Fabrazyme 5 mg powder for concentrate for solution for infusion

Package leaflet: Information for the user

Fabrazyme^® 5 mg powder for concentrate for solution for infusion

Agalsidase beta

1. What Fabrazyme is and what it is used for

Fabrazyme contains the active substance agalsidase beta and is used as enzyme replacement therapy in Fabry disease, where the level of α-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.

Fabrazyme is indicated in adults, children and adolescents aged 8 years and older.

2. What you need to know before you use Fabrazyme

3. How to use Fabrazyme

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 at the end of this leaflet).

Always use this medicine exactly as your doctor has told you. Check with your doctor if you are not sure.

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

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

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

4. Possible side effects

Like all medicines, this medicine 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 (“infusion related reactions”). Severe life-threatening allergic reactions (“anaphylactoid reactions”) have been reported in some patients. If you experience any serious side effect, you should contact your doctor immediately.

Very common symptoms (may affect more than 1 in 10 people) include chills, fever, feeling cold, nausea, vomiting, headache and abnormal feelings in the skin such as burning or tingling. Your doctor may decide to lower the infusion rate or give you additional medicines to prevent such reactions from occurring.

List of other side effects:

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

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

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

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

Not known (frequency cannot be estimated from the available data):

• lower blood oxygen levels
• serious inflammation of the vessels

In some patients initially treated at the recommended dose, and whose dose was later reduced for an extended period, some symptoms of Fabry disease were reported more frequently.

5. How to store Fabrazyme

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 label after ‘EXP’. The expiry date refers to the last day of that month.

Unopened vials

Store in a refrigerator (2°C – 8°C).

Reconstituted and diluted solutions

The reconstituted solution cannot be stored and should be promptly diluted. The diluted solution can be held for up to 24 hours at 2°C – 8°C.

Do not throw away any medicines via wastewater or household waste. Ask your pharmacist how to throw away medicines you no longer use. These measures will help protect the environment.

6. Contents of the pack and other information

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