ELAPRASE 2 mg/ml CONCENTRATE for SOLUTION for INFUSION

Introduction

Package Leaflet: Information for the User

Elaprase 2mg/ml concentrate for solution for infusion

idursulfase

?This medicine is subject to additional monitoring, which will help to quickly identify new safety information. You can help by reporting any side effects you may get. The last section of this leaflet includes information on how to report side effects.

Read all of this leaflet carefully before you start using this medicine, because it contains important information for you.

• Keep this leaflet, you may need to read it again.
• If you have any further questions, ask your doctor, pharmacist, or nurse.
• This medicine has been prescribed for you only. Do not pass it on to others. It may harm them, even if their signs of illness are the same as yours.
• If you get any side effects, talk to your doctor, pharmacist, or nurse. This includes any possible side effects not listed in this leaflet. See section 4.

Contents of the pack and other information

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

1. What is Elaprase and what is it used for

Elaprase is used as enzyme replacement therapy to treat children and adults with Hunter syndrome (Mucopolysaccharidosis II) when the level of the enzyme iduronate-2-sulfatase in the body is lower than normal, helping to improve the symptoms of the disease. When you have Hunter syndrome, a carbohydrate called glycosaminoglycan, which is normally metabolized by the body, is not metabolized and slowly accumulates in various cells of the body. This causes abnormal functioning of these cells, and consequently causes problems in several organs that can lead to tissue destruction and organ dysfunction and failure. The typical organs where glycosaminoglycan accumulates are the spleen, liver, lungs, heart, and connective tissue. In some patients, glycosaminoglycan also accumulates in the brain. Elaprase contains an active substance called idursulfase, which acts as a replacement for the enzyme that is at a low level, and thus breaks down this carbohydrate in the affected cells.

Enzyme replacement therapy is usually given as long-term treatment.

2. What you need to know before you use Elaprase

Do not use Elaprase

• if you have had severe or life-threatening allergic reactions to idursulfase or any of the other ingredients of this medicine (listed in section 6) that could not be controlled with medical treatment.

Warnings and precautions

Talk to your doctor or nurse before you start using this medicine.

If you are being treated with Elaprase, you may experience side effects during or after the infusion (see section 4, Possible side effects). The most common symptoms are itching, rash, hives, fever, headache, increased blood pressure, and flushing of the face. In most cases, this medicine can still be given to you even if these symptoms occur. If you experience an allergic reaction after receiving this medicine, you should contact your doctor immediately. You may be given other medicines, such as antihistamines and corticosteroids, to treat or help prevent allergic reactions.

Your doctor will stop the infusion immediately if severe allergic reactions occur and will start the appropriate treatment. You may need to stay in the hospital.

The nature of your genotype (a genetic makeup of all active genes in human cells, which determines individual characteristics and specific features of each person) may influence your therapeutic response to this medicine, as well as your risk of developing antibodies and experiencing infusion-related side effects. In individual cases, so-called "neutralizing antibodies" may develop, which may decrease the activity of Elaprase and your response to treatment. The long-term effects of antibody development on treatment response have not been established. Talk to your doctor for more information.

Keeping a record

In order to improve the traceability of biological medicinal products, the name and batch number of the product administered should be clearly recorded. Talk to your healthcare professional if you have any questions.

Other medicines and Elaprase

No interactions of this medicine with other medicines are known.

Tell your doctor, pharmacist, or nurse if you are taking, have recently taken, or might take any other medicines.

Pregnancy and breast-feeding

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 using this medicine.

Driving and using machines

This medicine has no or negligible influence on the ability to drive and use machines.

Elaprase contains sodium

This medicine contains 11.1 mg of sodium (a major component of table salt/cooking salt) in each vial. This is equivalent to 0.6% of the maximum daily intake of sodium recommended for an adult.

3. How to use Elaprase

Follow the instructions for administration of this medicine exactly as told by your doctor. If you are not sure, talk to your doctor again.

This medicine will be given to you under the supervision of a doctor or nurse with experience in the treatment of Hunter syndrome or other inherited metabolic disorders.

The recommended dose is an infusion of 0.5 mg (half a milligram) per kilogram of body weight.

Elaprase must be diluted in a 9 mg/ml (0.9%) sodium chloride solution for infusion before use. After dilution, this medicine is given through a vein (by drip). The infusion usually lasts between 1 to 3 hours and will be given to you every week.

