Prospect: information for the user

KANUMA 2mg/ml concentrate for solution for infusion

sebelipasa alfa (sebelipasa alfa)

This medicine is subject to additional monitoring, which will speed up the detection of new information about its safety. You can contribute by reporting any adverse effects that you or your child may experience. The final part of section4 includes information on how tocommunicate these adverse effects.

Read this prospect carefully before receiving this medicine, because it contains important information for you.

• Keep this prospect, as you may need to read it again.
• If you have any doubts, consult your doctor.
• If you experience adverse effects, consult your doctor even if they do not appear in this prospect. See section4.

1.What is KANUMA and for what it is used

2.What you need to know before receiving KANUMA

3.How KANUMA is administered

4.Possible adverse effects

5.Storage of KANUMA

6.Contents of the package and additional information

KANUMA contains the active ingredient sebelipasa alfa. Sebelipasa alfa is similar to the naturally produced enzyme, lysosomal acid lipase (LAL), which the body uses to break down fats. It is used to treat patients of all ages with lysosomal acid lipase deficiency (LAL deficiency).

LAL deficiency is a genetic disorder that causes liver damage, elevated blood cholesterol levels, and other complications due to the accumulation of certain types of fats (cholesterol esters and triglycerides).

How KANUMA works

This medication is an enzyme replacement therapy, which means it replaces the defective or absent LAL enzyme in patients with LAL deficiency. It acts by reducing the accumulation of fat that causes medical complications, including developmental disorders, liver damage, and cardiac complications. It also improves blood lipid concentrations, including elevated levels of low-density lipoprotein (LDL) cholesterol (bad cholesterol) and triglycerides.

You should not receive Kanuma

• If you or your child have experienced potentially fatal allergic reactions to sebelipasa alfa that cannot be treated when you or your child receive the medication again, to eggs or to any of the other components of this medication (including in section6).

Warnings and precautions

• If you or your child receive treatment with KANUMA, you may experience some adverse effect during the infusion to administer the medication or in the hours that follow (see section4). This is known as infusion reaction and in some cases can be severe. It may include an allergic reaction that could be fatal and require medical treatment. The first time you or your child receive KANUMA, a healthcare professional will keep you under observation for 1hour to detect any signs of an infusion reaction.Consult a doctor immediately if you or your child experience a severe infusion reaction of this type.If you or your child experience an infusion reaction, you may be given additional medications to treat or help prevent other reactions. These medications include antihistamines, fever reducers, and/or corticosteroids (a type of anti-inflammatory).

In the event of a severe infusion reaction, your doctor may stop the infusion of KANUMA and begin administering appropriate medical treatment to you or your child.

• During treatment, antibodies against KANUMA, also known as anti-drug antibodies, may develop in your blood. Consult your doctor if you experience a decrease in the effectiveness of KANUMA.

• This medication may contain egg proteins. If you or your child have a history of allergy to eggs, you must inform your doctor or nurse (SeeYou should not receive KANUMA).

Use of KANUMA with other medications

Inform your doctor if you or your childaretaking, have taken recently, or may need to take any other medication.

Pregnancy

There are no data on the use ofsebelipasa alfa in pregnant women. As a precaution, you should not receive KANUMA if you are pregnant.

Breastfeeding

The excretion of sebelipasa alfa in breast milk is unknown. Consult your doctor if you are breastfeeding or intend to do so.Your doctor will then help you decide whether to stop breastfeeding or stop treatment withKANUMAconsidering the benefits of breastfeeding for the child and the benefits of treatment withKANUMAfor the mother.

If you are pregnant or breastfeeding, think you may be pregnant, or intend to become pregnant, consult your doctor before using this medication.

Driving and operating machines

The influence of KANUMA on the ability to drive and operate machines may be small. The adverse effects of sebelipasa alfa include dizziness that may affect the ability to drive or operate machines.

KANUMA contains sodium

This medication, when diluted with a solution for infusion for intravenous administration of sodium chloride 9mg/ml (0.9%) contains 33mg of sodium (main component of table salt/for cooking) at the recommended dose. This is equivalent to 1.7% of the maximum daily sodium intake recommended for an adult. Consult your doctor if you or your child are following a low-sodium diet.

The dose you or your child will receive will be based on your body weight.

Infants (<6 months)

For patients who present signs and symptoms of the disease when infants, the recommended initial dose is 1 mg/kg or 3 mg/kg once a week. Dose adjustments may be considered based on your child's response to treatment.

Children and adults

The recommended dose is 1 mg per kg of body weight once every two weeks through an intravenous infusion system. Dose adjustments may be considered based on how well you or your child responds to treatment.

Each infusion will take approximately 1-2 hours. You or your child may be kept under observation for an additional hour after the infusion. KANUMA treatment should be initiated as early as possible and is designed for long-term use.

Your doctor or nurse will administer KANUMA to you or your child through an intravenous infusion in a vein. The medication must be diluted before it is administered to you or your child.

Like all medicines, this medicine can cause side effects, although not everyone will experience them.

