ALDURAZYME 500 U Koncentrat do sporządzania roztworu infuzyjnego

Treść ulotki

1. Aldurazyme 100 U/mL concentrate for solution for infusion
   1. What is Aldurazyme and what it is used for
   2. What you need to know before you take Aldurazyme
   3. How to take Aldurazyme
   4. Possible side effects
   5. How to store Aldurazyme
   6. Contents of the pack and other information

Aldurazyme 100 U/mL concentrate for solution for infusion

Laronidase

Read this leaflet carefully before you use this medicine as it contains important
information.

• Keep this leaflet. You may need to read it again.
• If you have any questions, ask your doctor or pharmacist.
• This medicine has been prescribed for you only. Do not pass it on to others even if their symptoms are the same as yours because it could be harmful.
• If you get any side effects, even those not listed in this leaflet, tell your doctor or pharmacist. See section 4.

Contents of this leaflet:

• 1. What Aldurazyme is and what it is used for
• 2. What you need to know before you use Aldurazyme
• 3. How to use Aldurazyme
• 4. Possible side effects
• 5. How to store Aldurazyme
• 6. Contents of the pack and other information

1. What is Aldurazyme and what it is used for

Aldurazyme is used to treat patients with MPS I (Mucopolysaccharidosis I). It is administered
to treat the non-neurological manifestations of the disease.
People affected by MPS I disease have a low or absent level of an enzyme called α-L-
iduronidase, which is able to break down certain specific substances (glycosaminoglycans) in the body. Consequently, these substances are not broken down and processed correctly, accumulating in
many tissues of the body and causing the symptoms of MPS I.
Aldurazyme is an artificial enzyme called laronidase, which is able to replace the natural enzyme deficient
in MPS I disease.

2. What you need to know before you take Aldurazyme

Do not take Aldurazyme
If you are allergic (hypersensitive) to laronidase or any of the other ingredients of this medicine (listed in section 6).

Warnings and precautions
Talk to your doctor before using Aldurazyme.
Contact your doctor immediately if treatment with Aldurazyme causes:

• Allergic reactions, including anaphylaxis (a severe allergic reaction) - see section 4 “Possible side effects”. Some of these reactions may be life-threatening. Symptoms may include respiratory failure/difficulty breathing (inability of the lungs to function properly), stridor (a high-pitched breathing sound) and other disturbances due to airway obstruction, rapid breathing, excessive contraction of the airway muscles causing difficulty breathing (bronchospasm), lack of oxygen in body tissues (hypoxia), low blood pressure, slowed heartbeat or itchy rash (urticaria).
• Infusion-associated reactions, i.e. any undesirable effect that occurs during the infusion, or within the day of the infusion (see symptoms under section 4, “Possible side effects”).

If you have these reactions, the Aldurazyme infusion must be stopped immediately; your doctor will start appropriate treatment.
These reactions may be particularly severe in case of previous upper airway obstruction related to MPS I.
You may be given other medicines to help prevent allergic-type reactions, such as antihistamines, medicines to reduce fever (e.g. paracetamol) and/or corticosteroids.
The doctor will also decide whether you can continue to receive Aldurazyme.

Other medicines and Aldurazyme
Tell your doctor if you are taking medicines containing chloroquine or procaine, due to the possible risk of decreased action of Aldurazyme.
Tell your doctor or pharmacist if you are taking or have recently taken any other medicines, including those available without a prescription.

Pregnancy, breastfeeding and fertility
There is insufficient experience regarding the use of Aldurazyme in pregnant women. Aldurazyme should not be taken during pregnancy unless absolutely necessary.
It is not known whether Aldurazyme is secreted into breast milk. It is recommended to stop breastfeeding during treatment with Aldurazyme.
The effects of Aldurazyme on fertility are unknown.
If you are pregnant, suspect you are pregnant, are planning to become pregnant, or are breastfeeding, ask your doctor or pharmacist for advice before using this medicine.

Driving and operating machinery
The effects on the ability to drive and operate machinery have not been studied.

Aldurazyme contains sodium
This medicine contains 30 mg of sodium (the main component of table salt) per vial.
This is equivalent to 1.5% of the maximum recommended daily intake of sodium for an adult.

3. How to take Aldurazyme

Instructions for use, dilution and administration
The concentrate for infusion solution must be diluted before administration and is
formulated for intravenous use (see information for healthcare professionals).
The administration of Aldurazyme should be carried out in a suitable clinical setting where
resuscitation equipment is immediately available in case of need to manage
a medical emergency.
Home infusion
Your doctor may consider that you can undergo home infusion of Aldurazyme if it is
safe and convenient to do so. If you experience a side effect during an Aldurazyme infusion, the
infusion staff can stop the infusion and start appropriate medical treatment.
Dosage
The recommended dosage of Aldurazyme is 100 U/kg of body weight, administered once a
week by intravenous infusion. The initial infusion rate of 2 U/kg/hour may be
gradually increased every fifteen minutes, if tolerated, up to a maximum of 43 U/kg/hour. The
total volume of the infusion should be administered over approximately 3-4 hours.
Use this medicine following exactly the instructions of your doctor. If you have any doubts consult
your doctor.

