What is it and how is it used?

Fabrazyme is used as enzyme replacement therapy in Fabry disease, where the level of ?-galactosidase enzyme activity is absent or lower than normal. If you suffer from Fabry disease a fat substance, called globotriaosylceramide (GL-3), is not removed from the cells of your body and starts to accumulate in the walls of the blood vessels of your organs.

Fabrazyme is indicated for use as long-term enzyme replacement therapy in patients with a confirmed diagnosis of Fabry disease.

Table of Contents
What do you have to consider before using it?
How is it used?
What are possible side effects?
How should it be stored?
Further information

What do you have to consider before using it?

Do not use Fabrazyme
If you have experienced an allergic anaphylactic reaction to agalsidase beta or if you are allergic (hypersensitive) to any of the other ingredients of Fabrazyme.

Take special care with Fabrazyme

If you are treated with Fabrazyme, you may develop infusion associated reactions. An infusion-associated reaction is any side effect occurring during the infusion or until the end of the infusion day (See 4 ?Possible Side Effects?). If you experience a reaction like this, you should tell your doctor immediately. You may need to be given additional medicines to prevent such reactions from occurring.

Different groups of patients using Fabrazyme

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

Using other medicines
There are no known interactions with other medicinal products. Fabrazyme should not be administered with chloroquine, amiodarone, benoquin or gentamicin due to a theoretical risk of decreased agalsidase beta activity. Please tell your doctor or pharmacist if you are taking or have recently taken any other medicines, including medicines obtained without a prescription.

Using Fabrazyme with food and drink
Interactions with food and drink are unlikely.

Pregnancy and breast-feeding

Use of Fabrazyme during pregnancy is not recommended. There is no experience with the use of Fabrazyme in pregnant women. Fabrazyme may get into breast milk. Use of Fabrazyme during breast-feeding is not recommended. Ask your doctor or pharmacist for advice before taking this medicine.

How is it used?

Fabrazyme 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 (see information for Health Care Professionals)

Fabrazyme is only used under the supervision of a doctor who is knowledgeable in the treatment of Fabry disease.

The recommended dose of Fabrazyme for adults and children 8 ? 16 years is 1 mg/kg body weight, once every 2 weeks. No changes in dose are necessary for patients with kidney disease.

If you use more Fabrazyme than you should

There are no cases of overdose of Fabrazyme reported. Doses up to 3 mg/kg body weight have shown to be safe.

If you forget to use Fabrazyme

If you have missed an infusion of Fabrazyme, please contact your doctor.

What are possible side effects?

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

In clinical studies side effects were mainly seen while patients were being given the medicine or shortly after. If you experience any serious side effect or side effects not listed, please tell your doctor immediately.

In clinical trials the following side effects were reported:

Very common (occurring in more than 1 in 10 patients):

  • chills ? abnormal touch feeling (pins ? feeling cold and needles)
  • fever ? nausea
  • headache ? vomiting

Common (occurring in 1 in 100 to 1 in 10 patients):

  • chest pain ? sleepiness ? fatigue
  • difficulty in breathing ? increased heart beat ? flushing
  • pallor ? abdominal pain ? pain
  • itching ? back pain ? throat tightness
  • abnormal tear secretion ? rash ? dizziness
  • feeling weak ? low heart rate ? palpitations
  • tinnitus ? lethargy ? decreased sensitivity to pain
  • nasal congestion ? syncope ? burning sensation
  • diarrhoea ? cough ? wheezing
  • redness ? abdominal discomfort ? urticaria
  • muscle pain ? swelling face ? pain at the extremities
  • increased blood pressure ? joint pain ? nasopharyngitis
  • sudden swelling of the face ? decreased blood pressure ? hot flush or throat
  • oedema in extremities ? chest discomfort ? feeling hot
  • vertigo ? face oedema ? hyperthermia
  • stomach discomfort ? exacerbated difficulty in ? decreased mouth sensitivity breathing
  • muscle spasms ? muscle tightness ? musculoskeletal stiffness

Uncommon (occurring in 1 in 1000 to 1 in 100 patients):

  • tremor ? itching eyes ? low heart rate due to conduction disturbances
  • red eyes ? ear swelling ? increased sensitivity to pain
  • ear pain ? bronchospasm ? upper respiratory tract congestion
  • throat pain ? runny nose ? red rash
  • fast breathing ? heart burn ? (mottled purplish) skin discoloration
  • itchy rash ? skin discomfort ? coldness of the extremities
  • feeling hot and cold ? musculoskeletal pain ? injection site blood clotting
  • difficulty swallowing ? rhinitis ? skin discoloration
  • infusion site pain ? influenza-like illness ? oedema
  • infusion site reaction ? malaise

Unknown frequency

  • Serious allergic reactions ? serious inflammation of the ? lower blood oxygen levels vessels

If any of the 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.

Unopened vials Store in a refrigerator (2 °C ? 8 °C).
Do not use Fabrazyme after the expiry date which is stated on the labelling after the letters ?EXP?.

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.

Further information

What Fabrazyme contains

  • The active substance is agalsidase beta, one vial contains 5 mg.
  • Other ingredients are:
  • Mannitol
  • Sodium phosphate monobasic, monohydrate
  • Sodium phosphate dibasic, heptahydrate.

