Prospecto:information for the user

VPRIV 400units powder for solution for infusion

velaglucerase alfa

Read this prospect carefully before starting to use this medicine, because it contains important information for you.

• Keep this prospect, as you may need to read it again.
• If you have any doubts, consult your doctor.
• Ifyou experienceadverse effects, consult your doctor,even if they are not listed in this prospect (see section4).

VPRIV is a long-term enzyme replacement therapy (ERT) for patients with type 1 Gaucher's disease.

Gaucher's disease is a genetic disorder caused by the absence of an enzyme or the presence of a defective enzyme, known as glucocerebrosidase. When this enzyme is absent or does not function correctly, a substance called glucocerebroside accumulates in the body's cells. The accumulation of this material causes the signs and symptoms that are present in Gaucher's disease.

VPRIV contains a substance called velaglucerase alfa that is designed to replace the missing or defective enzyme, glucocerebrosidase, in patients with Gaucher's disease.

No useVPRIV

• If you are very allergic to velaglucerase alfa or any of the other components of this medication (listed in section 6).

Warnings and precautions

Consult your doctor before using VPRIV.

• If you receive treatment with VPRIV, you may experience adverse effects during or after infusion (see section 4, Possible adverse effects).These reactions are called infusion-related reactions and may manifest as a hypersensitivity reaction with symptoms such as nausea, rash, difficulty breathing, back pain, chest discomfort, urticaria, joint pain, or headache.
• Aside from hypersensitivity symptoms, infusion-related reactions may manifest as dizziness, high blood pressure, fatigue, fever, itching, blurred vision, or vomiting.
• If you experience any of these symptoms, you must inform your doctor immediately.
• You may be administered other medications to treat or help prevent future reactions. These medications may include antihistamines, antipyretics, and corticosteroids.

Inform your doctor if you have previously experienced any infusion-related reactions with any other TSE for Gaucher's disease.

Children

Do not use in children under 4 years of age, as there is no experience with the use of this medication in this age group.

Use of VPRIV with other medications

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

Pregnancy

Gaucher's disease may be more active in women during pregnancy and the weeks following delivery.Women with Gaucher's disease who are pregnant or considering becoming pregnant should consult with their doctor before using this medication.

Breastfeeding

The transfer of VPRIV to breast milk is unknown. If you are breastfeeding or planning to breastfeed, you must consult with your doctor before receiving this medication. Your doctor will help you decide whether to stop breastfeeding or interrupt the use of VPRIV, taking into account the benefits of breastfeeding for the baby and the benefits of VPRIV for the mother.

Consult your doctor or pharmacist before receiving this medication.

Driving and operating machinery

The influence of VPRIV on the ability to drive and operate machinery is negligible.

VPRIV contains sodium

This medication contains less than 23 mg of sodium (1 mmol) per vial, which is essentially "sodium-free".

This medication should be used exclusively under the proper supervision of a doctor with experience in treating Gaucher's disease; this person will also determine the dose to be received.

It should be administered by a doctor or nurse through intravenous infusion.As long as it is under the supervision of a doctor, VPRIV can be self-administered (by you or your caregiver) after receiving the relevant training from the doctor and/or nurse. Self-administration should take place in the presence of an adult responsible.

Dose

The recommended dose is 60units/kg administered every two weeks.

If you are currently receiving treatment for Gaucher's disease with another TSE and your doctor wants to switch you to VPRIV, you can initially receive VPRIV at the same dose and frequency that you have been receiving the other TSE.

Use in children and adolescents

VPRIV can be given to children and adolescents (4to 17years old) at the same dose and frequency as in adults.

Use in elderly patients

VPRIV can be administered to elderly patients (over 65years old) at the same dose and frequency as in adults.

Response to treatment

Your doctor will monitor your response to treatment, and may modify the dose (more or less) over time.

If you tolerate the infusions well in the clinic, it may be possible for your doctor or nurse to administer them at home.

Administration

VPRIV is supplied in a vial in the form of compacted powder that is mixed with sterile water and then diluted in sodium chloride solution 9mg/ml (0.9%) before intravenous infusion.

Once prepared, your doctor or nurse will administer the medication by drip into a vein (intravenous infusion) over a period of 60minutes.

For self-administration, the dose and infusion rate should not be modified without the consent of the doctor responsible for your treatment.

If you use more VPRIV than you should

If you think you have used more VPRIV than you should, contact your doctor.

If you use less VPRIV than you should

If you think you have used less VPRIV than you should, contact your doctor.

If you forget to use VPRIV

If you have missed a VPRIV infusion, contact your doctor.

If you interrupt treatment with VPRIV

If you interrupt treatment with VPRIV, contact your doctor. If you interrupt treatment, you may experience symptoms again.If you have any other questions about the use of this medication, ask your doctor.

Like all medicines, VPRIV may cause side effects, although not everyone will experience them.

Commonly (may affect 1 in 10 people), patients experienced a severe allergic reaction, with difficulty breathing, chest discomfort (chest tightness), nausea, facial swelling, lip, tongue, or throat inflammation (anaphylactic/anaphylactoid reactions). Allergic skin reactions such as urticaria, severe rash, or itching are also common. If any of these reactions occur, inform your doctor immediately.

