A treatment for Fabry disease received U.S. Food and Drug Administration (FDA) approval for marketing on April 25, 2003. Genzyme General's drug Fabrazyme (agalsidase beta), which has already been available in many other countries, will now be sold in the U.S. This approval ended the competition with Transkaryotic Therapies, Inc., to receive U.S. marketing approval for a treatment for Fabry disease (see articles, The Fabry Enzyme Race and The Fabry Enzyme Race Heats Up).
As part of its agreement with the FDA, Genzyme will continue to study Fabry disease, the effects of Fabrazyme, and will include children in its studies.
Fabry disease is a fat storage disorder caused by a deficiency of an enzyme, alpha-galactosidase A, involved in the breakdown of fats. Since fat doesn't break down properly, part of it (globotriaosylceramide, also called Gb3 or GL-3) accumulates in organs of the body and causes damage to the kidneys, heart, and brain, and causes pain throughout the body. Fabrazyme was designed to replace the deficient enzyme.
First specific treatment for Hurler syndrome
On April 30, 2003, a treatment for mucopolysaccharidosis I (MPS I) received U.S. FDA approval for marketing. Aldurazyme (laronidase) is an intravenous infusion given once per week for life to people with MPS I. Based on the results of clinical studies treatment with Aldurazyme will offer a great improvement in the lives of individuals affected by MPS I.
As part of its agreement with the FDA, Genzyme will continue to study MPS I, the effects of Aldurazyme, and will conclude its study of the use of Aldurazyme in children under 5 years old. Aldurazyme has also been approved in the 15 European Union countries, Iceland and Norway.
In MPS I, a storage disorder, the enzyme alpha-L-iduronidase is deficient. This enzyme breaks down long chains of sugar molecules so the body can dispose of them. Without the enzyme, the big molecules build up and progressively damage parts of the body. MPS I may range from mild, called Scheie syndrome, to severe, called Hurler syndrome).
Last updated 1/1/2006
