VPRIV

Package leaflet: Information for the user

VPRIV 400 Units powder for solution for infusion

velaglucerase alfa
Please 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 questions, ask your doctor.
• This medicine has been prescribed for you only. Do not give it to other people, even if their symptoms are the same as yours, as it may be harmful.
• If you experience any side effects, including those not listed in this leaflet, tell your doctor (see section 4).

Contents of this leaflet

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

1. What VPRIV is and what it is 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 the deficiency or malfunction of an enzyme called glucocerebrosidase. When this enzyme is deficient or malfunctioning, a substance called glucocerebroside accumulates within cells of the body, leading to the signs and symptoms of Gaucher disease.
VPRIV contains a substance called velaglucerase alfa, which is used to replace the deficient or defective enzyme, glucocerebrosidase, in patients with Gaucher disease.

2. What you need to know before using VPRIV

Do not use VPRIV

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

Warnings and precautions
Talk to your doctor before receiving VPRIV

• If you are treated with VPRIV, you may develop unwanted effects during or after the infusion (see section 4, Possible side effects). These effects are called infusion-related reactions and may present as hypersensitivity reactions, with symptoms such as nausea, rash, difficulty breathing, back pain, chest discomfort (chest tightness), hives, joint pain, or headache.
• In addition to hypersensitivity symptoms, infusion-related reactions may also include dizziness, hypertension, fatigue, fever, itching, blurred vision, or vomiting. If you experience any of these symptoms, you must inform your doctor immediately.
• You may be prescribed other medicines to treat or prevent future reactions. These medicines may include: antihistamines, antipyretics, or corticosteroids.

Tell your doctor if you have previously experienced an infusion reaction to other enzyme replacement therapies (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.
Other medicines and VPRIV
Inform your doctor if you are taking, have recently taken, or might take any other medicines.
Pregnancy
Gaucher disease may worsen during pregnancy and for several weeks after delivery. Women with Gaucher disease who are pregnant or planning a pregnancy should consult their doctor before receiving this medicine.
Breast-feeding
It is not known whether VPRIV passes into breast milk. If you are breast-feeding or intend to breast-feed, consult your doctor before receiving this medicine. Your doctor will help you decide whether to discontinue breast-feeding or to discontinue VPRIV use, taking into account the benefits of breast-feeding for the child and the benefits of VPRIV for the mother.
Driving and using machines
VPRIV has no effect or negligible effect on the ability to drive vehicles or operate machinery.
VPRIV contains sodium
This medicine contains 12.15 mg of sodium (a main component of table salt) per vial. This corresponds to 0.6% of the maximum daily recommended dietary intake of sodium for an adult.

3. How to use VPRIV

This medicine should only be used under the appropriate medical supervision of a physician experienced in the treatment of Gaucher disease, who will also determine the dose you should receive.
This medicine is administered by intravenous infusion by a doctor or nurse.
Under continued medical supervision, VPRIV may be self-administered (by you or by a caregiver) after adequate training provided by the doctor and/or nurse.
Self-administration must be performed in the presence of a responsible adult.

Dosage
The recommended dose is 60 Units/kg administered every other week.
If you are currently being treated with another enzyme replacement therapy (ERT) for Gaucher disease and your doctor wishes to prescribe VPRIV, you may start VPRIV therapy at the same dose and frequency as your previous ERT.

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

Use in elderly patients
VPRIV may be administered to elderly patients (aged over 65 years) 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 (increase or decrease) over time.
If you tolerate the infusions well when administered in a hospital setting, your doctor or nurse may administer VPRIV to you in your home.

Administration
VPRIV is supplied in a vial as a compact powder, which must be mixed with sterile water and further diluted with sodium chloride 9 mg/ml (0.9%) infusion solution before intravenous infusion.
Once prepared, the medicine will be administered to you by a doctor or nurse via intravenous infusion (through an intravenous drip into a vein) over a period of 60 minutes.
For self-administration, the prescribed dose and infusion rate must not be changed without the agreement of your 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 have missed an infusion of VPRIV, contact your doctor.

If you stop using VPRIV
If you stop treatment with VPRIV, contact your doctor. If you discontinue treatment, symptoms may return.
If you have any questions about how to use this medicine, consult your doctor.

