FRIDAY, April 25, 2003 (HealthDayNews) -- The U.S. Food and Drug Administration has approved the first-ever treatment for a rare genetic disorder that leads to an early death for the majority of its victims, who are mostly male.
People with Fabry disease lack an enzyme -- alpha-galactosidase A -- which causes fat deposits to build up over many years in blood vessels and organs. This leads to debilitating pain and a high-risk of heart and kidney failure.
Fabrazyme (agalsidase beta) replaces the missing enzyme. Given intravenously every two weeks, it cleared the harmful fat deposits in two-thirds of clinical trial recipients, the FDA says. But the agency warns that recipients must be monitored for the possibility of serious reactions to the drug, characterized by fever, chest tightness, blood pressure changes, abdominal pain, and headache.
The medication, produced by Genzyme Corp., was approved under the FDA's Orphan Drug Act, which provides grants to companies that develop products to treat rare "orphan" diseases and allows the drugs to be marketed before their effectiveness is entirely proven. Fabry affects only about 5,000 people worldwide.
Here is the FDA Talk Paper describing the medication. For more information about Fabry disease, visit the National Institute of Neurological Disorders and Stroke.
