Fabrazyme Facts

^{What is Fabrazyme?}

Fabrazyme ^® (agalsidase beta) is a medication designed to replace the alpha-GAL enzyme, which is missing or insufficient in people with Fabry disease. This type of medication is known as an enzyme replacement therapy (or ERT).

^{How Does it Work?}

People with Fabry disease are missing or have insufficient amounts of the alpha-GAL enzyme. Without enough of this enzyme, a fatty substance called globotriaosylceramide (GL-3) builds up in the cells throughout the body.

Fabrazyme is intended to replace the alpha-GAL enzyme, which breaks down accumulated GL-3. Treatment with Fabrazyme every two weeks may improve the signs and symptoms of Fabry disease, since Fabrazyme has been shown to reduce GL-3 levels in certain cells. The relationship between GL-3 reduction and the improvement of specific signs and symptoms, however, has not been established.

^{How is Fabrazyme Treatment Given?}

Fabrazyme is given by intravenous (IV) infusion, meaning that the medication is infused directly into your bloodstream through a vein. Fabrazyme cannot be taken orally as a pill or liquid because it is an enzyme that would be destroyed in your digestive system. Infusion into the bloodstream bypasses your body’s digestive processes and allows Fabrazyme to go to work in cells throughout the body.

^{What is the Recommended Dosage?}

The recommended dosage of Fabrazyme is 1.0 milligram (mg) for each kilogram (kg) of body weight given once every 2 weeks as an IV infusion. For instance, a man weighing 70 kg (approximately 154 pounds) would receive 70 mg of Fabrazyme.

^{What are the Possible Side Effects of Fabrazyme?}

The most serious and common side effects reported with Fabrazyme are infusion reactions, meaning that they occurred as a result of the infusion. Infusion reactions occurred in many people treated with Fabrazyme, and some of the reactions were severe. Side effects may include: increase in heart rate, increase in blood pressure up to a few hours after infusion, throat tightness, chest pain/tightness, shortness of breath, fever, chills, abdominal pain, itching, hives, nausea, vomiting, lip or ear swelling, rash, decrease in blood pressure, muscle pain and headache.

To reduce the potential for an infusion reaction, your health care provider will give you anti-fever medications (for example, acetaminophen or ibuprofen) before the infusion. Infusion reactions occurred in some people after receiving anti-fever medications, antihistamines, and oral steroids.