Product Information for the User

Elaprase 2 mg/ml Concentrate for Solution for Infusion

idursulfase

This product is subject to additional monitoring, which will expedite the detection of new information about its safety. You can contribute by reporting any adverse effects you may experience. The final part of section 4 includes information on how to report these adverse effects.

Read this entire product information carefully before starting to use this product, as it contains important information for you

• Keep this product information, as you may need to refer to it again.
• If you have any questions, consult your doctor, pharmacist, or nurse.
• This product has been prescribed to you alone, and you should not give it to others who may have the same symptoms as you, as it may harm them.
• If you experience any adverse effects, consult your doctor, pharmacist, or nurse, even if they are not listed in this product information. See section 4.

1.What Elaprase is and for what it is used

2.What you need to know before starting to use Elaprase

3.How to use Elaprase

4.Possible adverse effects

5.Storage of Elaprase

6.Contents of the pack and additional information

Elaprase is used as an enzyme replacement therapy to treat children and adults with Hunter syndrome (Mucopolisacaridosis 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 suffering from Hunter syndrome, a carbohydrate called glycosaminoglycan, which is normally metabolized by the body, does not metabolize and slowly accumulates in various cells of the body. This causes abnormal functioning of these cells, and as a result, causes problems in several organs that can lead to tissue destruction and organ dysfunction and insufficiency.The typical organs in which the glycosaminoglycan accumulates are the spleen, liver, lungs, heart, and connective tissue. In some patients, glycosaminoglycan also accumulates in the brain.Elaprase contains an active ingredient called idursulfase, which acts as a replacement for the enzyme that is found at a low level, and thus breaks down this carbohydrate in affected cells.

Enzyme replacement therapy is generally administered as long-term treatment.

No use Elaprase

• If you have had severe or potentially life-threatening allergic reactions to idursulfase or any of the other components of this medication (listed in section6), and these reactions could not be controlled with appropriate medical treatment.

Warnings and precautions

Consult your doctor or nurse before starting to use this medication.

If you are being treated with Elaprase, you may experience adverse effects during or after infusion (see section4, Possible adverse effects). The most common symptoms are itching, rash, urticaria, fever, headache, increased blood pressure, and facial flushing. In most cases, you can continue to receive this medication even if these symptoms appear. If you experience an allergic reaction after receiving this medication, you must contact your doctor immediately. You may be given other medications, such as antihistamines and corticosteroids, to treat or help prevent allergic reactions.

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

Your genetic makeup(a genetic configuration of all active genes in human cells, which determines individual and specific characteristics of each person)may influence your response to this medication, as well as your risk of developing antibodies and presenting infusion-related adverse effects.In individual cases, so-called "neutralizing antibodies" may develop, which can reduce the activity of Elaprase and your response to treatment. The long-term effects of antibody development on treatment response have not been established.Consult your doctorfor more information.

Keeping a record

To improve the traceability of biological medications, the name and batch number of the product administered must be clearly recorded. Talk to your healthcare professional if you have any doubts.

Other medications and Elaprase

No interactions of this medication with other medications are known.

Inform your doctor, pharmacist, or nurse if you are taking, have taken recently, or may need to take any other medication.

Pregnancy and breastfeeding

If you are pregnant or breastfeeding, or if you think you may be pregnant or plan to become pregnant, consult your doctor or pharmacist before using this medication.

Driving and operating machines

The influence of this medication on the ability to drive and operate machines is negligible or insignificant.

Elaprase contains sodium

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

Follow exactly the administration instructions for this medication as indicated by your doctor. If in doubt, consult your doctor again.

This medication will be administered under the supervision of a doctor or nurse experienced in the treatment of Hunter syndrome or other hereditary metabolic disorders.

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

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

Consult with your doctor in case of an overdose of this medication.

If you forgot to use Elaprase

If you have forgotten a dose of Elaprase, contact your doctor.

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

Like all medicines, this medicine can cause side effects, although not everyone will experience them.

The majority of side effects are mild or moderate and related to perfusion; however, certain side effects can be severe. Over time, the number of these reactions associated with perfusion decreases.

If you have difficulty breathing, with or without blue skin, report this to your doctor immediately and seek immediate medical attention.

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

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

• Perfusion-related reaction (see the section titled “Warnings and precautions”)

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

• Dizziness, tremors
• Increased heart rate, irregular heart rhythm, blue skin
• Increased blood pressure, decreased blood pressure
• Difficulty breathing, cough, low oxygen levels in the blood
• Swollen tongue, indigestion
• Joint pain
• Inflammation at the injection site, limb inflammation, facial inflammation

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

• Accelerated breathing

Side effects of unknown frequency (cannot be estimated from available data):

• Severe allergic reactions

Reporting side effects

If you experience any type of side effect, consult your doctor, pharmacist, or nurse, even if it is a possible side effect that does not appear in this leaflet. You can also report them directly through the national notification system included in theAppendix V. By reporting side effects, you can contribute to providing more information about the safety of this medicine

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

Do not use this medication after the expiration date that appears on the label and on the box after CAD. The expiration date is the last day of the month indicated.

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

Do not freeze

Do not use this medication if you observe discoloration or the presence of foreign particles.

Medicines should not be disposed of through drains or in the trash. Ask your pharmacist how to dispose of the containers and medications that you no longer need. In this way, you will help protect the environment.

Composition of Elaprase

The active ingredient is idursulfase, which is a form of the human enzyme iduronate-2-sulfatase. Idursulfase is produced in a line of human cells using genetic engineering techniques (this 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 components are Polisorbate 20, sodium chloride, sodium phosphate dibasic, heptahydrate, sodium phosphate monobasic, monohydrate, and water for injection.

Appearance of the product and contents of the package

This medicine is a concentrate for solution for infusion. It is supplied in a glass vial in the form of a colorless, transparent to slightly opalescent solution.

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

Elaprase is supplied in sizes of 1, 4, and 10 vials per package. Some package sizes may only be marketed.

Marketing Authorization Holder

Takeda Pharmaceuticals International AG Ireland Branch

Block 2 Miesian Plaza

50-58 Baggot Street Lower

Dublin 2

D02 HW68

Ireland

Responsible for manufacturing

Takeda Pharmaceuticals International AG Ireland Branch

Block 2 Miesian Plaza

50-58 Baggot Street Lower

Dublin 2

D02 HW68

Ireland

Last review date of this leaflet: 11/2022.

This medicine has been authorized under«exceptional circumstances». This type of authorization means that due to the rarity of the disease, it has not been possible to obtain complete information about this medicine.

The European Medicines Agency will review any new information available for this medicine annually and this leaflet will be updated as necessary.

Other sources of information

Further information on this medicine is available on the website of the European Medicines Agency:http://www.ema.europa.eu. There are also links to other websites about rare diseases and orphan medicines.

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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This information is intended solely for healthcare professionals:

Instructions for use, handling, and disposal

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

1. Dilute the required volume of Elaprase concentrate for solution for infusion in 100 ml of sodium chloride solution for infusion (0.9%) for infusion. It is recommended to administer the total volume of the infusion using a 0.2 µm in-line filter. 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 gently mixed, but not shaken.

1. Check visually that the solution does not contain particles or show discoloration before administration. Do not shake.

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

For single use only. The disposal of unused product and all materials that have come into contact with it will be carried out in accordance with local regulations.