4. Possible adverse reactions

Like all medicines, this medicine can cause adverse reactions, although not everybody gets them.
A common frequency (may affect up to 1 in 10 people), patients have developed a severe allergic reaction, with difficulty breathing, chest discomfort (chest tightness), feeling unwell (nausea), swelling of the face, lips, tongue or throat (anaphylactic/anaphylactoid reactions); skin allergic reactions such as hives, severe rash or itching are also common. If you experience such adverse reactions, inform your doctor immediately.
Most adverse reactions, including allergic reactions, occurred during or shortly after the infusion. These reactions are known as infusion-related reactions.

• If a severe reaction occurs at the clinical centre, your doctor will immediately stop the intravenous infusion and start appropriate medical treatment.
• If a severe infusion-related reaction, including anaphylaxis, occurs during home administration, immediately stop the infusion, seek emergency medical help immediately, and contact your doctor.
• If you experience severe reactions and/or loss of effectiveness of the medicine, your doctor may order a blood test to check whether you have developed antibodies that may affect treatment outcome.
• 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 have 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 high blood pressure (reported at common frequency), blurred vision and vomiting (reported at uncommon frequency). If you experience such adverse reactions, inform your doctor immediately.
Other adverse reactions include:
Very common adverse reactions (may affect more than 1 in 10 people):

• bone pain
• weakness/loss of strength
• stomach ache

Common adverse reactions (may affect up to 1 in 10 people):

• increased time needed for bleeding from a cut to stop, which may result in easy/spontaneous bleeding/easy bruising
• skin redness
• rapid heartbeat
• development of antibodies to VPRIV (see section 2)
• reduction in blood pressure

Reporting of adverse reactions
If you experience any adverse reaction, including those not listed in this leaflet, talk to your doctor or pharmacist. You can also report adverse reactions directly via the national reporting system listed in Annex V. By reporting adverse reactions, you can help provide more information on the safety of this medicine.

5. How to store 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
Exp. The expiry date refers to the last day of that month.
Store in the refrigerator (2°C – 8°C).
Do not freeze.
Keep the vial in the outer packaging to protect the medicine from light.
Reconstituted and diluted infusion solution:
Use immediately. Do not exceed 24 hours at 2°C – 8°C.
Do not use this medicine if the solution changes colour or contains foreign particles.
Do not dispose of any medicine via wastewater or household waste. Ask your pharmacist how to dispose of medicines you no longer use. This will help protect the environment.

6. Package Contents and Other Information

What VPRIV contains

• The active substance is velaglucerase alfa. Each vial contains 400 Units of velaglucerase alfa. After reconstitution, each 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”).

Description of VPRIV and contents of the pack
20 ml glass vial containing a white to off-white powder for solution for infusion.
Pack sizes of 1, 5 or 25 vials.
Not all pack sizes may be marketed.

Marketing Authorisation Holder and Manufacturer

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
Shire Pharmaceuticals Ireland Limited
Block 2 & 3 Miesian Plaza
50–58 Baggot Street Lower
Dublin 2
Ireland

For further information about this medicine, please contact the local representative of the Marketing Authorisation Holder:

België/Belgique/Belgien Lietuva
Takeda Belgium NV Takeda, UAB
Tél/Tel: +32 2 464 06 11 Tel: +370 521 09 070
[email protected] [email protected]

България Luxembourg/Luxemburg
Такеда България ЕООД Takeda Belgium NV
Тел.: +359 2 958 27 36 Tél/Tel: +32 2 464 06 11
[email protected] [email protected]

Česká republika Magyarország
Takeda Pharmaceuticals Czech Republic s.r.o. Takeda Pharma Kft.
Tel: +420 234 722 722 Tel.: +36 1 270 7030
[email protected] [email protected]

Danmark Malta
Takeda Pharma A/S Drugsales Ltd
Tlf.: +45 46 77 10 10 Tel: +356 21419070
[email protected] [email protected]

Deutschland Nederland
Takeda GmbH Takeda Nederland B.V.
Tel: +49 (0)800 825 3325 Tel: +31 20 203 5492
[email protected] [email protected]

