What is it and how is it used?
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.
What do you have to consider before using it?
Do not use VPRIV
Take special care with VPRIV
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.
How is it used?
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.
What are 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 occured. If any of these happen tell your doctor immediately.
In studies with VPRIV the following side effects were reported:
The very common side effects are:
The common side effects are:
If any of the side effects gets serious, or if you notice any side effects not listed in this leaflet, please tell your doctor.
How should it be stored?
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.
