CEREZYME 400 units powder for concentrate for infusion solution

Introduction

Package Leaflet:information for the user

Cerezyme 400Units powder for concentrate for solution for infusion

Imiglucerase

Read all of this leaflet carefully before you start using 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 or pharmacist.
• This medicine has been prescribed for you only. Do not pass it on to others. It may harm them, even if their signs of illness are the same as yours.
• If you get any side effects, talk to your doctor or pharmacist. This includes any possible side effects not listed in this leaflet. See section 4.

Contents of the pack

1. What is Cerezyme and what is it used for
2. What you need to know before you are given Cerezyme
3. How Cerezyme is given
4. Possible side effects
5. How to store Cerezyme

1. Contents of the pack and other information

1. What is Cerezyme and what is it used for

Cerezyme contains the active substance imiglucerase and is used to treat patients with confirmed diagnosis of Gaucher disease type I or III who show signs of the disease such as: anaemia (lack of red blood cells), tendency to bleed easily (due to lack of platelets, a type of blood cell), enlargement of the spleen or liver or bone disease (bone disease).

People with Gaucher disease have low levels of an enzyme called acid beta-glucosidase. This enzyme helps the body to control levels of glucocerebroside. Glucocerebroside is a natural substance in the body made up of sugar and fat. When you have Gaucher disease, levels of glucocerebroside can be too high.

Cerezyme is made up of an artificial enzyme called imiglucerase that can replace the natural enzyme acid beta-glucosidase that is lacking or not active enough in patients with Gaucher disease.

The information in this leaflet applies to all patient groups, including children, adolescents, adults and the elderly.

2. What you need to know before you are given Cerezyme

Do not use Cerezyme

• if you are allergic to imiglucerase or any of the other ingredients of this medicine (listed in section 6).

Warnings and precautions

Talk to your doctor or pharmacist before you start using Cerezyme:

• if you are being treated with Cerezyme, you may have an allergic reaction while you are being given the medicine or soon after. If you have this type of reaction, you should tell your doctor immediately. Your doctor will check if you have an allergic reaction to imiglucerase.
  • some patients with Gaucher disease have high blood pressure in the lungs (pulmonary hypertension). The cause may be unknown or due to heart, lung or liver problems. This can happen whether or not you are being treated with Cerezyme. However, if you have difficulty breathing, you should tell your doctor.

Other medicines and Cerezyme

Tell your doctor or pharmacist if you are taking, have recently taken or might take any other medicines.

Cerezyme must not be given mixed with other medicines in the same infusion (drip).

Pregnancy and breast-feeding

If you are pregnant or breast-feeding, think you may be pregnant or are planning to have a baby, ask your doctor or pharmacist for advice before using this medicine.

Cerezyme should be used with caution during pregnancy and breast-feeding.

Cerezyme contains sodium

This medicine contains 41 mg of sodium (main component of cooking/table salt) in each vial. This is equivalent to 2% of the maximum daily intake of sodium recommended for an adult. It is given through an intravenous solution of sodium chloride at 0.9%. This should be taken into account in patients on a low-sodium diet.

3. How Cerezyme is given

Instructions for proper use

Cerezyme is given through a drip connected to a vein (through intravenous infusion).

It is supplied as a powder to be mixed with sterile water before administration.

Cerezyme can only be given under the supervision of a doctor who is familiar with the treatment of Gaucher disease. Your doctor may suggest that you follow the treatment at home if you meet certain criteria. Contact your doctor if you wish to receive treatment at home.

The dose you receive will be specific to you. Your doctor will adjust the dose based on the severity of your symptoms, among other factors. The recommended dose is 60 units/kg body weight given every two weeks.

Your doctor will closely monitor your response to treatment and may change your dose (increase or decrease) until the best dose to control your symptoms is found.

Once this dose is determined, your doctor will check your response to make sure you are using the right dose, probably every 6-12 months.

There is no information on the effect of Cerezyme on the cerebral symptoms of patients with chronic neuropathic Gaucher disease. Therefore, a special dosage regimen cannot be recommended.

ICGG Gaucher Registry

Your doctor may ask you to register your patient data in the "ICGG Gaucher Registry" (ICGG: International Collaborative Gaucher Group). The purpose of this registry is to expand knowledge about Gaucher disease and to check the functioning of enzyme replacement therapy, such as Cerezyme. This should lead to improved safety and efficacy of the use of Cerezyme. Your patient data will be recorded anonymously: no one will know that it is your data.

If you use more Cerezyme than you should

No cases of overdose with Cerezyme have been reported.

If you miss a dose of Cerezyme

If you miss a dose for any reason, contact your doctor.

If you have any other questions about the use of this product, ask your doctor or pharmacist.

4. Possible side effects

Like all medicines, this medicine can cause side effects, although not everybody gets them.

