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| |  France Allows Fabrazyme (Agalsidase Beta) Treatment For Fabry Disease CAMBRIDGE, MA -- January 18, 2001 -- Genzyme General, a division of Genzyme Corp., announced that it has begun treating patients in France with Fabrazyme™ (agalsidase beta) under an Authorisation Temporaire d'utilisation (ATU) from the French Medicines Agency. The ATU allows patients with Fabry disease in France to receive treatment with Fabrazyme before marketing authorization for the product is granted in the European Union. Genzyme has also secured reimbursement for Fabrazyme through the French social security system. Fabrazyme is Genzyme's investigational enzyme replacement therapy for patients with Fabry disease. An ATU is granted in France for products deemed to be important in treating certain debilitating or life-threatening diseases for which no effective treatment is available. Genzyme has submitted a marketing application for Fabrazyme in the European Union and a decision by regulatory authorities is anticipated during the first half of this year. "We are grateful to the French authorities for expediting the processes that have made it possible for Fabry patients in France to begin treatment with Fabrazyme," said Richard A. Moscicki, M.D., senior vice president and chief medical officer at Genzyme Corp. Genzyme General also announced today the launch of a global registry that will serve as a repository of medical information on Fabry disease. The data collected will be used to advance the understanding of Fabry disease, evaluate the impact of treatment, support the establishment of standard treatment models, and improve the quality of care for Fabry patients through publication of registry findings. "The Fabry registry is a key component of Genzyme's commitment to provide resources and support to physicians who are caring for Fabry patients," said Dr. Moscicki. "Drawing on the success of our Gaucher disease registry, we will work to make the Fabry registry the largest source of clinical information on Fabry disease in the world." Fabry disease is caused by a deficiency of the enzyme alpha-galactosidase. This deficiency results in the body's inability to break down certain glycolipids which then accumulate in the lining of the blood vessels within the kidney, heart, skin and other organs. The progressive accumulation of these lipids in the blood vessels results in symptoms such as kidney failure, stroke, cardiovascular disease, severe pain and numbness. Due to organ complications, death typically occurs around age 40 for the estimated 2,000- 4,000 people worldwide afflicted with the disorder. Fabrazyme is a recombinant form of alpha-galactosidase intended to replace the deficient enzyme and prevent the lipid accumulation that leads to the devastating symptoms of Fabry disease. Genzyme General is currently seeking approval to market Fabrazyme in the United States, Europe, and Australia. Fabrazyme has received orphan drug designation in the United States, Europe, Japan, and Australia. Genzyme General has three therapeutic products on the market and a strong pipeline of therapeutic products in development focused on the treatment of genetic disorders and other chronic debilitating diseases with well-defined patient populations. Genzyme General is a division of the biotechnology company Genzyme Corporation. Related Link: Genzyme Corporation.
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