Cerezyme 400 U Powder for concentrate for solution forinfusi - United Kingdom[英国药品]

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Cerezyme 400 U Powder for concentrate for solution forinfusi

2015-03-21 14:17:37 来源: 作者: 【大中小】浏览:420次评论:0条

For doctors

What is it and how is it used?

Cerezyme is used to treat patients who have a confirmed diagnosis of Type I or Type 3 Gaucher disease, who show signs of the disease such as: anaemia (low number of red blood cells), a tendency to bleeding easily (due to low numbers of platelets – a type of blood cell), spleen or liver enlargement or bone disease.

People with Gaucher disease have low levels of an enzyme called acid -glucosidase. This enzyme helps the body control levels of glucosylceramide. Glucosylceramide is a natural substance in the body, made of sugar and fat . In Gaucher disease glucosylceramide levels can get too high.

Cerezyme is an artificial enzyme called imiglucerase -this can replace the natural enzyme acid -glucosidase which 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.

What do you have to consider before using it?

Do not use Cerezyme

Take special care with Cerezyme

Using other medicines
Please tell your doctor or pharmacist if you are taking or have recently taken any other medicines, including medicines obtained without a prescription.

Cerezyme should not be given as a mixture with other medicinal products in the same infusion (drip).

Pregnancy and breast-feeding
Ask your doctor or pharmacist for advice before using this medicine if you are pregnant or think you may be pregnant. Cautious use of Cerezyme during pregnancy and breastfeeding is recommended.

Important information about some of the ingredients of Cerezyme
This medicinal product contains sodium and is administered in 0.9% sodium chloride intravenous solution. To be taken into consideration by patients on a controlled sodium diet.

How is it used?

Instructions for proper use
Cerezyme is given through a drip into a vein (by intravenous infusion).

It is supplied as a powder which will be mixed with sterile water before it is given.

Cerezyme is only used under the supervision of a doctor who is knowledgeable in the treatment of Gaucher disease. Your Doctor may advise that you can be treated at home provided you meet certain criteria. Please contact your Doctor if you would like to be treated at home.

Your dose will be specific to you. Your doctor will work out your dose based on how severe your symptoms are, and other factors. The recommended dose of Cerezyme is 60 units/kg body weight given once every 2 weeks.

Your doctor will keep a close check on your response to your treatment, and may change your dose (up or down) until he/she finds the best dose to control your symptoms.

Once this dose is found your doctor will still keep a check on your responses to make sure you are using the right dose. This might be every 6 to 12 months.

There is no information on the effect of Cerezyme on brain-based symptoms of patients with chronic neuronopathic Gaucher disease. Therefore no special dosage regimen can be recommended.

The ICGG Gaucher Registry
You can ask your doctor to register your patient information into the “ICGG Gaucher Registry”. The aims of this Registry are to increase the understanding of Gaucher disease and to check how well enzyme replacement therapy, like Cerezyme, works. This should lead to improvement in the safe and effective use of Cerezyme. Your patient data will be registered anonymously– nobody will know it is information about you.

If you use more Cerezyme than you should
There are no cases of overdose of Cerezyme reported.

If you forget to use Cerezyme
If you have missed an infusion, please contact your doctor.

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

What are possible side effects?

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

If you experience any serious side effects or side effects not listed below, please tell your doctor immediately.

Common side effects (occurring in more than 1 in 100 patients) are:

Uncommon side effects (occurring in more than 1 in 1000 patients) are:

Some side effects were seen primarily while patients were being given the medicine or shortly after. These have included itching, flushing, hives/localised swelling of the skin or lining of the mouth or throat, chest discomfort, increased heart rate, bluish skin, breathlessness, a sensation of tingling, pricking, burning or numbness of the skin, fall in blood pressure and backache. If you experience any of these symptoms, please tell your doctor immediately. You may need to be given additional medicines to prevent an allergic reaction (e.g. antihistamines and/or corticosteroids).

If any of these side effects gets serious, or if you notice any side effects not listed in this leaflet, please tell your doctor or pharmacist.

How should it be stored?

Keep out of the reach and sight of children.

Do not use Cerezyme after the expiry date printed on the labelling after the letters “EXP”. The expiry date refers to the last date of that month.

Unopened vials Store in a refrigerator 2 C 8 C

Diluted solution:
It is recommended that Cerezyme is used immediately after it has been mixed with sterile water. The mixed solution in the vial cannot be stored and should be promptly diluted in an infusion bag; only the diluted solution can be held for up to 24 hours if it is kept cool (2°C – 8°C) and in the dark.

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.

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For doctors

What is it?

