Fabrazyme

What is Fabrazyme used for?

Fabrazyme is a prescription medicine that is used to treat Fabry disease.

Description

Agalsidase beta is a recombinant human α-galactosidase A enzyme with the same amino acid sequence as the native enzyme. Purified agalsidase beta is a homodimeric glycoprotein with a molecular weight of approximately 100 kD. The mature protein is comprised of two subunits of 398 amino acids (approximately 51 kD), each of which contains three N-linked glycosylation sites. The enzyme α-galactosidase A catalyzes the hydrolysis of GL-3 and other α-galactyl-terminated neutral glycosphingolipids, such as galabiosylceramide and blood group B substances to ceramide dihexoside and galactose. The specific activity of agalsidase beta is approximately 70 U/mg (one unit is defined as the amount of activity that results in the hydrolysis of 1 µmole of a synthetic substrate, p-nitrophenyl-α-D-galactopyranoside, per minute under the assay conditions).

Agalsidase beta is produced by recombinant DNA technology in a Chinese hamster ovary mammalian cell expression system.

Fabrazyme (agalsidase beta) for injection is intended for intravenous infusion. It is supplied as a sterile, nonpyrogenic, preservative-free, white to off-white, lyophilized cake or powder for reconstitution with Sterile Water for Injection, USP. Each 35 mg vial contains 37 mg of agalsidase beta, as well as 222 mg mannitol, 20.4 mg sodium phosphate monobasic monohydrate, and 59.2 mg sodium phosphate dibasic heptahydrate. Following reconstitution as directed, 35 mg of agalsidase beta (7 mL) may be extracted from each 35 mg vial.

Each 5 mg vial contains 5.5 mg of agalsidase beta, as well as 33.0 mg mannitol, 3.0 mg sodium phosphate monobasic monohydrate, and 8.8 mg sodium phosphate dibasic heptahydrate. Following reconstitution as directed, 5 mg of agalsidase beta (1 mL) may be extracted from each 5 mg vial.

Mechanism of Action

Fabrazyme (agalsidase beta) provides an exogenous source of α-galactosidase A in Fabry disease patients. Agalsidase beta is internalized and transported into lysosomes where it exerts enzymatic activity and reduces accumulated GL-3.

Before taking Fabrazyme, tell your doctor:

• If you are allergic to Fabrazyme; any part of this medicine; or any other drugs, foods, or substances. Tell your doctor about the allergy and what signs you had.

This medicine may interact with other drugs or health problems.

Tell your doctor and pharmacist about all of your drugs (prescription or OTC, natural products, vitamins) and health problems. You must check to make sure that it is safe for you to take Fabrazyme with all of your drugs and health problems. Do not start, stop, or change the dose of any drug without checking with your doctor.

What are some things I need to know or do while I take Fabrazyme?

• Tell all of your health care providers that you take Fabrazyme. This includes your doctors, nurses, pharmacists, and dentists.
• Allergic reactions have happened with Fabrazyme. Rarely, some reactions can be very bad or life-threatening. Talk with the doctor.
• Other drugs may be given before Fabrazyme to help avoid side effects.
• Tell your doctor if you are pregnant or plan on getting pregnant. You will need to talk about the benefits and risks of using Fabrazyme while you are pregnant.
• Tell your doctor if you are breast-feeding. You will need to talk about any risks to your baby.

How is Fabrazyme best taken?

Use Fabrazyme as ordered by your doctor. Read all information given to you. Follow all instructions closely.

• It is given as an infusion into a vein over a period of time.

What do I do if I miss a dose?

• Call your doctor to find out what to do.

What are the side effects of Fabrazyme that I need to call my doctor about immediately?

WARNING/CAUTION: Even though it may be rare, some people may have very bad and sometimes deadly side effects when taking a drug. Tell your doctor or get medical help right away if you have any of the following signs or symptoms that may be related to a very bad side effect:

• Signs of an allergic reaction, like rash; hives; itching; red, swollen, blistered, or peeling skin with or without fever; wheezing; tightness in the chest or throat; trouble breathing, swallowing, or talking; unusual hoarseness; or swelling of the mouth, face, lips, tongue, or throat.
• Change in hearing.
• Hearing loss.
• Ringing in ears.
• Low mood (depression).
• Dizziness or passing out.
• Shortness of breath.
• Some patients have severe side effects during the infusion. Tell your doctor if you have a burning, numbness, or tingling feeling that is not normal; chest pain; fast or slow heartbeat; feeling hot or cold; feeling sleepy; fever or chills; flushing; headache; diarrhea; muscle pain or weakness; pain in the arms or legs; pale skin; stomach pain; stuffy nose; swelling; tiredness; upset stomach or throwing up; or any other bad effects during the infusion.

What are some other side effects of Fabrazyme?

All drugs may cause side effects. However, many people have no side effects or only have minor side effects. Call your doctor or get medical help if any of these side effects or any other side effects bother you or do not go away:

• Signs of a common cold.
• Cough.
• Back pain.
• Anxiety.
• Muscle spasm.
• Throat irritation.
• Tooth pain.

These are not all of the side effects that may occur. If you have questions about side effects, call your doctor. Call your doctor for medical advice about side effects.

You may report side effects to the FDA at 1-800-332-1088. You may also report side effects at https://www.fda.gov/medwatch.

If overdose is suspected:

If you think there has been an overdose, call your poison control center or get medical care right away. Be ready to tell or show what was taken, how much, and when it happened.

How do I store and/or throw out Fabrazyme?

• If you need to store Fabrazyme at home, talk with your doctor, nurse, or pharmacist about how to store it.

Label

PRINCIPAL DISPLAY PANEL – 5 MG VIAL CARTON

• NDC 58468-0041-1     Rx only
• Fabrazyme®
  agalsidase beta
• 5 mg
• For Intravenous Infusion Only
• One single-dose vial
• Discard unused portion
• SANOFI GENZYME

PRINCIPAL DISPLAY PANEL – 35 MG VIAL CARTON

• NDC 58468-0040-1     Rx only
• Fabrazyme®
  agalsidase beta
• 35 mg
• For Intravenous Infusion Only
• One single-dose vial
• Discard unused portion
• SANOFI GENZYME