Eesti Norge
Takeda Pharma OÜ Takeda AS
Tel: +372 6177 669 Tlf: +47 800 800 30
[email protected] [email protected]

Ελλάδα Österreich
Takeda ΕΛΛΑΣ Α.Ε. Takeda Pharma Ges.m.b.H.
Tηλ: +30 210 6387800 Tel: +43 (0) 800-20 80 50
[email protected] [email protected]

España Polska
Takeda Farmacéutica España, S.A Takeda Pharma Sp. z o.o.
Tel: +34 917 90 42 22 Tel.: +48223062447
[email protected] [email protected]

France Portugal
Takeda France SAS Takeda Farmacêuticos Portugal, Lda.
Tél: +33 1 40 67 33 00 Tel: +351 21 120 1457
[email protected] [email protected]

Hrvatska România
Takeda Pharmaceuticals Croatia d.o.o. Takeda Pharmaceuticals SRL
Tel: +385 1 377 88 96 Tel: +40 21 335 03 91
[email protected] [email protected]

Ireland Slovenija
Takeda Products Ireland Ltd Takeda Pharmaceuticals farmacevtska družba d.o.o.
Tel: 1800 937 970 Tel: +386 (0) 59 082 480
[email protected] [email protected]

Ísland Slovenská republika
Vistor hf. Takeda Pharmaceuticals Slovakia s.r.o.
Sími: +354 535 7000 Tel: +421 (2) 20 602 600
[email protected] [email protected]

Italia Suomi/Finland
Takeda Italia S.p.A. Takeda Oy
Tel: +39 06 502601 Puh/Tel: 0800 774 051
[email protected] [email protected]

Κύπρος Sverige
A.POTAMITIS MEDICARE LTD Takeda Pharma AB
Τηλ: +357 22583333 Tel: 020 795 079
[email protected] [email protected]

Latvija United Kingdom (Northern Ireland)
Takeda Latvia SIA Takeda UK Ltd
Tel: +371 67840082 Tel: +44 (0) 3333 000 181
[email protected] [email protected]

Further sources of information
More detailed information on this medicine is available on the website of the European Medicines Agency: https://www.ema.europa.eu. Links to other websites on rare diseases and their therapeutic treatments are also provided.

The following information is intended for healthcare professionals only:

VPRIV is a powder for solution for infusion. It must be diluted and reconstituted and is intended for intravenous infusion only. VPRIV is for single use only and must be administered using a 0.2 or 0.22 µm filter. Any unused solution should be discarded. VPRIV must not be administered with other medicinal products in the same infusion, as compatibility with other drugs in solution has not been evaluated. The total infusion volume must be administered over 60 minutes. Use aseptic technique.

Prepare VPRIV as follows:

1. Determine the number of vials to reconstitute based on the patient's body weight and prescribed dose.
2. Remove the required number of vials from the refrigerator. Reconstitute each vial with sterile water for injections:
   Vial capacity   Sterile water for injections
   400 Units     4.3 ml
3. After reconstitution, gently mix the contents of the vials. Do not shake.
4. Before further dilution of the reconstituted medicinal product, visually inspect the solution in the vials: it should appear clear or slightly opalescent and colourless. Do not use if there is a change in colour or presence of foreign particulate matter.
5. Withdraw the calculated volume of medicinal product from the appropriate number of vials. A portion of the solution will remain in the vial:
   Vial capacity   Volume to be withdrawn
   400 Units     4.0 ml
6. Dilute the total required volume in 100 ml of 0.9% sodium chloride infusion solution (9 mg/ml). Gently mix. Do not shake. Begin infusion within 24 hours after reconstitution.

From a microbiological standpoint, the medicinal product should be used immediately. If not used immediately, the user is responsible for ensuring storage conditions and duration prior to use. Do not exceed 24 hours at a temperature of 2 °C–8 °C.
Do not dispose of the medicinal product via wastewater or household waste. Dispose of unused medicine and waste materials derived from this medicinal product in accordance with local regulations.
Traceability
To improve traceability of biological medicinal products, clearly record the name and batch number of the administered medicinal product.