash
Fever
Reduced oxygen in the blood, increased blood pressure, rapid breathing, development
of proteins in the blood

Very common side effects (may affect 1 or more in 10 people) reported in
children and adolescents (from 4 to 18 years of age) and in adults are:
Hypersensitivity (chills, eczema, laryngeal edema, nausea, itching and hives)
Dizziness
Stomach pain, diarrhea
Fatigue, fever

Common side effects (may affect up to 1 in 10 people) reported in children and
adolescents (between 4 and 18 years of age) and adults are:
Severe allergic reaction (anaphylactic reaction)
Fast heartbeat
Skin redness, low blood pressure
Shortness of breath
Stomach swelling
Skin rash, red and swollen skin
Chest discomfort, reaction at the infusion site
Frequency, type and severity of adverse reactions in children are similar to those in adults.

Reporting of side effects
If you or your child gets any side effect, talk to your doctor. You can also report side effects directly
through the national reporting system listed in Annex V. By reporting side effects you can help
provide more information on the safety of this medicine.

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 on the carton after
EXP/Scad. The expiry date refers to the last day of that month.
Store in a refrigerator (2 °C-8 °C). Do not freeze. Do not shake. Store in the original
packaging to protect the medicine from light.
For diluted solutions, immediate use is recommended. If not used immediately, the diluted
solution may be stored for up to 24 hours at a temperature of 2-8 °C or up to 12 hours at a
temperature below 25 °C.
Do not dispose of any medicine in wastewater or household waste. Ask your pharmacist how to
dispose of medicines you no longer use. This will help protect the environment.

6. Contents of the pack and other information

What KANUMA contains

• The active substance is sebelipase alfa. Each mL of concentrate contains 2 mg of sebelipase alfa. Each vial contains 20 mg of sebelipase alfa in 10 mL.
• The other ingredients are sodium citrate (see section 2, “KANUMA contains sodium”), citric acid monohydrate, human serum albumin and water for injections.

Description of KANUMA and contents of the pack
KANUMA is supplied as a concentrate for infusion solution (sterile concentrate). It is a
solution from transparent to slightly opalescent and from colourless to slightly coloured.
Pack: 1 vial containing 10 mL of concentrate.

Marketing authorisation holder and manufacturer
Marketing authorisation holder:
Alexion Europe SAS
103-105 rue Anatole France
92300 Levallois-Perret
France
Manufacturer:
Almac Pharma Services
Seagoe Industrial Estate
Craigavon BT63 5UA
United Kingdom
Alexion Pharma International Operations Limited
College Business and Technology Park
Blanchardstown Road North
Dublin 15
D15 R925
Ireland
For further information on this medicinal product, contact the local representative of the marketing
authorisation holder:

België/Belgique/Belgien
Alexion Pharma Belgium
Tél/Tel: +32 0 800 200 31

Lietuva
UAB AstraZeneca Lietuva
Tel: +370 5 2660550

България
АстраЗенека България ЕООД
Teл.: +359 24455000

Luxembourg/Luxemburg
Alexion Pharma Belgium
Tél/Tel: +32 0 800 200 31

Česká republika
AstraZeneca Czech Republic s.r.o.
Tel: +420 222 807 111

Magyarország
AstraZeneca Kft.
Tel.: +36 1 883 6500

Danmark
Alexion Pharma Nordics AB
Tlf: +46 0 8 557 727 50

Malta
Alexion Europe SAS
Tel: +353 1 800 882 840

Deutschland
Alexion Pharma Germany GmbH
Tel: +49 (0) 89 45 70 91 300

Nederland
Alexion Pharma Netherlands B.V.
Tel: +32 (0)2 548 36 67

Eesti
AstraZeneca
Tel: +372 6549 600

Norge
Alexion Pharma Nordics AB
Tlf: +46 (0)8 557 727 50

Ελλάδα
AstraZeneca A.E.
Τηλ: +30 210 6871500

Österreich
Alexion Pharma Austria GmbH
Tel: +41 44 457 40 00

España
Alexion Pharma Spain, S.L.
Tel: +34 93 272 30 05

Polska
AstraZeneca Pharma Poland Sp. z o.o.
Tel.: +48 22 245 73 00

France
Alexion Pharma France SAS
Tél: +33 1 47 32 36 21

Portugal
Alexion Pharma Spain, S.L. - Sucursal em
Portugal
Tel: +34 93 272 30 05

Hrvatska
AstraZeneca d.o.o.

