FAQs About Fabrazyme for Fabry Disease

Fabrazyme is an approved treatment for Fabry disease patients age 8 and older. It was developed by Sanofi Genzyme. Following are answers to some frequently asked questions about the treatment.

How does Fabrazyme work?

Fabrazyme is an enzyme replacement therapy. In patients with Fabry disease, there is a GLA gene mutation in which cells cannot produce enough functional alpha-galactosidase A enzyme. As a result, a molecule called globotriaosylceramide (Gb3 or GL-3) builds up in the body. Fabrazyme contains the enzyme agalsidase beta, which is similar to alpha-galactosidase A. It can therefore lower GL-3 levels in the body.

How will I take it and how often?

Fabrazyme is an intravenous (IV) infusion that you take every two weeks. The first few infusions occur at a doctor’s office or infusion center so that you can be monitored for any reactions. Eventually, you may be able to have the infusions at home with the help of a nurse.

Does Fabrazyme work in all patients?

A clinical trial in 58 patients, ages 16 to 61, found that after five months of treatment with Fabrazyme 69% of patients had normal or near-normal levels of GL-3 in the blood vessels of their kidneys. GL-3 levels were reduced to normal or near-normal in the heart in 72% of patients and 100% of patients had normal levels of GL-3 in their skin.

A different study in 16 children, ages 8 to 16, found that 12 of the patients had elevated levels of GL-3 in the blood vessels of their skin. At 24 and 48 weeks of treatment, all of the children had normal or near-normal levels of GL-3 in their skin.

Are there side effects?

In very rare cases, Fabrazyme may cause a serious allergic reaction called anaphylaxis.

You also may experience side effects related to the infusion itself. However, these should subside the longer you use Fabrazyme. To reduce the risk of having an infusion-related reaction, your doctor may give you acetaminophen and antihistamines before the infusion. Slowing the rate of infusion may help.

The most common side effects of Fabrazyme are upper respiratory infections, chills, fever, headache cough, a burning or tingling sensation in the hands and feet, fatigue, swelling of the limbs, dizziness and rash.

Are there reasons I shouldn’t take Fabrazyme?

Patients with heart problems may be at greater risk of infusion-related side effects and should be monitored more closely during infusions.

Researchers have not evaluated Fabrazyme in children younger than 8 or in adults older than 65.

There also is little data available about women who become pregnant while taking Fabrazyme. However, they have not shown any increased risk of miscarriage, major birth defects, or risk of complications for the mother or child. There currently is no data about the effects of taking Fabrazyme while breastfeeding.

Last updated: Jan. 22, 2021

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Fabry Disease News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.