5 mg powder for concentrate for solution for infusion. 
agalsidas 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 enzyme activity of α-galactosidase is absent or lower than normal. If you suffer from Fabry’s disease, a fatty substance, called globotriaosylceramide (GL-3), is not removed from the cells of your body and instead accumulates in the blood vessel walls of your organs.

Fabrazyme is intended as a long-term enzyme replacement therapy in patients with a diagnosis of Fabry disease.

Fabrazyme is intended for adults, children, and adolescents 8 years of age and older.

2. What you need to know before you use Fabrazyme

Do not use Fabrazyme

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

Warnings and cautions

Talk to your doctor or pharmacist before using Fabrazyme.

If you are being treated with Fabrazyme, you may experience infusion-related reactions. An infusion-related reaction is a side effect that occurs during infusion one or until the end of the infusion day (see section 4). If you get such a reaction, you must contact a doctor immediately. You may need additional medicines to prevent such a reaction (eg antihistamines or paracetamol ).

Children and young people

No clinical studies have been performed in children 0 – 4 years of age. The risks and benefits of Fabrazyme in children aged 5 to 7 years have not yet been established and no dose can therefore be recommended for this age group.

Other medicines and Fabrazyme

Tell your doctor or pharmacist if you are taking, have recently taken, or might take any other medicines. 
Tell your doctor if you are taking medicines containing chloroquine, amiodarone, benzoquinone, or gentamicin. There is a theoretical risk of reduced agalsidase beta activity

Pregnancy, breastfeeding, and fertility

The use of Fabrazyme during pregnancy is not recommended. There is no experience with the use of Fabrazyme in pregnant women. Fabrazyme may be excreted in human milk. The use of Fabrazyme during breast-feeding is not recommended. Studies have not been performed to investigate the effect of Fabrazyme on fertility.

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 taking this medicine.

Driving and using machines

Do not drive or use any tools or machines if you feel dizzy, sleepy, faint, or dizzy during or shortly after administering one of Fabrazyme (see section 4). Consult a doctor first.

Fabrazyme contains sodium

This medicine contains less than 1 mmol sodium (23 mg) per vial, ie essentially ‘sodium-free’.

3. How to use Fabrazyme

Fabrazyme is given by a drip into a vein (by intravenous infusion ). It is supplied as a powder to be mixed with sterile water before administration (see Information for doctors and healthcare professionals at the end of this leaflet). 
Always use this medicine exactly as your doctor has told you. Consult a doctor if you are unsure.

Fabrazyme is only given under the supervision of a doctor who has experience in the treatment of Fabry’s disease. Your doctor may recommend that you be treated at home provided you meet certain criteria. Contact your doctor if you want to receive the treatment at home.

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

Use for children and adolescents

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

If you use more Fabrazyme then you should 

Doses of up to 3 mg/kg body weight are harmless.

If you forget to use Fabrazyme

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

If you have any further questions on 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.

In clinical trials, adverse reactions were observed mainly during or shortly thereafter, the patients received the medicine (“infusion-related reactions”). Severe life-threatening allergic reactions (“anaphylactic reactions”) have been reported in some patients. If you experience any serious side effects, talk to your doctor immediately.

Very common symptoms (may affect 1 in 10) include chills, fever, chills, nausea, vomiting, headache, and sensations in the skin such as burning or stinging sensation. Your doctor may decide to slow down the infusion rate or give you additional medicines to prevent such reactions from happening.

List of other side effects:

chest pain breathing difficultiespalloritchingabnormal tear secretionfeeling weaktinnitus (ringing in the ears)nasal congestiondiarrhearednessmuscle pain high blood pressure sudden swelling of the face or throat edema (swelling) in arms and legs feeling dizzy stomach upset muscle cramps somnolence increased heart rate abdominal pain back pain as low heart rate extreme fatiguefainting attackscoughdiscomfort in the abdomen swollen face joint pain lowered blood pressure chest discomfortfacial edema aggravated difficulty in breathing muscle tension fatigue flare rednesspainfeeling of tightness in the throatdizzinesspalpitationdecreased pain sensitivityburning sensationalizing breathinghivespain in arms and less inflammation of the nose and throat heatwave feeling of warmthhyperthermiaincreased sensitivity in the mouthmusculoskeletal stiffness
tremorRed eyewear pain sore throat rapid breathingitchy rash feeling of heat and cold difficulty swallowing pain at the infusion site reaction at the infusion site itching in the eyes swollen ears bronchospasm (spasm of the trachea)Runny nose heartburn discomfort from the skinmusculoskeletal painrhinitis ( inflammation of the nose)flu-like illness general malaise low heart rate due to disturbances in the heart’s conduction system increased pain sensitivityblocked upper respiratory tract rash(mottled bluish-red) skin discoloration could arm and less blood clotting at the injection site skin discoloration edema (swelling)
decreased oxygen level in the blood severe inflammation of the blood vessels  

For some patients who started treatment with the recommended dose and who then received a reduced dose for a longer period of time, some symptoms of Fabry’s 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 expiration date is the last day of the specified month.

Unopened bottles 
Store in a refrigerator (2 ° C – 8 ° C)

Prepared and diluted solutions 
The reconstituted solution cannot be stored but must be diluted immediately. The diluted solution can be stored for up to 24 hours at 2 ° C -8 ° C.

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 packaging and other information

Content declaration

  • The active substance is agalsidase beta, one bottle contains 5 mg.
  • The other ingredients are:
    • Mannitol
    • Sodium hydrogen phosphate monohydrate
    • Sodium dihydrogen phosphate heptahydrate

What the medicine looks like and the contents of the pack

Fabrazyme is supplied as a white to off-white powder. After dilution, it is a clear, colorless liquid, free from foreign particles. The reconstituted solution must be further diluted. 
Pack sizes: 1, 5, and 10 bottles per carton. Not all pack sizes may be marketed.

Marketing Authorization Holder and Manufacturer

Marketing Authorization Holder 
Genzyme Europe BV, Paasheuvelweg 25, 1105 BC Amsterdam, The Netherlands.

Manufacturer

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

Contact the representative of the marketing authorization holder to find out more about this medicine:

Sanofi AB

Tel: +46 (0) 8 634 50 00

Muhammad Nadeem

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