What is a Patient Information Leaflet and why is it useful?

The Patient Information Leaflet (PIL) is the leaflet included in the pack with a medicine. It is written for patients and gives information about taking or using a medicine. It is possible that the leaflet in your medicine pack may differ from this version because it may have been updated since your medicine was packaged.

Black triangle. This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information.

Below is a text only representation of the Patient Information Leaflet. The original can be viewed in PDF format using the link above.

The text only version may be available from RNIB in large print, Braille or audio CD. For further information call RNIB Medicine Leaflet Line on 0800 198 5000. The product code(s) for this leaflet is: EU/1/15/1033/001 .

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. 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 and breast-feeding

You should not be given KANUMA if you are pregnant unless clearly necessary. It is not known whether sebelipase alfa passes into breast milk; therefore it is recommended that you stop breast-feeding or using KANUMA treatment while breast-feeding.

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

Each 10 ml vial contains 33 mg sodium. 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. The recommended dose is 1 mg per kg body weight once every other week through a drip into a vein. For patients who have signs and symptoms of the disease when they are infants, the recommended starting dose is 1 mg/kg once weekly. 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. Dose adjustments may be considered based on how well you or your child responds to treatment. 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 side effects reported in infants (1 to 6 months old) are:

Eyelid swelling

Agitation

Irritability

Decreased muscle tone

Fast heartbeat

High blood pressure

Paleness

Difficulty breathing

Wheezing

Cough

Stuffy or swollen nose

Sneezing

Diarrhoea

Heartburn (reflux diseases)

Dry heaving

Vomiting

Itching

Hives

Rash

Raised rash

Red swollen skin

Chills

Fever

Swelling

Rapid breathing

Not enough oxygen in the blood

Common side effects reported in children and adolescents (4 to 18 years old) and adults are:

Infection of the urinary system

Severe allergic reaction (anaphylactic reaction)

Swelling of the eyelids

Temporary increased cholesterol or triglyceride (fats) levels in the blood

Fast heartbeat

Anxiety

Sleeplessness

Dizziness

Low blood pressure

Redness in face

Shortness of breath

Swelling of the throat

Diarrhoea

Stomach ache

Stomach bloating

Nausea

Hives

Rash

Itching

Red swollen skin

Increased menstrual bleeding

Shivers

Chest discomfort

Swelling

Tiredness

Hardened area around the infusion site

Fever

Frequency, type and severity of adverse reactions in children are the same as in adults.

Reporting of side effects

If you or your child gets 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: www.mhra.gov.uk/yellowcard 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
e-mail: medsafety@hpra.ie

Adverse events should also be reported to

Alexion Pharma UK Ltd
e-mail: uk.adverseevents@alexion.com
Freephone (UK): 0800 321 3902
Freephone (Ireland): 1 800 936 544

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 (2 mg/ml).
  • The other ingredients are trisodium citrate dihydrate (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. It is a solution that is clear to slightly opalescent, and colourless to slightly coloured.

Pack sizes: 1 vial containing 10 ml of concentrate.

Marketing Authorisation Holder and Manufacturer

Marketing Authorisation Holder

Alexion Europe SAS
1-15, avenue Edouard Belin
92500 Rueil-Malmaison
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 June 2018.

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.