March 15, 2021

The U.S. FDA has approved an expanded indication for Fabrazyme® (agalsidase beta), manufactured by Genzyme, to treat patients who are at least two years old and have confirmed Fabry disease. The drug was previously approved only for use in patients eight years of age and up.

A rare genetic disorder, Fabry disease stops the body from making alpha-galactosidase A, an enzyme needed to break down a type of fat known as globotriaosylceramide (GL3). This in turn prevents the body’s cells from using GL3 properly, causing it to build up in the tissues. The damage caused by the excess GL3 can lead to chronic pain, vision problems, hearing loss, kidney damage, increased risk of heart attack and stroke, and gastrointestinal problems.

Fabrazyme, which first received FDA approval in 2003, works by reducing the amount of GL3 in the body. Recommended dosing is 1mg/kg of the patient’s body weight administered as an intravenous (IV) infusion once every two weeks. The infusion rate is based both on the patient’s weight and how well treatment is tolerated. Appropriate medical support must be available at the time of administration due to the potential for anaphylaxis and severe infusion-associated reactions.

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