VPRIV 400 Units powder for solution for infusion

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 missing or defective enzyme named glucocerebrosidase. When this enzyme is missing or does not work properly, a substance called glucocerebroside builds up inside cells in the body. The build-up of this material causes the signs and symptoms found in Gaucher disease.

VPRIV is designed to replace the missing or defective enzyme, glucocerebrosidase, in patients with Gaucher disease.

Do not use VPRIV

• If you are allergic (hypersensitive) to velaglucerase alfa or any of the other ingredients of VPRIV.

Take special care with VPRIV

• If you are treated with VPRIV, you may experience a side effect during or following the infusion (see section 4, possible side effects). This is known as an infusion-related reaction and can sometimes be severe.
• Infusion-related reactions include dizziness, headache, nausea, low or high blood pressure, tiredness, and fever. If you experience an infusion-related reaction, you must tell your doctor immediately.
• If you have an infusion-related reaction you may be given additional medicines to treat or help prevent future reactions. These medicines may include antihistamines, antipyretics, and corticosteroids.
• If the infusion-related reaction is severe, your doctor will stop the intravenous infusion immediately and start giving you appropriate medical treatment.
• Most of the time you can still be given VPRIV even if you experience an infusion-related reaction.

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

Children

VPRIV should not be used in children under the age of 2 years.

Using other medicines

Please tell your doctor if you are taking or have recently taken any other medicines, including medicines obtained without a prescription.

Pregnancy and breast-feeding

Gaucher disease may become more active in a woman during pregnancy and for a few weeks after birth. Women with Gaucher disease who are considering pregnancy should talk with their doctor.

VPRIV has not been studied in pregnant women. Studies in animals do not show harmful effects from VPRIV. Caution should be exercised when using VPRIV in pregnancy.

VPRIV has not been studied in women who are breast-feeding and it is not known whether VPRIV appears in breast milk. However, VPRIV contains a protein that may be digested by the child. Cautious use of VPRIV during breast feeding is recommended.

Ask your doctor or pharmacist for advice before taking any medicine.

Driving and using machines

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

Important information about one of the ingredients of VPRIV

Each 200 Units vial of this medicine contains 6.07 mg sodium. Each 400 Units vial of this medicine contains 12.15 mg sodium. This should be taken into consideration by patients on a controlled sodium diet.

VPRIV is only to be used under appropriate medical supervision of a doctor who is knowledgeable in the treatment of Gaucher disease. VPRIV is given by a doctor or nurse by intravenous infusion.

Dose

The usual dose is 60 Units/kg given every other week.

If you are currently being treated for Gaucher disease with another ERT and your doctor wants to change you to VPRIV, you can initially receive VPRIV at the same dose and frequency you had been receiving the other ERT. In clinical studies, doses ranging from 15 Units/kg to 60 Units/kg have been used.

Use in children and adolescents

VPRIV may be given to children and adolescents (2 to ?17 years of age) at the same dose and frequency as in adults.

Response to treatment

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

If you are tolerating your infusions well in the clinic, your doctor or nurse may administer your infusions at home.

Administration

VPRIV is supplied in a vial as a packed powder which is mixed with sterile water and further diluted in sodium chloride 9 mg/ml (0.9%) solution prior to intravenous infusion.

After preparation, your doctor or nurse will give VPRIV to you through a drip into a vein (by intravenous infusion) over a period of 60 minutes.

If you use more VPRIV than you should

If you feel ill whilst receiving the infusion, tell your doctor or nurse immediately.

If you forget to have VPRIV

If you have missed an infusion, please contact your doctor.

If you stop using VPRIV

Discuss changes in treatment with your doctor.
If you have any further questions on the use of this medicine, ask your doctor.

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

In studies with VPRIV, side effects were mainly seen while patients were being infused with the medicine or shortly after (infusion-related reactions). These side effects have included headache, dizziness, decreased blood pressure, increased blood pressure, nausea, tiredness, and fever/body temperature increased. If you experience any side effect like these, please tell your doctor immediately. The majority of these side effects were mild in intensity.

However a few patients experienced an allergic skin reaction such as severe rash or itching. A severe allergic reaction, with difficulty breathing, swelling of the face, lips, tongue or throat occured. If any of these happen tell your doctor immediately.

