Kanuma

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

• If you or your child has experienced lifethreatening allergic reactions to sebelipase alfa that cannot be managed when you or your child receives the medicine again, or to egg or any of the other ingredients of this medicine (listed in section 6).

Warnings and precautions

• If treated with KANUMA, you or your child may experience a side effect while you or your child is being given the medicine or during the hours following the infusion (see section 4). This is known as an infusion reaction which can sometimes be severe, and may include an allergic reaction that could be lifethreatening and require medical treatment. The first time that you or your child are given KANUMA you should be observed by a healthcare professional for 1 hour to watch for any signs of an infusion reaction. If you or your child experiences a severe infusion reaction like this, 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. These medicines may include antihistamines, feverreducing medicines and/or corticosteroids (a type of anti-inflammatory medicines).

If the infusion reaction is severe, your doctor may stop KANUMA infusion and start giving you or your child appropriate medical treatment.

• The development of blood proteins against KANUMA, also called antidrug antibodies, may occur during the treatment. Talk to your doctor if you experience decreased efficacy with KANUMA
• This medicine may contain egg proteins. If you or your child has an egg allergy or a history of allergies to eggs, tell your doctor or nurse (see You must not be given KANUMA).

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 may have a minor influence on the ability to drive and use machines. Adverse effects of sebelipase alfa include dizziness which could affect the ability to drive or use machines.

KANUMA contains sodium

This medicine, when diluted with sodium chloride 9 mg/mL (0.9%) solution for infusion 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 or 3 mg/kg once weekly. Dose adjustments may be considered based on how well 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. Dose adjustments may be considered based on how well you or your child responds to treatment.

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, rapid breathing, fast heartbeat, chest discomfort, mild swelling of eyelids, red eyes, runny nose, flushing, hives, itching, diarrhoea, paleness, wheezing, low blood oxygen, skin redness and irritability. 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 (may affect more than 1 in 10 people) side effects reported in infants (1 to 6 months old) are:

Hypersensitivity (irritability, agitation, vomiting, urticaria, eczema, pruritus, pallor and drug hypersensitivity), severe allergic reactions (anaphylactic reactions)

Eyelid swelling

Fast heartbeat

Difficulty breathing

Diarrhoea, vomiting

Rash, raised rash

Fever

Decreased oxygen in the blood, blood pressure increased, rapid breathing, development of blood proteins

Very Common (may affect 1 in 10 people or more) side effects reported in children and adolescents (4 to 18 years old) and adults are:

Hypersensitivity (chills, eczema, laryngeal oedema, nausea, pruritus and urticaria)

Dizziness

Stomach ache, diarrhoea

Tiredness, fever

Common (may affect up to 1 in 10 people) side effects reported in children and adolescents (4 to 18 years old) and adults are:

Severe allergic reaction (anaphylactic reaction),

Fast heartbeat

Skin redness, low blood pressure

Shortness in breath

Stomach bloating

Rash, red 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 effects, talk to your doctor. This includes any possible side effects not listed in this leaflet. You can also report side effects directly via the national reporting system detailed below:

Ireland

HPRA Pharmacovigilance

Website: www.hpra.ie

United Kingdom (Northern Ireland)

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 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

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

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

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 any information about this medicine, please contact the local representative of the Marketing Authorisation Holder: 

België/Belgique/Belgien 

Alexion Pharma Belgium 

Tél/Tel: +32 0 800 200 31 

България 

АстраЗенека България ЕООД 

Teл.: +359 24455000 

Česká republika 

AstraZeneca Czech Republic s.r.o. 

Tel: +420 222 807 111 

Danmark 

Alexion Pharma Nordics AB 

Tlf: +46 0 8 557 727 50 

Deutschland 

Alexion Pharma Germany GmbH 

Tel: +49 (0) 89 45 70 91 300 

Eesti 

AstraZeneca 

Tel: +372 6549 600 

Ελλάδα 

AstraZeneca A.E. 

Τηλ: +30 210 6871500 

España 

Alexion Pharma Spain, S.L. 

Tel: +34 93 272 30 05 

France 

Alexion Pharma France SAS 

Tél: +33 1 47 32 36 21 

Hrvatska 

AstraZeneca d.o.o. 

Tel: +385 1 4628 000 

Ireland 

Alexion Europe SAS 

Tel: +353 1 800 882 840 

Ísland 

Alexion Pharma Nordics AB 

Sími: +46 0 8 557 727 50 

Italia 

Alexion Pharma Italy srl 

Tel: +39 02 7767 9211  

Κύπρος 

Alexion Europe SAS 

Τηλ: +357 22490305 

Latvija 

SIA AstraZeneca Latvija 

Tel: +371 67377100 

Lietuva 

UAB AstraZeneca Lietuva 

Tel: +370 5 2660550 

Luxembourg/Luxemburg 

Alexion Pharma Belgium 

Tél/Tel: +32 0 800 200 31 

Magyarország 

AstraZeneca Kft. 

Tel.: +36 1 883 6500 

Malta 

Alexion Europe SAS 

Tel: +353 1 800 882 840 

Nederland 

Alexion Pharma Netherlands B.V. 

Tel: +32 (0)2 548 36 67 

Norge 

Alexion Pharma Nordics AB 

Tlf: +46 (0)8 557 727 50  

Österreich 

Alexion Pharma Austria GmbH 

Tel: +41 44 457 40 00 

Polska 

AstraZeneca Pharma Poland Sp. z o.o. 

Tel.: +48 22 245 73 00 

Portugal 

Alexion Pharma Spain, S.L. - Sucursal em Portugal  

Tel: +34 93 272 30 05 

România 

AstraZeneca Pharma SRL 

Tel: +40 21 317 60 41  

Slovenija 

AstraZeneca UK Limited 

Tel: +386 1 51 35 600 

Slovenská republika 

AstraZeneca AB, o.z. 

Tel: +421 2 5737 7777 

Suomi/Finland 

Alexion Pharma Nordics AB 

Puh/Tel: +46 0 8 557 727 50  

Sverige 

Alexion Pharma Nordics AB 

Tel: +46 0 8 557 727 50 

United Kingdom (Northern Ireland) 

Alexion Europe SAS 

Tel: +44 (0) 800 028 4394 

 This leaflet was last revised in June 2023.

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.