ALDURAZYME^® 100 U/ml concentrate for solution for infusion

Laronidase

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Aldurazyme is used to treat patients with MPS I disease (Mucopolysaccharidosis I). It is given to treat the non-neurological manifestations of the disease.

People with MPS I disease have either a low level or no level of an enzyme called α-L-iduronidase, which breaks down specific substances (glycosaminoglycans) in the body. As a result, these substances do not get broken down and processed by the body as they should. They accumulate in many tissues in the body, which causes the symptoms of MPS I.

Aldurazyme is an artificial enzyme called laronidase. This can replace the natural enzyme which is lacking in MPS I disease.

Instruction for use - dilution and administration

The concentrate for solution for infusion has to be diluted before administration and is for intravenous use (see information for health care professionals).

Administration of Aldurazyme should be carried out in an appropriate clinical setting where resuscitation equipment to manage medical emergencies would be readily available.

Home infusion

Your doctor may consider that you can have home infusion of Aldurazyme if it is safe and convenient to do so. If you get a side effect during an infusion of Aldurazyme, your infusion staff member may stop the infusion and start appropriate medical treatment.

Dosage

The recommended dosage regimen of Aldurazyme is 100 U/kg body weight given once every week as an intravenous infusion. The initial infusion rate of 2 U/kg/h may be gradually increased every fifteen minutes, if tolerated, to a maximum of 43 U/kg/h. The total volume of the administration should be delivered in approximately 3-4 hours.

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

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

Like all medicines, this medicine can cause side effects, although not everybody gets them

Side effects were mainly seen while patients were being given the medicine or shortly after (infusion-associated reactions). If you experience any reaction like this, you should contact your doctor immediately. The number of these reactions decreased the longer that patients were on Aldurazyme. The majority of these reactions were mild or moderate in intensity. However, severe systemic allergic reaction (anaphylactic reaction) has been observed in patients during or up to 3 hours after Aldurazyme infusions. Some of the symptoms of such a severe allergic reaction were life-threatening and included extreme difficulty breathing, swelling of the throat, low blood pressure, and low oxygen level in the body. A few patients who had a prior history of severe MPS I related upper airway and pulmonary involvement, experienced severe reactions including bronchospasm (airway constriction), respiratory arrest, and swelling of the face. The frequency of bronchospasm and respiratory arrest is unknown. The frequency of severe allergic reaction (anaphylactic reaction) and swelling of the face is considered common and may affect up to 1 in 10 people.

Very common symptoms (may affect more than 1 in 10 people) which were not serious include

• headache,
• nausea,
• abdominal pain,
• rash,
• joint disease,
• joint pain,
• back pain,
• pain in arms or legs,
• flushing,
• fever, chills,
• increased heart rate,
• increased blood pressure,
• reaction at the infusion site such as swelling, redness, build-up of fluid, discomfort, itchy rash, pale colour of the skin, discoloured skin, or sensation of being warm.

Other side effects include the following:

Common (may affect up to 1 in 10 people)

• increased body temperature
• tingling
• dizziness
• cough
• difficulty in breathing
• vomiting
• diarrhoea
• rapid swelling under the skin in areas such as the face, throat, arms and legs which can be life-threatening if throat swelling blocks the airway
• hives
• itching
• hair loss
• cold sweat, heavy sweating
• muscle pain
• paleness
• cold hands or feet
• feeling hot, feeling cold
• fatigue
• influenza like illness
• pain at injection site
• restlessness

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

• allergic reactions (hypersensitivity)
• abnormally slower heart rate
• increased or abnormally high blood pressure
• voice box swelling
• bluish color of the skin (due to lower levels of oxygen in the blood)
• fast breathing
• redness of the skin
• leakage of the medicine into the surrounding tissue at the site of injection, where it can cause damage
• inability of the lungs to work properly (respiratory failure)
• throat swelling
• high-pitched breathing sound
• obstruction of airways causing difficulty in breathing
• lip swelling
• tongue swelling
• swelling especially of the ankles and feet due to fluid retention
• drug specific antibody, a blood protein produced in response to medicine
• antibody that neutralizes the effect of medicine

Keep this medicine out of the sight and reach of children.

You should not be given this medicine after the expiry date which is stated on the label after the letters EXP. The expiry date refers to the last day of that month.

Unopened vials:

Store in a refrigerator (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.

This leaflet does not contain all the information about your medicine. If you have any questions or are not sure about anything, ask your doctor or pharmacist.