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| |  ASHG: Replagal (Agalsidase) Safe/Effective for Fabry Disease CAMBRIDGE, MA -- October 16, 2001 -- Transkaryotic Therapies, Inc. today announced results from an open-label extension of its six-month pivotal trial of Replagal™ (agalsidase alfa) enzyme replacement therapy for the treatment of Fabry disease. The data was presented today at the 51st Annual Meeting of the American Society of Human Genetics in San Diego, California. Twenty-five patients with Fabry disease participated in the open-label extension study for an additional 12 months after the conclusion of the six- month, double-blind, placebo-controlled pivotal trial. All patients received 0.2 mg/kg of Replagal by a 40 minute intravenous infusion every two weeks. Dr. Raphael Schiffmann of the National Institutes of Health (NIH), lead investigator of the study, made today's presentation, reporting that treatment with Replagal is well-tolerated, has an excellent safety profile, and could have broad therapeutic effects for patients with Fabry disease. In the initial double-blind trial, patients who were randomized to placebo experienced a significant decline in renal function. This finding is consistent with the natural history of the disease. However, once these patients crossed over to Replagal, Dr. Schiffmann reported that they experienced statistically significant improvements in renal function as measured by glomerular filtration rate (p=0.025). In addition, when treated with Replagal for 12 to 24 months, patients experienced a significant improvement in renal function (p=0.03). Statistically significant improvements were also reported with decreases in pain and globotriaosylceramide (Gb3) levels. Cold and warm detection thresholds significantly improved in both the hand and foot, and body weight significantly increased in both groups. Infusions were very well-tolerated, and there were no new infusion reactions after six months of treatment. All patients were transferred to home therapy at the conclusion of the study and have now been receiving their infusions at home for over one year. "The long-term data presented today confirm and expand upon the results observed in the initial six month study presented last year," said Dr. Schiffmann. "We have demonstrated improvement in multiple outcome measures -- pain management, kidney function, cerebral blood flow, and quality of life measures. Based on these data, we expect that treatment with Replagal could improve and lengthen the lives of patients with Fabry disease." Replagal is a human alpha-galactosidase A produced by genetic engineering technology in a human cell line. Patients receive 0.2 mg/kg of Replagal every other week over a 40 minute intravenous infusion. In the United States and Europe, many patients receive Replagal at home rather than in a hospital setting. Fabry disease is an inherited rare genetic disorder caused by deficient activity of the lysosomal enzyme alpha-galactosidase A affecting both males and females. In patients with Fabry disease, Gb3 accumulates in various organs and tissues of the body due to the deficiency of alpha-galactosidase A. Many cells are damaged by Gb3 including epithelial cells of the kidney, myocardial cells, cells of the autonomic nervous system, and endothelial, perithelial, and smooth muscle cells in the large vessels. As a result, the deposits of this material can result in extreme pain, severe kidney damage, cardiovascular disease, and stroke. Due to its rarity and vast array of symptoms, diagnosis is often difficult and affected individuals have a significantly reduced quality of life and a greatly shortened life expectancy. SOURCE: Transkaryotic Therapies, Inc. Related Link: Transkaryotic Therapies, Inc.
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