Elaprase 2 mg/ml concentrate for solution for infusion

Elaprase is used as enzyme replacement therapy to treat Hunter syndrome (Mucopolysaccharidosis II) when the level of the enzyme iduronate-2-sulfatase in the body is lower than normal. If you suffer from Hunter syndrome, a carbohydrate called glycosaminoglycan which is normally broken down by your body is not broken down and slowly accumulates in various organs in your body causing these cells to function abnormally, thereby causing problems for various organs in your body which can lead to tissue destruction and organ failure. Elaprase works by acting as a replacement for the enzyme that is at a low level, thereby breaking down this carbohydrate in affected cells.

Enzyme replacement therapy is usually administered as a long-term treatment.

Do not use Elaprase

If you are allergic (hypersensitive) to idursulfase or any of the other ingredients of Elaprase.

Take special care with Elaprase

If you are treated with Elaprase you may experience reactions during or following an infusion (see section 4 Possible Side Effects). The most common symptoms are itching, rash, hives, fever, headache, increased blood pressure, and flushing (redness). Most of the time you can still be given Elaprase even if these symptoms occur. If you experience an allergic side effect following administration of Elaprase, you should contact your doctor immediately. You may be given additional medicines such as antihistamines and corticosteroids to treat or help prevent allergic-type reactions.

If severe allergic (anaphylactic-type) reactions occur, your doctor will stop the infusion immediately, and start giving you suitable treatment. You may need to stay in hospital.

Using other medicines

There is no known interaction of Elaprase with other medicines.

Please tell your doctor or pharmacist if you are taking or have recently taken any other medicines, including medicines obtained without a prescription.

Using Elaprase with food and drink

Due to the way the product is broken down by the body interactions with food and drink are unlikely.

Pregnancy and breast-feeding

Use of Elaprase during pregnancy is not recommended. Elaprase may get into breast milk therefore you should talk to your doctor or pharmacist before using this medicine if you are breast-feeding.

Driving and using machines

It is not known if Elaprase will affect the ability to drive or use machines.

Elaprase should only be used under the supervision of a doctor or healthcare professional (e.g. nurse) who is knowledgeable in the treatment of Hunter syndrome or other inherited metabolic disorders. Elaprase has to be diluted in 9 mg/ml (0.9%) sodium chloride solution for infusion before use. The usual dose is an infusion of 0.5 mg (half a milligram) for every kg you weigh. After dilution Elaprase is given through a vein (drip feed). The infusion will normally last for 1 to 3 hours and will be given every week.

If you use more Elaprase than you should

There is no experience with overdoses of Elaprase.

If you forget to have Elaprase

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

Like all medicines, Elaprase can cause side effects, although not everybody gets them. Most side effects are mild to moderate and associated with the infusion, however some side effects may be serious. Over time the number of these infusion-associated reactions decreases.

Very common side effects (more than 1 per 10) are:

• Headache
• Increased blood pressure, flushing (redness)
• Shortness of breath, wheezing
• Abdominal pain, nausea
• Chest pain
• Hives, rash, itching
• Fever
• Infusion site swelling
• Infusion related reaction (see Section entitled ?Take special care with Elaprase?)

Common side effects (more than 1 per 100 but less than 1 per 10)) are:

• Dizziness, tremor
• Rapid heart beat, irregular heart beat, bluish skin
• Decreased blood pressure
• Difficulty breathing, cough, quickened breathing, low oxygen levels in your blood
• Diarrhoea, swollen tongue
• Rednessof the skin
• Pain in the joints
• Swelling of the extremities, facial swelling

Unknown frequency side effects

• Serious allergic reactions

If you have problems breathing, with or without bluish skin, tell your doctor immediately.

If any of the side effects becomes serious, or if your notice any side effects not listed in this leaflet, please tell your doctor.

Keep out of reach and sight of children.
Do not use Elaprase 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.
Store in a refrigerator (2°C ? 8°C)
Do not freeze

Do not use Elaprase if you notice that there is discolouration or presence of foreign particles. 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.

What Elaprase contains

The active substance is idursulfase, which is a form of the human enzyme iduronate-2-sulfatase. Idursulfase is produced in a human cell line by genetic engineering technology.
Each vial of Elaprase contains 6 mg of idursulfase. Each ml contains 2 mg of idursulfase.

The other ingredients are Polysorbate 20
Sodium chloride
Sodium phosphate dibasic, heptahydrate
Sodium phosphate monobasic, monohydrate
Water for injections

What Elaprase looks like and contents of the pack

Elaprase is a concentrate for solution for infusion. It is supplied in a glass vial as a clear to slightly opalescent, colourless solution.
Each vial contains 3 ml of concentratefor solution for infusion.
Elaprase is supplied in pack sizes of 1, 4 and 10 vials per carton
Not all pack sizes may be marketed.

Marketing Authorisation Holder

Shire Human Genetic Therapies AB, Svärdvägen 11D, 182 33 Danderyd, Sweden

Manufacturer

Shire Human Genetic Therapies AB, Åldermansgatan 13, 227 64 Lund, Sweden

This leaflet was last approved

This medicine has been authorised under ?exceptional circumstances?.
This means that because of the rarity of this disease it has been impossible to get complete information on this medicine.
The European Medicines Agency (EMA) will review any new information on the medicine every year and this leaflet will be updated as necessary.

More detailed information on this medicine is available on the European Medicines Agency (EMA) web site: http://www.ema.europa.eu. There are also links to other web sites about rare disease and treatments. ---------------------------------------------------------------------------------------------------------------------------------

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

Instructions for use, handling and disposal

1. Calculate the total dose to be administered and number of Elaprase vials needed.

2. Dilute the total volume of Elaprase concentrate for solution for infusion required in 100 ml of 9 mg/ml sodium chloride solution for infusion (0.9%w/v). Care must be taken to ensure the sterility of the prepared solutions since Elaprase does not contain any preservative or bacteriostatic agent; aseptic technique must be observed. Once diluted, the solution should be mixed gently, but not shaken.

3. The solution should be inspected visually for particulate matter and discolouration prior to administration. Do not shake.

4. It is recommended that administration is started as soon as possible. The chemical and physical stability of the diluted solution has been demonstrated for 8 hours at 25°C.

5. Do not infuse Elaprase concomitantly in the same intravenous line with other agents.

6. For single use only. Any unused product or waste material should be disposed of in accordance with local requirements.