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.

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).

2. What you need to know before KANUMA is given

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.

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