Cerezyme is a powder that is made up into a solution for infusion (a drip into a vein). It contains the active substance imiglucerase.

What is it used for?

Cerezyme is used for the long-term treatment of patients with Gaucher disease. Gaucher disease is a rare inherited disorder, in which people do not have enough of an enzyme called acid beta-glucosidase, which normally breaks down a fatty waste product called glucosylceramide. Without the enzyme, glucosylceramide builds up in the body, typically in the liver, spleen and bone marrow, which produces the symptoms of the disease: anaemia (low red blood cell counts), tiredness, easy bruising and a tendency to bleed, an enlarged spleen and liver, and bone pain and breaks.

Cerezyme is used in patients who have type 1 Gaucher disease, which does not affect the nerve cells, or type 3 Gaucher disease, which progresses slowly and affects the nerve cells. The patients must have symptoms that are not affecting the nervous system, including one or more of the following conditions:

The medicine can only be obtained with a prescription.

How is it used?

Patients with Gaucher disease should be managed by doctors who are knowledgeable about the disease’s treatment. Cerezyme is usually given by infusion every two weeks. The dose and how often the infusions are given need to be adjusted according to each individual patient’s symptoms and response to treatment. The first few infusions should be given slowly, but after these, the speed of infusion can be increased under the supervision of a doctor or nurse. After training, the patient or carer can give the infusion at home, if their doctor believes it is appropriate.

How does it work?

Gaucher disease has previously been treated using an enzyme called alglucerase, which was prepared from human placentas. Imiglucerase, the active substance in Cerezyme, is a copy of this enzyme, which is produced by a method known as ‘recombinant DNA technology’: the enzyme is made by a cell that has received a gene (DNA), which enables it to produce the enzyme. Imiglucerase replaces the missing enzyme in Gaucher disease, helping to break down glucosylceramide and stopping it building up in the body.

How has it been studied?

For type 1 Gaucher disease, Cerezyme has been studied in three studies involving a total of 40 patients. This is an acceptable number because the disease is rare. The studies compared the ability of Cerezyme and alglucerase to control the symptoms of the disease, such as increasing the number of red blood cells and platelets in the blood, and decreasing the size of the liver and spleen.

For type 3 Gaucher disease, which is extremely rare, the company presented data from published articles and from a special register of Gaucher disease patients.

What benefits has it shown during the studies?

The studies have shown that Cerezyme is as safe and effective as alglucerase in controlling the symptoms of Gaucher disease. It has also been shown that patients may safely switch from alglucerase to Cerezyme treatment.

What is the risk associated?

The most common side effects with Cerezyme (seen in between 1 and 10 patients in 100) are dyspnoea (difficulty breathing), coughing, urticaria (hives) or angioedema (swelling beneath the skin), pruritus (itching), rash and hypersensitivity (allergic) reactions. For the full list of all side effects reported with Cerezyme, see the package leaflet. Patients can develop antibodies (proteins that are produced in response to Cerezyme and can affect treatment) and they should be monitored for any allergic reactions to Cerezyme.

Cerezyme should not be used in people who may be hypersensitive (allergic) to imiglucerase or any of the other ingredients.

Why has it been approved?

The CHMP decided that Cerezyme gives effective control of the non-neurological symptoms of types 1 and 3 Gaucher disease. The Committee decided that Cerezyme’s benefits are greater than its risks and recommended that it be given marketing authorisation.

Further information

The European Commission granted a marketing authorisation valid throughout the European Union for Cerezyme to Genzyme Europe B.V. on 17 November 1997. The marketing authorisation is valid for an unlimited period.

For more information about treatment with Cerezyme, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist.

This summary was last updated in 08-2010.

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Name

Cerezyme 400 U Powder for concentrate for solution for
infusion

Composition

Each vial contains 400 units* of imiglucerase**.

After reconstitution, the solution contains 40 units (approximately 1.0 mg) of imiglucerase per ml (400 U/10 ml).

* An enzyme unit (U) is defined as the amount of enzyme that catalyses the hydrolysis of one micromole of the synthetic substrate para-nitrophenyl -D-glucopyranoside (pNP-Glc) per minute at 37°C.
** Imiglucerase is a modified form of human acid -glucosidase and is produced by recombinant DNA technology using a mammalian Chinese Hamster Ovary (CHO) cell culture, with mannose modification for targeting macrophages.

Excipients:
For a full list of excipients, see section 6.1.
This medicinal product contains sodium and is administered in 0.9% sodium chloride intravenous solution (see section 6.6). After reconstitution, the solution contains 1.24 mmol sodium (400 U/10 mL). To be taken into consideration by patients on a controlled sodium diet.