Most side effects, including allergic reactions, occurred during infusion or shortly after. These reactions are called infusion-related reactions.

• If a severe reaction occurs, your doctor will immediately stop the intravenous infusion and begin administering the appropriate medical treatment.
• In the event of home administration and a severe infusion-related reaction, including anaphylaxis, immediately stop the infusion, seek urgent medical attention, and contact your doctor.
• If severe reactions occur and/or there is a loss of effect of this medication, your doctor will perform a blood test to detect the presence of antibodies that may affect the outcome of your treatment.
• Your doctor or nurse may decide to continue administering VPRIV even if you experience some infusion-related reactions. Your condition will be closely monitored.

Other infusion-related reactions that occurred very commonly (may affect more than 1 in 10 people) include headache, dizziness, fever/increased body temperature, back pain, joint pain, and fatigue, as well as increased blood pressure (reported frequently), blurred vision, and vomiting (reported infrequently). If any of these reactions occur, inform your doctor immediately.

Among other side effects, the following are included:

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

• bone pain

• weakness/loss of strength
• stomach pain

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

• prolonged time for a wound to stop bleeding, which may cause easy bleeding, spontaneous bleeding, and hematoma formation
• skin redness
• rapid heart rate
• development of antibodies against VPRIV (see section 2)
• decreased blood pressure

Reporting side effects

If you experience side effects, consult your doctor or pharmacist, even if they are possible side effects not listed in this prospectus. 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 on the safety of this medication.

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

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

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

Do not freeze.

Store the vial in the outer packaging to protect it from light.

Reconstituted and diluted solution for infusion:

Use immediately. Do not exceed 24hours at a temperature of 2°C to 8°C.

Do not use VPRIV if the solution is discolored or if it presents foreign particles.

Medicines should not be disposed of through the drains or in the trash.Ask your pharmacist how to dispose of the medicines you no longer need.This will help protect the environment.

Composition of VPRIV

• The active ingredient is velaglucerase alfa.

Each vial contains 400 units of velaglucerase alfa.

After reconstitution, 1 ml of solution contains 100 units of velaglucerase alfa.

• The other components are sucrose, sodium citrate dihydrate, citric acid monohydrate, and polisorbate 20 (see section 2 “VPRIV contains sodium”).

Appearance of the product and contents of the pack

20 ml glass vial with a white to off-white colored plunger for solution for infusion.

Packs of 1, 5 or 25 vials.

Only some pack sizes may be marketed.

Marketing authorization holder and responsible persons for manufacturing

Marketing authorization holder

Takeda Pharmaceuticals International AG Ireland Branch

Block 2 Miesian Plaza

50-58 Baggot Street Lower

Dublin 2

D02 HW68

Ireland

Responsible person for manufacturing

Takeda Pharmaceuticals International AG Ireland Branch

Block 2 Miesian Plaza

50-58 Baggot Street Lower

Dublin 2

D02 HW68

Ireland

Shire Pharmaceuticals Ireland Limited

Block 2 & 3 Miesian Plaza

50 – 58 Baggot Street Lower

Dublin 2

Ireland

For further information about this medicinal product, please consult the representative of the marketing authorization holder in your country:

Last update of the summary of product characteristics: 11/2024.

Other sources of information

The detailed information on this medicinal product is available on the website of the European Medicines Agency:http://www.ema.europa.eu.It also provides links to other websites on rare diseases and orphan medicinal products.

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This information is intended for healthcare professionals only:

VPRIV is a powder for solution for infusion.It requires reconstitution and dilution, and is intended for intravenous infusion only.VPRIV is for single use only, and is administered through a 0.2 or 0.22 µm filter.Discard any unused solution.VPRIV should not be infused with other medicinal products in the same infusion, as compatibility in solution with other products has not been evaluated.The total volume of the infusion should be administered within 60 minutes.

Use aseptic techniques.

To prepare VPRIV, follow these steps:

1. Determine the number of vials to reconstitute based on the patient's weight and the prescribed dose.
2. Remove the required number of vials from the refrigerator.Reconstitute each vial using sterile water for injection:

1. Once reconstituted, mix the vials gently. Do not shake.
2. Before dilution, visually inspect the solution in the vials; the solution should be transparent to slightly opalescent, and colourless; do not use if the solution is discoloured or if particles are observed in suspension.
3. Remove the calculated volume of the drug from the corresponding number of vials. A remainder of solution will be left in the vial:

1. Dilute the total required volume in 100 ml of sodium chloride 9 mg/ml (0.9%) solution for infusion.Mix gently.Do not shake.Start the infusion within 24 hours after reconstitution.

From a microbiological point of view, use the medicinal product immediately.If not used immediately, the time period for storage under the conditions defined in the leaflet and the conditions for storage prior to use are the responsibility of the user.Do not exceed 24 hours at a temperature between 2 °C and 8 °C.

Do not dispose of any unused medicinal product or waste materials via the sewage system or by disposal in the normal household refuse.Eliminate any unused medicinal product or waste materials in accordance with local requirements.

Traceability

To improve the traceability of biological medicinal products, clearly record the name and batch number of the medicinal product administered.