If you miss an Aldurazyme infusion
If you have missed an Aldurazyme infusion, consult your doctor.

If you take more Aldurazyme than you should
If the dose of Aldurazyme administered is too high or the infusion is too fast, adverse
drug reactions may occur. Too rapid infusion of Aldurazyme can cause nausea,
abdominal pain, headache, dizziness and difficulty breathing (dyspnea). In such situations,
the infusion must be stopped or the infusion rate slowed down immediately. The doctor
will decide whether further intervention is necessary.
If you have any doubts about the use of this medicine, ask your doctor or pharmacist.

4. Possible side effects

Like all medicines, this medicine can cause side effects, although not everyone will experience them.
Side effects have been observed mainly during the administration of the drug or
shortly after (infusion-related reactions). If you should experience similar reactions, you must

inform your doctor immediately. The number of undesirable reactions decreased with continued
use of Aldurazyme. The majority of undesirable reactions were of mild to
moderate intensity. However, a systemic allergic reaction (anaphylactic reaction) has been observed in patients
during the infusion of Aldurazyme or up to 3 hours after its completion. Some symptoms of this
severe allergic reaction were potentially life-threatening and included extreme difficulty breathing,
severe throat swelling, low blood pressure, and low oxygen levels in the body. In a limited number of
patients with a previous history of severe upper airway and lung impairment related to MPS I, serious
reactions have occurred, including bronchospasm, respiratory arrest, and facial swelling. The frequency of
bronchospasm and respiratory arrest is unknown, while the frequencies of the severe allergic reaction
(anaphylactic reaction) and facial swelling are considered common, meaning they can affect up to 1 in 10 people.
Very common symptoms (may affect more than 1 in 10 people) that were not serious include

• headache,
• nausea,
• abdominal pain,
• skin rash,
• joint disease,
• joint pain,
• back pain,
• arm and leg pain,
• hot flashes, fever,
• chills,
• increased heart rate,
• increased blood pressure
• reaction at the infusion site, such as swelling, redness, fluid accumulation, discomfort, itchy rash, paleness, skin color changes, or a feeling of warmth

Other side effects include the following:

Common (may affect more than 1 in 10 people)

• increased body temperature
• tingling
• dizziness
• cough
• difficulty breathing
• vomiting
• diarrhea
• rapid subcutaneous swelling in areas such as the face, throat, arms, and legs, which can be life-threatening if swelling of the throat blocks the airways
• urticaria (hives)
• itching
• hair loss
• cold sweats, excessive sweating
• muscle pain
• paleness
• cold hands or feet
• feeling of warmth, feeling of cold
• fatigue
• flu-like illness
• pain at the injection site
• restlessness

Not known (frequency cannot be estimated from available data)

• allergic reactions (hypersensitivity)
• abnormally slow heartbeat
• abnormally increased or high blood pressure
• swelling of the larynx
• bluish discoloration of the skin (due to lower oxygen levels in the blood)
• rapid breathing
• skin redness
• leakage of the drug into surrounding tissues at the injection site, where it can cause damage
• inability of the lungs to function properly (respiratory failure)
• swelling of the throat
• wheezing sounds
• airway obstruction causing difficulty breathing
• swelling of the lips
• swelling of the tongue
• swelling, especially of the ankles and feet, due to fluid retention
• presence of drug-specific antibodies, blood proteins produced in response to the medicine
• presence of antibodies that neutralize the effect of the medicine

Reporting of side effects
If you get any side effects, talk to your doctor or pharmacist. This includes any side effects not listed in this leaflet. You can also report side effects directly through
the national reporting system indicated in Annex V
By reporting side effects you can help provide more information on the safety
of this medicine.

5. How to store Aldurazyme

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.
Sealed vials:
Store in a refrigerator (2 ºC – 8 ºC).
Do not dispose of any medicine in wastewater or household waste. Ask your pharmacist how to
dispose of medicines you no longer use. This will help protect the environment.

6. Contents of the pack and other information

What Aldurazyme contains

• The active substance is laronidase. 1 mL of solution in the vial contains 100 U of laronidase. Each 5 mL vial contains 500 U of laronidase.
• The excipients are: sodium chloride, sodium phosphate monobasic monohydrate, sodium phosphate dibasic heptahydrate, polysorbate 80, water for injections.

Description of the appearance of Aldurazyme and contents of the pack
Aldurazyme is supplied as a concentrate for solution for infusion. It is a solution from clear to
slightly opalescent and from colourless to light yellow.
Pack sizes :1, 10 and 25 vials. Not all pack sizes may be marketed.