Use in children and adolescents

The recommended dose in children and adolescents is the same as for adults.

If you use more Elaprase than you should

Talk to your doctor if you have taken too much of this medicine.

If you miss a dose of Elaprase

If you miss an infusion of Elaprase, contact your doctor.

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

4. Possible side effects

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

Most side effects are mild or moderate and are related to the infusion; however, some side effects can be serious. Over time, the number of these infusion-related reactions decreases.

If you have difficulty breathing,with or without blue discoloration of the skin, tell your doctor immediately and seek immediate medical attention.

Very common side effects (may affect more than 1 in 10 people):

• Headache
• Flushing of the face
• Shortness of breath, wheezing
• Abdominal pain, nausea, vomiting, frequent and/or soft stools
• Chest pain
• Hives, rash, itching, redness of the skin
• Fever

• Infusion-related reaction (see section "Warnings and precautions")

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

• Dizziness, tremors
• Rapid heart rate, irregular heart rate, blue discoloration of the skin
• Increased blood pressure, decreased blood pressure
• Difficulty breathing, cough, low oxygen levels in the blood
• Swelling of the tongue, indigestion
• Joint pain
• Inflammation at the infusion site, inflammation of the limbs, facial swelling

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

• Rapid breathing

Rare side effects (cannot be estimated from the available data):

• Severe allergic reactions

Reporting of side effects

If you experience any side effects, talk to your doctor, pharmacist, or nurse, even if it is possible that they are not listed in this leaflet. You can also report side effects directly via the national reporting system listed in Appendix V. By reporting side effects, you can help provide more information on the safety of this medicine.

5. Storage of Elaprase

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 and carton after EXP. The expiry date is the last day of the month shown.

Store in a refrigerator (between 2°C and 8°C).

Do not freeze.

Do not use this medicine if you notice discoloration or the 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. This will help protect the environment.

6. Contents of the pack and other information

Composition of Elaprase

The active substance is idursulfase, which is a form of the human enzyme iduronate-2-sulfatase. Idursulfase is produced in a human cell line using genetic engineering techniques (which involves introducing genetic information into human cells in the laboratory, which will then produce the desired product).

Each vial of Elaprase contains 6 mg of idursulfase. Each ml contains 2 mg of idursulfase.

The other ingredients are Polysorbate 20, sodium chloride, disodium phosphate dihydrate, sodium phosphate monohydrate, and water for injections.

Appearance and pack contents

This medicine is a concentrate for solution for infusion. It is provided in a glass vial as a clear to slightly opalescent solution.

Each vial contains 3 ml of concentrate for solution for infusion.

Elaprase is available in pack sizes of 1, 4, and 10 vials. Not all pack sizes may be marketed.

Marketing authorisation holder

Takeda Pharmaceuticals International AG Ireland Branch

Block 2 Miesian Plaza

50-58 Baggot Street Lower

Dublin 2

D02 HW68

Ireland

Manufacturer

Takeda Pharmaceuticals International AG Ireland Branch

Block 2 Miesian Plaza

50-58 Baggot Street Lower

Dublin 2

D02 HW68

Ireland

Date of last revision of this leaflet: 11/2022.

This medicine has been authorised under "exceptional circumstances". This means that due to the rarity of the disease, it has not been possible to obtain complete information on this medicine.

The European Medicines Agency will review any new information that may become available every year and this leaflet will be updated as necessary.

Other sources of information

Detailed information on this medicine is available on the European Medicines Agency web site: http://www.ema.europa.eu. There are also links to other web sites about rare diseases and orphan medicines.

The leaflet can be found on the European Medicines Agency web site in all EU/EEA languages.

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This information is intended only for healthcare professionals:

Instructions for use, handling, and disposal

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

1. Dilute the required total volume of Elaprase concentrate for solution for infusion in 100 ml of 9 mg/ml (0.9%) sodium chloride solution for infusion. It is recommended to administer the total infusion volume using an in-line filter of 0.2 µm. Precautions should be taken to ensure the sterility of the prepared solutions, as Elaprase does not contain preservatives or bacteriostatic agents; aseptic technique should be observed. Once diluted, the solution should be mixed gently but not shaken.

1. The solution should be inspected visually for particulate matter and discoloration prior to administration. It should not be shaken.

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

1. Elaprase should not be infused concomitantly in the same intravenous line with other medicines.

For single use only. Disposal of unused product and all materials that have come into contact with it should be done according to local regulations.