Healthcare Industry News:  fast track designation 

Biopharmaceuticals FDA

 News Release - December 10, 2007

GTC Obtains FDA Orphan Drug Designation For ATryn(R)

FRAMINGHAM, Mass.--(HSMN NewsFeed)--GTC Biotherapeutics, Inc. ("GTC", Nasdaq: GTCB) announced today that the US Food and Drug Administration, or FDA, has designated ATryn® an orphan drug for the treatment of patients with hereditary antithrombin deficiency, or HD.

“ATryn® is the only recombinant human antithrombin product being developed to address the needs of this rare patient population who are at risk of developing serious and potentially life-threatening venous thromboses,” stated Geoffrey F. Cox, PhD, GTC’s Chairman and CEO. “The orphan drug designation is recognition of the importance of our developing a unique therapeutic for this rare patient population. We are close to completion of the Phase III comparative study to treat HD patients at risk for developing deep vein thrombosis or thromboembolism while undergoing surgery or childbirth. We plan to have top line results of the comparative study available in late December to early January, depending on final patient scheduling. The FDA recently granted fast track designation for ATryn® and provided permission for a rolling Biologics License Application, or BLA, submission. We will complete the submission after all clinical data is gathered, analyzed, and available for the BLA, which is planned to be in the first half of 2008.”

ATryn® is GTC’s recombinant form of human antithrombin, a protein with anticoagulant and anti-inflammatory properties that is normally present in human plasma. ATryn® is produced in the milk of goats that have incorporated the human antithrombin gene such that it is only expressed during lactation. This technology enables an alternative supply of antithrombin that is unconstrained by the limited availability of plasma-derived product.

ATryn® has been approved for sale in the European Union for the treatment of HD patients undergoing surgical procedures, marking the first time that any transgenically produced therapeutic protein had been approved anywhere in the world. LEO Pharma A/S, GTC’s commercial and development partner in Europe, Canada and the Middle East, has initiated commercial sales in Europe in the approved indication and initiated a Phase II study of ATryn® in the treatment of patients with disseminated intravascular coagulation, or DIC, associated with severe sepsis. DIC is a large unmet medical need with approximately 500,000 patients in the US and EU each year and up to 50% mortality.

Orphan drug designation provides GTC with seven years of marketing exclusivity upon product licensure and automatic waiver of the FDA's application user fee. The Orphan Drug Act is intended to encourage companies to develop therapies for the treatment of diseases that affect fewer than 200,000 individuals in the United States.

About GTC Biotherapeutics

GTC Biotherapeutics develops, produces, and commercializes therapeutic proteins through transgenic animal technology. In addition to ATryn®, GTC is working with LFB Biotechnologies to develop recombinant forms of human coagulation factor VIIa and a CD20 monoclonal antibody. Additional programs in the development pipeline include recombinant forms of human alpha-1 antitrypsin, human coagulation factors VIII and IX, and a monoclonal antibody to CD137. GTC’s intellectual property includes a patent in the United States through 2021 for the production of any therapeutic protein in the milk of any transgenic mammal. GTC’s transgenic production platform is particularly well suited to enabling cost effective development of proteins that are difficult to express in traditional recombinant production systems as well as those that are required in large volumes. Additional information is available on the GTC web site,

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including without limitation statements regarding the timing of completion of GTC’s current Phase III study of ATryn® and filing of the associated BLA. Such forward-looking statements are subject to a number of risks, uncertainties and other factors that could cause actual results to differ materially from future results expressed or implied by such statements. Factors that may cause such differences include, but are not limited to, the risks and uncertainties discussed in GTC's most recent Annual Report on Form 10-K and its other periodic reports filed with the Securities and Exchange Commission, including the uncertainties associated with conducting clinical studies, and the risks and uncertainties associated with dependence upon the actions of regulatory agencies. GTC cautions investors not to place undue reliance on the forward-looking statements contained in this release. These statements speak only as of the date of this document, and GTC undertakes no obligation to update or revise the statements, except as may be required by law.

Source: GTC Biotherapeutics

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