The frequency of possible side effects listed below is defined using the following convention Very common Common Uncommon Rare Very rare Not known affects more than 1 user in 10 affects 1 to 10 users in 100 affects 1 to 10 users in 1,000 affects 1 to 10 users in 10,000 affects less than 1 user in 10,000 frequency cannot be estimated from the available data.

In studies with VPRIV the following side effects were reported:

The very common side effects are:

• headache
• dizziness
• bone pain
• joint pain
• back pain
• infusion-related reaction
• weakness/loss of strength/fatigue
• fever/body temperature increased

The common side effects are:

• abdominal pain/nausea
• abnormal blood clotting
• decreased blood pressure
• increased blood pressure
• flushing
• rapid heart beat
• rash/hives
• serious allergic reactions
• developing antibodies to VPRIV

If any of the side effects gets serious, or if you notice any side effects not listed in this leaflet, please tell your doctor.

Keep out of the reach and sight of children.

Do not use VPRIV after the expiry date which is stated on the outer carton and vial after ?EXP?. The expiry date refers to the last day of that month.

Store in the refrigerator (2ºC - 8ºC).
Do not freeze.
Keep the vial in the outer carton in order to protect from light.
Do not use if the solution is discoloured or if foreign particles are present.

Medicines should not be disposed of via waste water or household waste. Ask your pharmacist how to dispose of medicines no longer required. These measures will help to protect the environment.

What VPRIV contains

• The active substance is velaglucerase alfa. One vial of VPRIV 200 Units powder contains 200 Units of velaglucerase alfa. One vial of VPRIV 400 Units powder contains 400 Units of velaglucerase alfa. After reconstitution the solution contains 100 Units of velaglucerase alfa per ml.
• The other ingredients are sucrose, sodium citrate dihydrate, citric acid monohydrate, and polysorbate 20.

What VPRIV looks like and contents of the pack

VPRIV is a powder for solution for infusion which comes as a white to off-white powder. VPRIV 200 Units powder is packaged in a 5 ml glass vial, with 1, 5 or 25 vials per carton. VPRIV 400 Units powder is packaged in a 20 ml glass vial, with 1, 5 or 25 vials per carton.

Not all pack sizes may be marketed.

Marketing Authorisation Holder and Manufacturer

Marketing Authorization Holder
Shire Pharmaceuticals Ireland Limited
5 Riverwalk
Citywest Business Campus
Dublin 24
Ireland

Manufacturer
Shire Human Genetic Therapies AB
Aldermansgata 13
227 64 Lund
Sweden

This leaflet was last approved in

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The following information is intended for medical or healthcare professionals only.

VPRIV is a powder for solution for infusion. VPRIV requires reconstitution and dilution and is intended for intravenous infusion only. VPRIV is for single?use only and is administered through a 0.22 µm filter. Vials are single-use only. Discard any unused solution. VPRIV should not be infused with other medicines in the same infusion as the compatibility in solution with other medicines has not been evaluated. The total volume of infusion should be delivered over a period of 60 minutes.

Use aseptic technique.

Prepare VPRIV as follows:

1. The number of vials to be reconstituted is determined based on the individual patient?s weight and the prescribed dose.

2. The required vials are removed from the refrigerator. Each vial is reconstituted using sterile water for injections: Vial size Water for Injections 200 Units 2.2 ml 400 Units 4.3 ml

3. Upon reconstitution, mix vials gently. Do not shake.

4. Prior to dilution, visually inspect the solution in the vials; the solution should be clear to slightly opalescent and colourless; do not use if the solution is discoloured, or if foreign particulate matter is present.

5. The calculated volume of medicinal product is withdrawn from the appropriate number of vials. Some solution will remain in the vial: Vial size Extractable volume 200 Units 2.0 ml 400 Units 4.0 ml

6. The total volume required is diluted in 100 ml of sodium chloride 9 mg/ml (0.9%) solution for infusion. Mix gently. Do not shake. The infusion should be initiated within 24 hours from the time of reconstitution.

From a microbiological point of view, the medicine should be used immediately. If not used immediately, in-use storage times and conditions prior to use are the responsibility of the user and must not exceed 24 hours at 2°C to 8°C.

Medicines should not be disposed of via waste water or household waste. Any unused medicine or waste material should be disposed of in accordance with local requirements