Agalsidase alfa

Agalsidase alfa is a recombinant DNA derived, enzyme replacement therapy (ERT) treatment for Fabry disease. It is marketed under the trade name Fabrazyme by Sanofi Genzyme.

Overview

Fabry disease is a rare, inherited disorder caused by a deficiency of the enzyme alpha-galactosidase A (α-Gal A). This deficiency leads to the accumulation of a specific type of fat, known as globotriaosylceramide (GL-3), in the body's cells. Over time, this accumulation can cause a range of symptoms and complications, including pain, kidney failure, and increased risk of heart attack and stroke.

Agalsidase alfa is a form of ERT that works by replacing the deficient α-Gal A enzyme. This allows the body to break down GL-3, preventing or slowing the progression of Fabry disease.

Clinical Use

Agalsidase alfa is administered intravenously, typically every two weeks. The dosage may be adjusted based on the patient's body weight and overall health. Like all medications, agalsidase alfa can cause side effects, the most common of which include fever, chills, nausea, and fatigue.

Efficacy and Safety

Clinical trials have demonstrated that agalsidase alfa can significantly reduce GL-3 accumulation in the body. It has also been shown to improve kidney function and reduce pain in people with Fabry disease. However, some patients may develop antibodies to the medication, which can reduce its effectiveness.

Regulatory Status

Agalsidase alfa was approved by the U.S. Food and Drug Administration (FDA) in 2003 and by the European Medicines Agency (EMA) in 2001. It is currently the only FDA-approved treatment for Fabry disease.

See Also

• Enzyme replacement therapy
• Fabry disease
• Sanofi Genzyme