VPRIV 400 units powder for solution for infusion

Introduction

Package Leaflet: Information for the User

VPRIV 400units powder for solution for infusion

velaglucerase alfa

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.
• This medicine has been prescribed for you only. Do not pass it on to others. It may harm them, even if their symptoms are the same as yours.
• If you experience any side effects, talk to your doctor. This includes any possible side effects not listed in this leaflet (see section 4).

Contents of the pack

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

1. What is VPRIV and what is it used for

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

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

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

2. What you need to know before you use VPRIV

Do not useVPRIV

• if you are severely allergic to velaglucerase alfa or any of the other ingredients of this medicine (listed in section 6).

Warnings and precautions

Talk to your doctor before using VPRIV

• If you are receiving treatment with VPRIV, you may experience side effects during or after the infusion (see section 4, Possible side effects). These reactions are called infusion-related reactions and may occur as an allergic reaction with symptoms such as nausea, rash, difficulty breathing, back pain, chest discomfort (chest tightness), urticaria, joint pain or headache.
• In addition to the symptoms of allergic reactions, infusion-related reactions may occur as dizziness, high blood pressure, fatigue, fever, itching, blurred vision or vomiting.

If you experience any of these symptoms, you should tell your doctor immediately.

• You may be given other medicines to treat or help prevent future reactions. These medicines may include antihistamines, antipyretics and corticosteroids.

Tell your doctor if you have previously experienced an infusion-related reaction with any other ERT for Gaucher disease.

Children

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

Using VPRIV withother medicines

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

Pregnancy

Gaucher disease may become more active in women during pregnancy and in the weeks following childbirth. Women with Gaucher disease who are pregnant or are planning to become pregnant should talk to their doctor before using this medicine.

Breast-feeding

It is not known whether VPRIV can pass into breast milk. If you are breast-feeding or thinking of breast-feeding, you should talk to your doctor before receiving this medicine. Your doctor will help you decide whether to stop breast-feeding or stop using VPRIV, taking into account the benefits of breast-feeding for the baby and the benefits of VPRIV for the mother.

Talk to your doctor or pharmacist before receiving this medicine.

Driving and using machines

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

VPRIV contains sodium

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

3. How to use VPRIV

This medicine should only be used under the supervision of a doctor who has experience in the treatment of Gaucher disease; this person will also determine the dose you should receive.

It must be administered by a doctor or nurse by intravenous infusion. Under the supervision of the doctor, VPRIV can be self-administered (by you or your caregiver) after you have received the relevant training from the doctor and/or nurse. Self-administration should take place in the presence of a responsible adult.

Dose

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

If you are currently receiving treatment for Gaucher disease with another ERT and your doctor wants to switch you to VPRIV, you may initially receive VPRIV at the same dose and frequency as you were receiving the other ERT.

Use in children and adolescents

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

Use inelderly patients

VPRIV can be administered to elderly patients (65 years of age or older) at the same dose and frequency as in adults.

Response to treatment

Your doctor will monitor your response to treatment and may adjust the dose (up or down) over time.

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

Administration

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

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

For self-administration, the dose and infusion rate should not be changed without the consent of the treating doctor.

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 miss an infusion of VPRIV, contact your doctor.

If you stop using VPRIV

If you stop using VPRIV, contact your doctor. If you stop using VPRIV, you may experience a return of symptoms. If you have any further questions on the use of this medicine, ask your doctor.

4. Possible side effects

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

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

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

• If a severe reaction occurs, your doctor will stop the intravenous infusion immediately and start the appropriate medical treatment.
• If you are administering the infusion at home and experience a severe infusion-related reaction, including anaphylaxis, stop the infusion immediately, seek urgent medical attentionand contact your doctor.
• If the reactions are severe and/or there is a loss of effect of this medicine, your doctor may 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 an infusion-related reaction. 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 commonly), blurred vision and vomiting (reported uncommonly). If any of these reactions occur, tell your doctor immediately.

Among the other side effects are:

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

• bone pain

• weakness/loss of strength
• stomach pain

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

• prolongation of the time it takes for a wound to stop bleeding that may cause easy bruising, spontaneous bleeding and hematoma formation
• redness of the skin
• rapid heartbeat
• development of antibodies against VPRIV (see section 2)
• decreased blood pressure

Reporting of side effects

If you experience any side effects, talk to your doctor or pharmacist. This includes any possible side effects 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 VPRIV

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 carton and label after the letters CAD. The expiry date is the last day of the month stated.

Store in a refrigerator (2°C - 8°C).

Do not freeze.

Keep the vial in the outer carton in order to protect from light.

Reconstituted and diluted solution for infusion:

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

Do not use VPRIV if the solution is discolored or if it contains any visible 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 to protect the environment.

6. Container Contents and Additional Information

VPRIV Composition

• 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 polysorbate 20 (see section 2 "VPRIV contains sodium").

Product Appearance and Container Contents

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

Packs of 1, 5, or 25 vials.

Only some pack sizes may be marketed.

Marketing Authorization Holder and Manufacturers

Marketing Authorization 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

Shire Pharmaceuticals Ireland Limited

Block 2 & 3 Miesian Plaza

50 – 58 Baggot Street Lower

Dublin 2

Ireland

For further information on this medicinal product, please contact the local representative of the marketing authorization holder:

Date of Last Revision of this Leaflet: 11/2024.

Other Sources of Information

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

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This information is intended solely for healthcare professionals:

VPRIV is a powder for solution for infusion. It requires reconstitution and dilution and is intended solely for intravenous infusion. 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 medications in the same infusion, as compatibility in solution with other products has not been evaluated. The total infusion volume should be administered over a period of 60 minutes.

Use aseptic techniques.

Prepare VPRIV as follows:

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

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

1. Dilute the total required volume in 100 ml of 9 mg/ml (0.9%) sodium chloride 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 in-use storage times and conditions are the responsibility of the user. Do not exceed 24 hours at a temperature between 2°C and 8°C.

Do not throw the medicinal product down the drain or into the household waste. Eliminate unused medicinal products and all materials that have come into contact with them in accordance with local regulations.

Traceability

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