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| |  FDA Grants Orphan Drug Designation to Recombinant Human Antithrombin III for Anticoagulation FRAMINGHAM, MA and CAMBRIDGE, MA -- May 9, 2000 -- Genzyme Transgenics Corp. (Nasdaq: GZTC) and Genzyme General announced that the United States Food and Drug Administration has granted Orphan Drug designation for their product recombinant human antithrombin III (rhATIII) for the treatment of ATIII dependent heparin resistant patients requiring anticoagulation. Orphan Drug designation for rhATIII provides Genzyme Transgenics and Genzyme General with seven years of marketing exclusivity upon receipt of FDA approval. Heparin is used as an anticoagulant in patients undergoing cardiopulmonary bypass (CPB) to prevent clotting, which can occur when blood comes into contact with the tubing and artificial surfaces of the heart-lung machine used to take over the heart's function during surgery. The binding of heparin to ATIII results in an increase in thrombin inhibitory activity. In January 2000, the companies announced primary endpoint results from their phase III clinical study evaluating the ability of transgenically produced rhATIII to provide effective anticoagulation in heparin resistant patients undergoing elective heart surgery requiring cardiopulmonary bypass. This study marks the first time a transgenic drug candidate has advanced to and completed a phase III clinical trial evaluation. The companies have completed enrollment in the second, identically designed phase III clinical trial and expect the analysis of the results to be completed later this year. "We are extremely pleased to have received Orphan Drug designation for rhATIII in this indication," said Jan van Heek, executive vice president, Genzyme Corporation. "We are very encouraged by the results of our first rhATIII phase III study and the potential to provide an exciting, novel treatment option for the future." The United States Orphan Drug Act of 1983 was created to provide incentives for companies to develop and market treatments for diseases affecting fewer than 200,000 people in the United States. Under the Orphan Drug Act, more than 200 new drugs have been developed and approved. Genzyme Transgenics and Genzyme General have established a joint venture, ATIII LLC, for the development, marketing, and distribution of rhATIII in the United States and Europe. Genzyme Transgenics Corporation applies transgenic technology to enable the development and production of recombinant proteins and monoclonal antibodies for medical uses. Primedica Corporation, Genzyme Transgenics' contract research organization, provides preclinical development and testing services to pharmaceutical, biotechnology, medical device, and other companies. Genzyme General develops and markets therapeutic products and diagnostic products and services. Genzyme General has three therapeutic products on the market and a strong pipeline of therapeutic products in development focused primarily on the treatment of rare genetic diseases. A division of the biotechnology company Genzyme Corporation, Genzyme General has its own common stock intended to reflect its value and track its economic performance. Related Link: Genzyme Corporation.
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