The FDA has approved Fabrazyme (agalsidase beta), the first treatment for people with Fabry's disease. This serious genetic metabolic disorder affects about 1 in 40,000 men. Though it is believed that fewer women suffer the most serious consequences of the disease, they can also be seriously affected.
Because of a deficiency in the enzyme alpha-galactosidase A, Fabry's disease causes certain fats to accumulate in the blood vessels over many years, damaging various tissues and organs such as the kidneys and heart. As a result, people with Fabry's disease often must cope with significant pain and disability, and they typically have a shortened life span.
Fabrazyme is a version of the human form of the natural enzyme produced by recombinant DNA technology. When given intravenously, this replacement of the missing enzyme reduces a particular type of fat accumulation in many types of cells, including blood vessels in the kidneys and other organs. It's believed that this reduction of fat deposition will prevent the development of life-threatening organ damage.
The FDA approved Fabrazyme in April under an early approval mechanism. This policy accelerates approval for therapies that treat serious or life-threatening illnesses when studies indicate that early favorable outcomes are likely to predict clinical benefit. In this case, the manufacturer of Fabrazyme, Genzyme Corp. of Cambridge, Mass., performed biopsies looking at the cells lining the blood vessels within the kidneys and other organs in people with Fabry's disease. Many of the cells examined have shown significant clearance of fat deposits. Genzyme has committed to continue its clinical trial to verify Fabrazyme's benefit, and has set up a voluntary patient registry to follow the long-term progress of people treated with Fabrazyme.
COPYRIGHT 2003 U.S. Government Printing Office
COPYRIGHT 2004 Gale Group
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