România
AstraZeneca Pharma SRL
Tel: +385 1 4628 000 Tel: +40 21 317 60 41

Ireland
Alexion Europe SAS
Tel: +353 1 800 882 840

Slovenija
AstraZeneca UK Limited
Tel: +386 1 51 35 600

Ísland
Alexion Pharma Nordics AB
Sími: +46 0 8 557 727 50

Slovenská republika
AstraZeneca AB, o.z.
Tel: +421 2 5737 7777

Italia
Alexion Pharma Italy srl
Tel: +39 02 7767 9211

Suomi/Finland
Alexion Pharma Nordics AB
Puh/Tel: +46 0 8 557 727 50

Κύπρος
Alexion Europe SAS
Τηλ: +357 22490305

Sverige
Alexion Pharma Nordics AB
Tel: +46 0 8 557 727 50

Latvija
SIA AstraZeneca Latvija
Tel: +371 67377100

United Kingdom (Northern Ireland)
Alexion Europe SAS
Tel: +44 (0) 800 028 4394

Other sources of information
More detailed information on this medicinal product is available on the website of the European Medicines
Agency: http://www.ema.europa.eu. Also, links to other websites on rare diseases and related
therapeutic treatments are provided.
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The following information is intended exclusively for healthcare professionals:
In the absence of compatibility studies, this medicinal product must not be mixed with other medicinal products.
Each vial of KANUMA is for single use only. KANUMA must be diluted with
sodium chloride 9 mg/mL (0.9%) infusion solution using aseptic technique.
The diluted solution must be administered to patients using a low-protein-binding infusion set equipped with a low-protein-binding 0.2 μm in-line filter, with a surface
area greater than 4.5 cm², if available, to prevent filter occlusion.
Preparation of sebelipase alfa infusion
KANUMA must be prepared and used according to the instructions below. Aseptic technique must be
used.

• a. The number of vials to be diluted for the infusion must be established based on the patient's body weight and the prescribed dose.
• b. It is recommended to allow vials of KANUMA to warm to a temperature between 15 and 25 ºC before dilution to minimise the possibility of sebelipase alfa protein particles forming in the solution. Vials should not be left out of the refrigerator for more than 24 hours before dilution for infusion. Vials should not be frozen, heated or microwaved and must be protected from light.
• c. Vials must not be shaken. Before dilution, the concentrate in the vials must be inspected visually; the concentrate must appear from transparent to slightly opalescent, from colourless to slightly coloured (yellow). Due to the protein nature of

the medicinal product, the concentrate in the vial may exhibit slight flocculation (fine translucent
fibres), which is acceptable for use.

• d. Do not use the concentrate if it appears turbid or if any foreign particulate matter is observed.
• e. Up to 10 mL of concentrate from each vial must be drawn slowly and diluted in sodium chloride 9 mg/mL (0.9%) infusion solution. For recommended total infusion volumes per weight range, see Table 1. The solution must be mixed gently and must not be shaken.

Table 1. Recommended infusion volumes*

* The infusion volume must be based on the prescribed dose and must be prepared to
achieve a final sebelipase alfa concentration of 0.1-1.5 mg/mL.
**For patients with LAL deficiency manifesting in the first 6 months of life who do not achieve
an optimal clinical response with a dose of 3 mg/kg.
Any unused medicinal product or waste material derived from such medicinal product should be disposed of in accordance with
local regulations.