Side effects have been observed in patients during administration or shortly after this (perfusion reactions). The most serious side effects may include an allergic reaction (observed very frequently [may affect more than 1 in 10 people] in infants under 6 months or frequently [may affect up to 1 in 10 people] in children and adults). The symptoms include difficulty breathing, rapid breathing, rapid heart rate, chest discomfort, mild eyelid inflammation, eye redness, runny nose, flushing, urticaria, itching, diarrhea, pallor, wheezing, low blood oxygen level, skin redness, and irritability.Consult a doctor immediately if you or your child experience symptoms of this type.If you or your child experience a perfusion reaction, you may be administered additional medications to treat or help prevent other reactions. In the event of a severe perfusion reaction, your doctor may stop the intravenous infusion of KANUMA and begin administering appropriate medical treatment.

Very common side effects (may affect more than 1 in 10 people) observed in infants (1 to 6 months of age):

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

Eyelid inflammation

Rapid heart rate

Difficulty breathing

Diarrhea, vomiting

Urticaria, urticaria with elevated lesions

Fever

Low blood oxygen level, increased blood pressure, rapid breathing, development of blood proteins

Very common side effects (may affect 1 in 10 people or more) observed in children and adolescents (4 to 18 years of age) and adults:

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

Dizziness

Abdominal pain, diarrhea

Fatigue, fever

Common side effects (may affect up to 1 in 10 people) observed in children and adolescents (4 to 18 years of age) and adults:

Severe allergic reaction (anaphylactic reaction)

Rapid heart rate

Skin redness, low blood pressure

Difficulty breathing

Abdominal distension

Urticaria, skin redness

Chest discomfort, reaction at the infusion site

The frequency, type, and intensity of side effects in children are similar to those observed in adults.

Reporting of side effects

If you or your child experience any type of side effect, consult your doctor, even if it is a possible side effect that does not appear in this prospectus. You can also report them directly through thenational notification system included in theAppendix V. By reporting side effects, you can contribute to providing more information on the safety of this medicine.

Keep this medication out of the sight and reach of children.

Do not use this medication after the expiration date that appears on the label and the box after EXP/CAD. The expiration date is the last day of the month indicated.

Store in refrigerator (between 2°C and 8°C). Do not freeze. Do not shake. Store in the original packaging to protect it from light.

It is recommended to use immediately the diluted solutions. If not used immediately, the diluted solution can be stored for a maximum of 24 hours at a temperature between 2°C and 8°C, or up to 12 hours at less than 25°C.

Medicines should not be thrown down the drains or in the trash. Ask your pharmacist how to dispose of the containers and the medicines that you no longer need. In this way, you will help to protect the environment.

Composition of KANUMA

• The active ingredient is sebelipase alfa. Each milliliter of concentrate contains 2mg of sebelipase alfa. Each vial contains 20mg of sebelipase alfa in 10ml of solution.
• The other components are sodium citrate (see the section «KANUMA contains sodium» in section2), citric acid monohydrate, human serum albumin, and water for injection.

Appearance of KANUMA and packaging contents

KANUMA is presented as a concentrate for solution for infusion (sterile concentrate). It is a clear to slightly opalescent, colorless to slightly colored solution.

Packaging size: 1vial with 10ml of concentrate.

Marketing authorization holder and manufacturer

Marketing authorization 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 InternationalOperations Limited

College Business and Technology Park

Blanchardstown Road North

Dublin 15

D15 R925

Ireland

For more information about this medicine, please contact the local representative of the marketing authorization holder:

Spain

Alexion Pharma Spain, S.L.

Phone: +34 93 272 30 05

Last review date of this leaflet:

Other sources of information

The detailed information about this medicine is available on the website of the European Medicines Agency:http://www.ema.europa.eu.There are also links to other websites about rare diseases and orphan medicines.

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This information is intended solely for healthcare professionals:

In the absence of compatibility studies, this medicine should not be mixed with other medicines.

Each vial of KANUMA is designed for single use only. KANUMA should be diluted in a sodium chloride 9mg/ml (0.9%) infusion solution using an aseptic technique.

The diluted solution should be administered to patients using an infusion device with low protein binding that has an in-line filter with a pore size of 0.2µm with low protein binding, and a surface area of at least 4.5cm2as available to prevent filter occlusion.

Preparation of sebelipase alfa infusion

KANUMA should be prepared and used following the steps outlined below. An aseptic technique should be used.

1. The number of vials to be diluted for the infusion should be determined based on the patient's weight and the prescribed dose.
2. It is recommended that the vials of KANUMA be allowed to reach a temperature of between 15°C and 25°C before dilution to minimize the possibility of sebelipase alfa protein particles forming in the solution. Vials should not be left out of the refrigerator for more than 24hours before infusion. Vials should not be frozen or heated (including microwave heating) and should be protected from light.
3. Vials should not be agitated. Before dilution, the concentrate in the vials should be visually inspected; the concentrate should be clear to slightly opalescent, colorless to slightly colored (yellowish). Due to the proteinaceous nature of the drug, it is possible to observe fine particles (e.g., thin translucent fibers) in the concentrate of the vial, which is considered acceptable for use.
4. The concentrate should not be used if it appears turbid or contains foreign matter.
5. Slowly withdraw 10ml of concentrate from each vial and dilute in a sodium chloride 9mg/ml (0.9%) infusion solution. The volumes of infusion recommended for each weight interval are shown in Table1. The solution should be mixed carefully without agitation.

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

** For patients with LAL deficiency in the first 6months of life who do not achieve optimal clinical response with a dose of 3mg/kg.

Disposal of unused medication and all materials that have come into contact with it should be carried out in accordance with local regulations.