Marketing Authorisation Holder and Manufacturer
Marketing Authorisation Holder
Sanofi B.V.
Paasheuvelweg 25
1105 BP Amsterdam
Netherlands.
Manufacturer
Genzyme Ireland Ltd, IDA Industrial Park, Old Kilmeaden Road, Waterford, Ireland
For further information on this medicinal product, contact the local representative of the Marketing
Authorisation Holder:

België/Belgique/Belgien/
Luxembourg/Luxemburg
Sanofi Belgium
Tél/Tel: + 32 2 710 54 00

Magyarország
SANOFI-AVENTIS Zrt.
Tel: +36 1 505 0050

България
Swixx Biopharma EOOD
Тел.: +359 (0)2 4942 480

Malta
Sanofi S.r.l.
Tel: +39 02 39394275

Česká republika
sanofi-aventis, s.r.o.
Tel: +420 233 086 111

Nederland
Sanofi B.V.
Tel: +31 20 245 4000

Danmark
sanofi A/S
Tlf: +45 45 16 70 00

Norge
sanofi-aventis Norge AS
Tlf: + 47 67 10 71 00

Deutschland
Sanofi-Aventis Deutschland GmbH
Tel.: 0800 04 36 996
Tel. aus dem Ausland: +49 69 305 7013

Österreich
sanofi-aventis GmbH
Tel: + 43 1 80 185 – 0

Eesti
Swixx Biopharma OÜ
Tel: +372 640 10 30

Polska
sanofi-aventis Sp. z o.o.
Tel: +48 22 280 00 00

Ελλάδα
Sanofi-Aventis Μονοπρόσωπη AEBE
Τηλ: +30 210 900 1600

Portugal
Sanofi – Produtos Farmacêuticos, Lda.
Tel: +351 21 35 89 400

España
sanofi-aventis, S.A.
Tel: +34 93 485 94 00

România
Sanofi Romania SRL
Tel: +40 (0) 21 317 31 36

France
Sanofi Winthrop Industrie
Tél: 0 800 222 555
Appel depuis l’étranger: +33 1 57 63 23 23

Slovenija
Swixx Biopharma d.o.o.
Tel: +386 1 235 51 00

Hrvatska
Swixx Biopharma d.o.o.
Tel: +385 1 2078 500

Slovenská republika
Swixx Biopharma s.r.o.
Tel: +421 2 208 33 600

Ireland
sanofi-aventis Ireland Ltd. T/A SANOFI
Tel: +353 (0) 1 403 56 00

Suomi/Finland
Sanofi Oy
Puh/Tel: + 358 201 200 300

Ísland
Vistor hf.
Sími: +354 535 7000

Sverige
Sanofi AB
Tel: +46 (0)8 634 50 00

Italia
Sanofi S.r.l.
Tel: 800536389

United Kingdom (Northern Ireland)
sanofi-aventis Ireland Ltd. T/A SANOFI
Tel: +44 (0) 800 035 2525

Κύπρος
C.A. Papaellinas Ltd.
Τηλ: +357 22 741741

Latvija
Swixx Biopharma SIA
Tel: +371 6 616 47 50

Lietuva
Swixx Biopharma UAB
Tel: +370 5 236 91 40

Other sources of information
More detailed information on this medicinal product is available on the website of the European
Medicines Agency: http://www.ema.europa.eu. Also, links to other websites on rare diseases
and related therapeutic treatments are provided.
This leaflet is available in all languages of the European Union/European Economic Area on the
website of the European Medicines Agency.

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The following information is intended exclusively for healthcare professionals:
Each vial of Aldurazyme is for single use. The concentrate for solution for infusion must be diluted with sodium chloride 9 mg/mL (0.9 %) solution for infusion,
using aseptic technique. It is recommended that the diluted Aldurazyme solution be administered to patients using an infusion set equipped with a 0.2 μm in-line filter.
From a microbiological safety point of view, the product must be used immediately. If it is not used immediately, the storage of the diluted solution (for infusion) must not
exceed 24 hours at 2 ºC – 8 ºC, provided that the dilution has been carried out under controlled and validated aseptic conditions.
Aldurazyme must not be mixed with other medicinal products within the same infusion.

Preparation of Aldurazyme infusion (use aseptic technique)

• Determine the number of vials to dilute, based on the individual patient's weight. Remove the necessary vials from the refrigerator approximately 20 minutes before, to bring them to room temperature (below 30 °C).
• Before dilution, visually inspect each vial to exclude the presence of particulate matter or discoloration. The solution, from clear to slightly opalescent and from colourless to light yellow, must not contain visible particles. Do not use vials that show particles or discoloration.
• Determine the total volume of the infusion based on the individual patient's weight, i.e. 100 mL (if the patient's weight is 20 kg or less), or 250 mL (if the weight exceeds 20 kg) of sodium chloride 9 mg/mL (0.9 %) solution for infusion.
• Withdraw and discard a volume of sodium chloride 9 mg/mL (0.9 %) solution for infusion from the infusion bag, equal to the total volume of Aldurazyme to be added.
• Withdraw the necessary volume from the Aldurazyme vials and combine the withdrawn volumes.
• Add the combined volumes of Aldurazyme to the sodium chloride 9 mg/mL (0.9 %) solution for infusion.
• Gently mix the infusion solution.
• Before use, visually inspect the solution to exclude the presence of particles. Use only clear and colourless solutions, free of visible particles.

Unused medicinal product or waste material derived from such medicinal product should be disposed of in
accordance with local regulations.