What Fabrazyme looks like and contents of the pack

Fabrazyme is supplied as a white to off-white powder. After reconstitution it is a clear, colourless liquid, free from foreign matter. The reconstituted solution must be further diluted. Package sizes: 1, 5 and 10 vials per carton. Not all pack sizes may be marketed.

Marketing Authorisation Holder and Manufacturer

Marketing authorisation holder

Genzyme Europe B.V., Gooimeer 10, NL-1411DD Naarden, The Netherlands.

Manufacturer

Genzyme Ltd., 37 Hollands Road, Haverhill, Suffolk CB9 8PU, United Kingdom.

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

ItaliaMalta Genzyme Srl ItaliaItalja, Tel 39 059 349811 BelgiëBelgiqueBelgien LuxemburgLuxembourg Genzyme Belgium N.V., TelTél 32 2 714 17 11

Magyarország Genzyme Europe B.V. Képviselet Tel 36 1 310 7440 Genzyme CEE GmbH . 359 2 971 1001

Nederland Genzyme Europe B.V., Tel 31 35 6991200 eská RepublikaSlovenská Republika Slovenija Genzyme Czech s.r.o., Tel 420 227 133 665

Österreich Genzyme Austria GmbH, Tel 43 1 774 65 38 DanmarkNorgeSverigeSuomiFinland Ísland Genzyme AS, DanmarkTanskaDanmörk, TlfPuh.Sími 45 32712600

Deutschland Genzyme GmbH, Tel 49 610236740 PolskaEestiLatvijaLietuva Genzyme Polska Sp. z o. o. PoolaPolijaLenkija, Tel 48 22 24 60 900

Genzyme Hellas Ltd. 30 210 99 49 270 Portugal Genzyme Portugal, S.A., Tel 351 21 422 0100

España Genzyme, S.L., Tel 34 91 6591670 România Genzyme CEE GmbH- Reprezentana pentru România Tel 40 21 243 42 28

France Genzyme S.A.S, Tél 33 0 825 825 863 United KingdomIreland Genzyme Therapeutics Ltd. United Kingdom, Tel 44 1865 405200

This leaflet was last approved in

Detailed information on this medicine is available on the European Medicines Agency (EMEA) web site: http://www.emea.europa.eu. There are also links to other websites about rare diseases and treatments.

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The following information is intended for medical or healthcare professionals only:

Instructions for use ? reconstitution, dilution and administration

The powder for concentrate for solution for infusion has to be reconstituted with water for injections, diluted with 0.9% sodium chloride intravenous solution and then administered by intravenous infusion.

From a microbiological point of view, the product should be used immediately. If not used immediately, in-use storage and conditions are the responsibility of the user. The reconstituted solution cannot be stored and should be promptly diluted; only the diluted solution can be held for up to 24 hours at 2ºC -8º C.

Use Aseptic Technique

1. Determine the number of vials to be reconstituted based on the individual patient's weight and remove the required vials from the refrigerator in order to allow them to reach room temperature (in approximately 30 minutes). Each vial of Fabrazyme is intended for single use only.

Reconstitution

2. Reconstitute each vial of Fabrazyme 5 mg with 1.1 ml water for injections. Avoid forceful impact of the water for injections on the powder and avoid foaming. This is done by slow drop-wise addition of the water for injection down the inside of the vial and not directly onto the lyophilized cake. Roll and tilt each vial gently. Do not invert, swirl or shake the vial.

3. The reconstituted solution contains 5 mg agalsidase beta per ml, and appears as a clear colourless solution. The pH of the reconstituted solution is approximately 7.0. Before further dilution, visually inspect the reconstituted solution in each vial for particulate matter and discoloration. Do not use the solution if foreign particles are observed or if the solution is discoloured.

4. After reconstitution it is recommended to promptly dilute the vials, to minimize protein particle formation over time.

5. Any unused product or waste material should be disposed of in accordance with local requirements.

Dilution

6. Prior to adding the reconstituted volume of Fabrazyme required for the patient dose, it is recommended to remove an equal volume of 0.9% sodium chloride intravenous solution, from the infusion bag.

7. Remove the airspace within the infusion bag to minimize the air/liquid interface.

8. Slowly, withdraw 1.0 ml (equal to 5 mg) of the reconstituted solution from each vial up to the total volume required for the patient dose. Do not use filter needles and avoid foaming.

9. Then slowly inject the reconstituted solution directly into the 0.9% sodium chloride intravenoussolution (not in any remaining airspace) to a final concentration between 0.05 mg/ml and 0.7 mg/ml. Determine the total volume of sodium chloride 0.9% solution for infusion (between 50 and 500 ml) based on the individual dose. For doses lower than 35 mg use a minimum of 50 ml, for doses 35 to 70 mg use a minimum of 100 ml, for doses 70 to 100 mg use a minimum of 250 ml and for doses greater than 100 mg use only 500 ml. Gently invert or lightly massage the infusion bag to mix the diluted solution. Do not shake or excessively agitate the infusion bag.

Administration

10. It is recommended to administer the diluted solution through an in-line low protein-binding 0.2 µm filter to remove any protein particles which will not lead to any loss of agalsidase beta activity. The initial infusion rate should be no more than 0.25 mg/min (15 mg/hour) to minimise the potential occurrence of infusion-associated reactions. After patient tolerance is established, the infusion rate may be increased gradually with subsequent infusions.

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