KANUMA 2mg/ml concentrate solution

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.

  • 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 effects not listed in this leaflet. See section 4.

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

  • 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 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 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, 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.
  • 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 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

Yellow Card Scheme
Website: https://yellowcard.mhra.gov.uk/

or search for MHRA Yellow Card in the Google Play or Apple App Store.

Ireland:

HPRA Pharmacovigilance
Earlsfort Terrace
IRL - Dublin 2
Tel: +353 1 6764971
Fax: +353 1 6762517
Website: www.hpra.ie

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 Unlimited Company
College Business and Technology Park
Blanchardstown
Dublin 15
Ireland

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.

Company Contact Details
Alexion Pharma UK Ltd
Address

3 Furzeground Way, Stockley Park, Uxbridge, Middlesex, UB111EZ, UK

Fax

+44 (0) 20 8744 6630

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Telephone

+44 (0) 208 744 6600

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0800 028 4394

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