InfoCons Consumer Protection informs you – Kanuma – Package Leaflet

Before anything else

Read all of this leaflet carefully before you are gi ven this medicine because it contains important information for you.

• Keep this leaflet. You may need to read it again.
• If you have any further questions, ask your doctor.
• If you get any side effects, talk to your doctor. This includes any possible side ef fects not listed in this leaflet. See section 4.

ℹ️ 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 wor ks 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 life -threatening 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 29 allergic reaction that could be life -threatening 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 react ion. 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, fever -reducing 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 anti -drug 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 preg nant 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 effec ts 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 so dium (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 considere d based on how well you or your child responds to treatment.

30 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 m edicines, 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 (se en 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 discomf ort, 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 med ical 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.

31 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 listed in Appendix 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 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 medicine s 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 sebelip ase 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, an d 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 32 Dublin 15 D15 R925 Ireland For any information about this medicine, please contact the loca l 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.

Tel: +385 1 4628 000 România AstraZeneca Pharma SRL 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 This leaflet was last revised in .

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. ————————————————————————————————————————— The following information is intended for healthcare pr ofessionals only:

In the absence of compatibility studies, this medicinal product must not be mixed with other medicinal products.

Each vial of KANUMA is intended for single use only. KANUMA has to be diluted with sodium chloride 9 mg/ml (0.9%) solution for infusion using aseptic technique.

The diluted solution should be administered to patients using a low -protein binding infusion set equipped with an in -line, low-protein binding 0.2 μm filter, with a surface area of greater than 4.5 cm2 as available in order to avoid filter occlusion.

Preparation of the sebelipase alfa infusion KANUMA should be prepared and used according to the following steps. Aseptic technique should be used. a. The number of vials to be diluted for infusion should be determined ba sed on the patient’s weight and prescribed dose. b. It is recommended to a llow KANUMA vials to reach a temperature between 15 °C and 25 °C prior to dilution to minimise the potential for the formation of sebelipase alfa protein particles in solution . The vial s should not be left outside the refrigerator longer than 24 hours prior to dilution for infusion. The vials should not be frozen, heated or microwaved and should be protected from light. c. The vials should not be shaken. Prior to dilution, the concentrate i n the vials should be inspected visually; the concentrate should be clear to slightly opalescent, colourless to slightly coloured (yellow). Due to the proteinaceous nature of the medicinal product, slight flocculation (e.g., thin translucent fibres) may be present in the vial concentrate and is acceptable for use. d. Do not use if the concentrate is cloudy, or if foreign particulate matter is present. e. Up to 10 ml of concentrate should be slowly withdrawn from each vial and diluted with sodium chloride 9 mg/ml (0.9%) solution for infusion. See Table 1 for recommended total infusion volumes by weight range. The solution should be mixed gently, and not be shaken.

Table 1: Recommended infusion volumes* 34 1 mg/kg dose 3 mg/kg dose 5 mg/kg dose** Weight range (kg) Total infusion volume (ml) Total Infusion Volume (mL) Total Infusion Volume (mL) 1-2.9 4 8 12 3-5.9 6 12 20 6-10.9 10 25 50 11-24.9 25 50 150 25-49.9 50 100 250 50-99.9 100 250 500 100-120.9 250 500 600

• The infusion volume should be based on the prescribed dose and should be prepared to a final sebelipase alfa concentration of 0.1 -1.5 mg/ml.

** For patients with LAL Deficiency presenting within 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 should be disposed of in accordance with local requirements.

📄 Package Leaflet – official source

This article reproduces the information in the official package leaflet, as authorised in the European Union. You can consult the original document here: Product information Kanuma (PDF, ec.europa.eu).

You can check this medicine at any time in the Union Register of medicinal products (EU number EU/1/15/1033).

⚠️ Important: This article is for information purposes only and does not replace medical advice or the package leaflet inside the medicine box. Read the leaflet carefully before use. For any questions about your treatment, talk to your doctor or pharmacist. If you experience side effects, tell your doctor or pharmacist and report them through your national reporting system.