KANUMA 2mg/ml concentrate solution

• 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.

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

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.

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 you or your child 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.

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 Yellow Card Scheme Website: www.mhra.gov.uk/yellowcard or search for MHRA Yellow Card in the Google Play or Apple App Store.

By reporting side effects you can help provide more information on the safety of this medicine.

• 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

Manufacturer:

For any information about this medicine, please contact the local representative of the Marketing Authorisation Holder: