Kanuma 2 mg/ml concentrate for solution for infusion
SpainTable of Contents
Package leaflet: Information for the user
Introduction
Package leaflet: information for the user
KANUMA 2 mg/ml concentrate for solution for infusion
sebelipase alfa (sebelipase alfa)
This medicinal product is subject to additional monitoring, which will facilitate the rapid identification of new safety information. You can help by reporting any adverse reactions you or your child may experience. Information on how to report adverse reactions is included at the end of section 4.
Read all of this leaflet carefully before receiving this medicine, because it contains important information for you.
- Keep this leaflet, as you may need to read it again.
- If you have any questions, ask your doctor.
- If you experience any adverse reactions, consult your doctor, even if they are not listed in this leaflet. See section 4.
Leaflet contents
- What KANUMA is and what it is used for
- What you need to know before receiving KANUMA
- How KANUMA is administered
- Possible adverse effects
- How to store KANUMA
- 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 enzyme naturally produced in the body, 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 disease that causes liver damage, increased blood cholesterol levels, and other complications due to the accumulation of certain types of fats (cholesteryl esters and triglycerides).
How KANUMA works
This medicine is an enzyme replacement therapy, meaning it replaces the defective or missing LAL enzyme in patients with LAL deficiency. It works by reducing the buildup of fat that leads to medical complications, including developmental disorders, liver damage, and cardiac complications. It also improves blood fat concentrations, including elevated levels of LDL cholesterol (bad cholesterol) and triglycerides.
2. What you need to know before receiving KANUMA
You must not receive KANUMA
- If you or your child have experienced potentially life-threatening allergic reactions to sebelipase alfa that cannot be managed when you or your child receive the medicine again, to egg, or to any of the other components of this medicine (listed in section 6).
Warnings and precautions
- If you or your child are being treated with KANUMA, you may experience some adverse effects during the infusion to administer the medicine or in the hours following (see section 4). This is known as an infusion reaction and in some cases may be serious. 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 monitor you or your child for 1 hour to detect any signs of an infusion reaction. Contact a doctor immediately if you or your child experience a serious infusion reaction of this type. If you or your child experience an infusion reaction, you may be given additional medicines to treat or help prevent further reactions. These medicines include antihistamines, fever-reducing medicines and/or corticosteroids (a type of anti-inflammatory).
If the infusion reaction is severe, your doctor may stop the KANUMA infusion and begin appropriate medical treatment for you or your child.
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During treatment, blood proteins against KANUMA may develop, also known as anti-drug antibodies. Consult your doctor if you experience a decrease in effectiveness with KANUMA.
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This medicine may contain egg proteins. If you or your child have a history of egg allergy, you must inform your doctor or nurse (see You must not receive KANUMA).
Use of KANUMA with other medicines
Inform your doctor if you or your child are taking, have recently taken, or might need to take any other medicines.
Pregnancy
There are no data regarding the use of sebelipase alfa in pregnant women. As a precautionary measure, you must not receive KANUMA if you are pregnant.
Breast-feeding
It is unknown whether sebelipase alfa is excreted in human milk. Consult your doctor if you are breast-feeding or intend to breast-feed. Your doctor will then help you decide whether to discontinue breast-feeding or to discontinue treatment with KANUMA, taking into account the benefit of breast-feeding for the child and the benefit of treatment with KANUMA for the mother.
If you are pregnant or breast-feeding, think you may be pregnant or plan to become pregnant, consult your doctor before using this medicine.
Driving and using machines
The influence of KANUMA on the ability to drive and use machines may be minor. Adverse effects of sebelipase alfa include dizziness, which could affect the ability to drive or use machines.
KANUMA contains sodium
This medicine, when diluted with a 9 mg/ml (0.9%) sodium chloride solution for intravenous infusion, contains 33 mg of sodium (the main component of table/cooking salt) at the recommended dose. This corresponds to 1.7% of the maximum daily sodium intake recommended for an adult. Consult your doctor if you or your child are on a low-sodium diet.
3. How KANUMA is administered
The dose you or your child will receive is based on body weight.
Infants (<6 months)
For patients who show signs and symptoms of the disease as infants, the recommended initial dose is 1 mg/kg or 3 mg/kg once weekly. Dose adjustments may be considered depending on how your child responds to treatment.
Children and adults
The recommended dose is 1 mg per kg of body weight once every two weeks via intravenous infusion. Dose adjustment may be considered based on how well you or your child responds to treatment.
Each infusion will take approximately 1–2 hours. Your doctor or nurse may keep you or your child under observation for an additional hour after the infusion. Treatment with KANUMA should be initiated as early as possible and is intended for long-term use.
Your doctor or nurse will administer KANUMA to you or your child by infusion into a vein. The medicine must be diluted before it is given to you or your child.
4. Possible adverse effects
Like all medicines, this medicine may cause adverse effects, although not everyone experiences them.
Adverse effects have been observed in patients during or shortly after administration (infusion reactions). The most serious adverse effects may include an allergic reaction (reported very commonly [may affect more than 1 in 10 people] in infants under 6 months of age, or commonly [may affect up to 1 in 10 people] in children and adults). Symptoms include difficulty breathing, rapid breathing, increased heart rate, chest discomfort, mild eyelid swelling, redness of the eyes, runny nose, flushing, hives, itching, diarrhoea, pallor, wheezing, low blood oxygen levels, skin redness, and irritability. Seek immediate medical advice if you or your child experience symptoms of this type. If you or your child experience an infusion reaction, you may be given additional medicines to treat or help prevent further reactions. If the infusion reaction is severe, your doctor may stop the intravenous infusion of KANUMA and initiate appropriate medical treatment.
