Velaglucerase alfa contains an enzyme that occurs naturally in the body in healthy people. Some people lack this enzyme because of a genetic disorder. Velaglucerase alfa helps replace this missing enzyme in people with Type I Gaucher disease.

Gaucher disease is a genetic condition in which the body lacks the enzyme needed to break down certain fatty materials (lipids). Lipids can build up in the body, causing symptoms such as easy bruising or bleeding, weakness, anemia, bone or joint pain, enlarged liver or spleen, or weakened bones that are easily fractured.

Velaglucerase may improve the condition of the liver, spleen, bones, and blood cells in people with Type I Gaucher disease. However, velaglucerase is not a cure for this condition.

Velaglucerase may also be used for purposes not listed in this medication guide.

An allergic reaction may occur during or shortly after infusion of this medicine. Tell your caregivers or get emergency medical help right away if you have any signs of a severe allergic reaction, such as headache, dizziness, fever, nausea, feeling weak or tired, and feeling like you might pass out.

Velaglucerase alfa is given as an infusion into a vein. A healthcare provider will give you this injection.

Velaglucerase alfa is usually given every other week. Follow your doctor's dosing instructions.

Velaglucerase alfa must be given slowly, and the infusion can take at least 1 hour to complete.

Velaglucerase alfa doses are based on weight (especially in children and teenagers). Your dose needs may change if you gain or lose weight.

Usual Adult Dose for Gaucher Disease:

Starting dose for patients naive to enzyme replacement therapy (ERT): 60 units/kg IV every other week

Switching from imiglucerase to velaglucerase alfa: Patients currently being treated with stable doses of imiglucerase may switch by starting this drug at the previous imiglucerase dose 2 weeks after the last imiglucerase dose.

Comments:
-This drug should be administered as a 60-minute IV infusion under the supervision of a healthcare professional.

Use: For long-term ERT for patients with type 1 Gaucher disease

Usual Pediatric Dose for Gaucher Disease:

4 years or older:
-Starting dose for patients naive to ERT: 60 units/kg IV every other week

-Switching from imiglucerase to velaglucerase alfa: Patients currently being treated with stable doses of imiglucerase may switch by starting this drug at the previous imiglucerase dose 2 weeks after the last imiglucerase dose.

Comments:
-This drug should be administered as a 60-minute IV infusion under the supervision of a healthcare professional.

Use: For long-term ERT for patients with type 1 Gaucher disease

Tell your doctor if you are pregnant or breastfeeding.

It is not known whether velaglucerase alfa will harm an unborn baby. However, having untreated Type I Gaucher disease during pregnancy may cause complications such as miscarriage or stillbirth. Type I Gaucher disease also can get worse during pregnancy and may cause medical problems in both mother and baby. The benefit of treating this disease with velaglucerase alfa may outweigh any risks to the baby.

Velaglucerase alfa is not approved for use by anyone younger than 4 years old.

Call your doctor for instructions if you miss an appointment for your velaglucerase alfa injection.

Since velaglucerase alfa is given by a healthcare professional in a medical setting, an overdose is unlikely to occur.

Follow your doctor's instructions about any restrictions on food, beverages, or activity.

Get emergency medical help if you have signs of an allergic reaction: hives; fever; chest discomfort, difficult breathing; swelling of your face, lips, tongue, or throat.

An allergic reaction may occur during or shortly after infusion of velaglucerase alfa. Tell your caregivers or get emergency medical help right away if you have any signs of a severe allergic reaction, such as headache, dizziness, fever, nausea, feeling weak or tired, and feeling like you might pass out.

Common side effects of velaglucerase alfa may include:

• headache;

• dizziness;

• feeling weak or tired;

• nausea, stomach pain;

• joint pain, back pain; or

• fever.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.