Patient information

VPRIV 400 Units powder for solution for infusion (velaglucerase alfa)

Read this information carefully before you start using this medicine.

• If you have any further questions, ask your doctor
• If any of the side effects gets serious, or if you notice any side effects not listed below, please tell your doctor

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 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.

2. Before you use VPRIV

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 breastfeeding

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

3. How to use VPRIV

VPRIV is only to be used under appropriate medical supervision of a doctor who is knowledgable 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 9mg/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.

4. Possible side effects

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 occurred. If any of these happen tell your doctor immediately.

The frequency of possible side effects listed below is defined using the following convention:

Frequency of possible side effects

Very common	affects more than 1 user in 10
Common	affects 1 to 10 users in 100
Uncommon	affects 1 to 10 users in 1,000
Rare	affects 1 to 10 users in 10,000
Very rare	affects less than 1 user in 10,000
Not known	frequency cannot be estimated from the available data

In studies with VPRIV the following side effects were reported:

The very common side effects are:

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

5. How to store VPRIV

• 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.

6. Further information

What VPRIV contains

• The active substance is 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 400 Units powder is packaged in a 20 ml glass vial, with 1 vial per carton.

Marketing Authorisation Holder and Manufacturer

Marketing Authorisation Holder:

Shire Pharmaceuticals Ireland Limited
5 Riverwalk
Citywest Business Campus
Dublin 24
Ireland

Manufacturer:

Shire Human Genetic Therapies AB
Åldermansgatan 13
227 64 Lund
Sweden

This information was last approved in August 2010

Detailed information on this medicine is available on the European Medicines Agency web site: http://www.ema.europa.eu. There are also links to other websites about rare diseases and treatments.

Reference

1. VPRIV Patient information leaflet.