Kanuma
sebelipase alfa
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.
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 KANUMA is and what it is used for
What you need to know before you are given KANUMA
How KANUMA is given
Possible side effects
How to store KANUMA
Contents of the pack and other information
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).
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.
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).
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.
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).
Tell your doctor if you or your child are using, have recently used or might use any other medicines.
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.
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.
KANUMA may have a minor influence on the ability to drive and use machines. Adverse effects of sebelipase alfa include dizziness which could affect the ability to drive or use machines.
This medicine, when diluted with sodium chloride 9 mg/mL (0.9%) solution for infusion 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.
The dose you or your child receives is based on your or your child’s body weight.
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.
The recommended dose is 1 mg per kg body weight once every other week through a drip into a vein. Dose adjustments may be considered based on how well the patient responds to treatment.
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.
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, rapid breathing, fast heartbeat, chest discomfort, 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 medical treatment.
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
Hypersensitivity (chills, eczema, laryngeal oedema, nausea, pruritus and urticaria) Dizziness
Stomach ache, diarrhoea Tiredness, fever
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.
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.
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.
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.
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 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
. There are also links to other websites about rare diseases and treatments.
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The following information is intended for healthcare professionals 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.
The number of vials to be diluted for infusion should be determined based on the patient’s weight and prescribed dose.
It is recommended to allow 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 vials 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.
The vials should not be shaken. Prior to dilution, the concentrate in 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.
Do not use if the concentrate is cloudy, or if foreign particulate matter is present.
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.
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) |
1-10 | 10 | 25 | 50 |
11-24 | 25 | 50 | 150 |
25-49 | 50 | 100 | 250 |
50-99 | 100 | 250 | 500 |
100-120 | 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.