Common(may affect up to 1 in 10 people):

• difficulty breathing
• cough
• urticaria/localised swelling of the skin or mucous membranes of the mouth or throat
• pruritus (itching)
• rash

Uncommon(may affect up to 1 in 100 people):

• dizziness
• headache
• tingling, itching, burning or numbness of the skin
• increased heart rate
• cyanosis (blue discoloration) of the skin
• flushing
• decreased blood pressure
• vomiting
• nausea
• abdominal colic pain
• diarrhoea
• arthralgia (joint pain)
• discomfort at the infusion site
• burning sensation at the infusion site
• swelling at the infusion site
• sterile abscess at the injection site
• chest discomfort
• fever
• chills
• fatigue
• back pain

Rare(may affect up to 1 in 1,000 people):

• anaphylactoid reactions

Some side effects have been seen mainly while patients were receiving the medicine or soon after. These include itching, flushing, urticaria/localised swelling of the skin or mucous membranes of the mouth or throat, chest discomfort, increased heart rate, cyanosis of the skin, difficulty breathing, tingling, itching, burning or numbness of the skin, decreased blood pressure and back pain. If you experience any of these symptoms, tell your doctor immediately. You may need other medicines to prevent an allergic reaction (e.g. antihistamines and/or corticosteroids).

Reporting of side effects

If you get any side effects, talk to your doctor or pharmacist. 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.

5. How to store Cerezyme

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 carton after EXP. The expiry date refers to the last day of the month stated.

Unopened vials: Store in a refrigerator (2°C - 8°C).

Diluted solution:

Cerezyme should be used immediately after mixing with sterile water. The mixed solution in the vial cannot be stored and must be diluted immediately in an infusion bag; the diluted solution can be stored for up to 24 hours if kept cool (2-8°C) and protected from light.

Medicines should not be disposed of via wastewater or household waste. Ask your pharmacist how to dispose of medicines no longer required. These measures will help to protect the environment.

6. Contents of the pack and other information

What is in Cerezyme

• The active substance is imiglucerase. Imiglucerase is a modified form of the human enzyme acid beta-glucosidase produced by recombinant DNA technology. Each vial contains 400 units of imiglucerase. After reconstitution, the solution contains 40 units of imiglucerase per millilitre.
• The other ingredients are: mannitol, sodium citrate, citric acid monohydrate and polysorbate 80.

Appearance of Cerezyme and pack contents

Cerezyme 400 Units is presented as a powder for concentrate for solution for infusion (in a vial – pack size of 1, 5 or 25 vials). Not all pack sizes may be marketed.

Cerezyme is supplied as a white or off-white powder. After reconstitution, it appears as a clear and colourless solution. The reconstituted solution should be further diluted.

Marketing Authorisation Holder and Manufacturer

Marketing Authorisation Holder

Sanofi B.V., Paasheuvelweg 25, 1105 BP Amsterdam, The Netherlands

Manufacturer

Genzyme Ireland Limited, IDA Industrial Park, Old Kilmeaden Road, Waterford, Ireland

You can get more information on this medicine by contacting the local representative of the Marketing Authorisation Holder:

Date of last revision of this leaflet:

Detailed information on this medicine is available on the European Medicines Agency web site: http://www.ema.europa.eu/. It also provides links to other web sites on rare diseases and their treatments.

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This information is intended only for healthcare professionals:

Instructions for use: reconstitution, dilution and administration

Each vial of Cerezyme is for single use. After reconstitution, each vial of Cerezyme contains 400 units of imiglucerase in 10.0 ml (40 units per ml).

Determine the number of vials to reconstitute based on the individual patient's dosage, and remove the vials from the refrigerator.

Use aseptic technique

Reconstitution

Reconstitute each vial with 10.2 ml of water for injections, avoiding vigorous impact of the water for injection on the powder and the formation of foam in the solution, mixing gently. The reconstituted volume is 10.6 ml. The pH of the reconstituted solution is approximately 6.2.

After reconstitution, the solution appears as a clear and colourless solution without any visible particles. The reconstituted solution should be further diluted. Before any further dilution, visually inspect the diluted solution in each vial for any visible particles or colour change. Do not use vials that contain visible particles or have changed colour.

After reconstitution, dilute the vials promptly and do not store them for later use.

Dilution

The reconstituted solution contains 40 units of imiglucerase per ml. The reconstituted volume allows for the exact withdrawal of 10.0 ml (equal to 400 units) from each vial. Withdraw 10.0 ml of the reconstituted solution from each vial, combine the withdrawn volumes and further dilute to a total volume of 100 to 200 ml with sodium chloride 0.9% intravenous solution. Mix the infusion solution gently.

Administration

It is recommended to administer the diluted solution through an in-line filter with a pore size of 0.2 µm and low protein binding to remove any particulate matter. This will not cause a decrease in imiglucerase activity. It is recommended to administer the diluted solution within 3 hours. The diluted product in sodium chloride 0.9% solution may retain its chemical stability for up to 24 hours if stored at 2-8°C and protected from light, but microbiological safety will depend on whether reconstitution and dilution are performed aseptically.

Cerezyme does not contain preservatives. Disposal of unused medicine and all materials that have been in contact with it should be done in accordance with local regulations.