Very common adverse effects (may affect more than 1 in 10 people) observed in infants (aged 1 to 6 months):
Hypersensitivity (irritability, restlessness, vomiting, urticaria, eczema, pruritus, pallor, and drug hypersensitivity), severe allergic reactions (anaphylactic reactions)
Eyelid swelling
Increased heart rate
Difficulty breathing
Diarrhoea, vomiting
Rash, rash with raised lesions
Fever
Decreased oxygen in the blood, increased blood pressure, rapid breathing, development of blood proteins
Very common adverse effects (may affect 1 in 10 people or more) observed in children and adolescents (aged 4 to 18 years) and adults:
Hypersensitivity (chills, eczema, laryngeal oedema, nausea, pruritus, and urticaria)
Dizziness
Stomach pain, diarrhoea
Fatigue, fever
Common adverse effects (may affect up to 1 in 10 people) observed in children and adolescents (aged 4 to 18 years) and adults:
Severe allergic reaction (anaphylactic reaction)
Increased heart rate
Skin redness, low blood pressure
Difficulty breathing
Abdominal swelling
Rash, skin redness
Chest discomfort, infusion site reaction
The frequency, type, and intensity of adverse effects in children are similar to those observed in adults.
Reporting of adverse effects
If you or your child experience any kind of adverse effect, consult your doctor, even if it is a possible adverse effect not listed in this leaflet. You may also report them directly through the national reporting system detailed in Appendix V. By reporting adverse effects, you can help provide more information on the safety of this medicine.
5. Storage of KANUMA
Keep this medicine out of the sight and reach of children.
Do not use this medicine after the expiry date stated on the label and carton after EXP/CAD. The expiry date refers to the last day of the month indicated.
Store in a refrigerator (between 2 °C and 8 °C). Do not freeze. Do not shake. Keep in the original packaging to protect from light.
Diluted solutions should be used immediately, if possible. If not used immediately, the diluted solution may be stored for up to 24 hours at a temperature between 2 °C and 8 °C, or up to 12 hours at less than 25 °C.
Medicines must not be disposed of via wastewater or household waste. Ask your pharmacist how to dispose of medicines and packaging no longer required. This will help protect the environment.
6. Contents of the pack and other information
Composition of KANUMA
- The active substance is sebelipase alfa. Each ml of concentrate contains 2 mg of sebelipase alfa. Each vial contains 20 mg of sebelipase alfa in 10 ml of solution.
- The other components are sodium citrate (see section 2, "KANUMA contains sodium"), citric acid monohydrate, human serum albumin, and water for injections.
Appearance of KANUMA and contents of the pack
KANUMA is presented 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 Limited
College Business and Technology Park
Blanchardstown Road North
Dublin 15
D15 R925
Ireland
For further information about this medicinal product, please contact the local representative of the Marketing Authorisation Holder:
Spain
Alexion Pharma Spain, S.L.
Tel: +34 93 272 30 05
Date of the most recent revision of this leaflet:
Other sources of information
Detailed information on this medicinal product is available on the European Medicines Agency website: http://www.ema.europa.eu. Links to other websites on rare diseases and orphan medicinal products are also available.
This 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 must be diluted in a 9 mg/ml (0.9%) sodium chloride solution for infusion using aseptic technique.
The diluted solution must be administered to patients using an infusion set with low protein binding, equipped with an in-line 0.2 µm filter with low protein binding and a filter surface area greater than 4.5 cm², as available, to avoid filter clogging.
Preparation of sebelipase alfa infusion
KANUMA must be prepared and used according to the steps outlined below. Aseptic technique must be used.
- The number of vials to be diluted for infusion will be determined based on the patient's body weight and the prescribed dose.
- It is recommended to allow KANUMA vials to reach a temperature between 15°C and 25°C before dilution to minimize the risk of protein particle formation in the solution. Vials must not be left outside the refrigerator for more than 24 hours prior to dilution for infusion. Vials must not be frozen or heated (including in a microwave) and must be protected from light.
- Do not shake the vials. Prior to dilution, visually inspect the concentrate in the vials; the concentrate should be clear to slightly opalescent and colourless to slightly coloured (yellowish). Due to the protein nature of the medicinal product, fine particles (e.g., thin translucent fibers) may be observed in the concentrate, which is considered acceptable for use.
- Do not use the concentrate if it appears cloudy or contains foreign particulate matter.
- Slowly withdraw up to 10 ml of concentrate from each vial and dilute it in a 9 mg/ml (0.9%) sodium chloride solution for infusion. Recommended total infusion volumes by weight range are shown in Table 1. The solution must be gently mixed without shaking.
Table 1: Recommended infusion volumes* | |||
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) |
1–2.9 | 4 | 8 | 12 |
3–5.9 | 6 | 12 | 20 |
6–10.9 | 10 | 25 | 50 |
11–24.9 | 25 | 50 | 150 |
25–49.9 | 50 | 100 | 250 |
50–99.9 | 100 | 250 | 500 |
100–120.9 | 250 | 500 | 600 |
- The volume of the infusion should be based on the prescribed dose and should be prepared to achieve a final sebelipase alfa concentration of 0.1–1.5 mg/mL.
** For patients with LAL deficiency in the first 6 months of life who do not achieve an optimal clinical response with a dose of 3 mg/kg.
Disposal of unused medicinal product and of all materials that have been in contact with it should be carried out in accordance with local regulations.