Monday, January 12, 2009

FDA panel pushes GTC transgenic drug toward approval

By Marc Songini

An advisory committee for the U.S. Food and Drug Administration has given the thumbs up for a key technology from transgenic therapeutics provider GTC Biotherapeutics Inc.

Framingham-based GTC (Nasdaq: GTCB) announced its ATryn antithrombin recombinant had been approved by the FDA’s Blood Products Advisory Committee. More specifically, the committee had voted that ATryn is “safe and efficacious” to prevent and treat venous thromboembolism in hereditary antithrombin deficient patients facing surgery or childbirth.

This is a major hurdle for GTC, as the FDA considers the recommendations of advisory committees when making its final decisions. Should ATryn be cleared, it will be the first recombinant human antithrombin available in the U.S., GTC officials said. It is already the first transgenically produced therapeutic approved in the European Union. The FDA’s target date for action on GTC’s Biologic License Application is Feb. 7, 2009; GTC has licensed ATryn to Ovation Pharmaceuticals Inc. for subsequent development and marketing in the U.S.

Antithrombin functions as a natural anticoagulant by regulating thrombin, which helps control blood clot formation. ATryn provides a reliable, pure and consistent supply of recombinant antithrombin. according to GTC officials. Those patients with hereditary antithrombin deficiency are at increased risk for venous blood clots, including pulmonary embolism and deep vein thrombosis. About one in 2,000 to one in 3,000 persons are likely to have hereditary antithrombin deficiency. Half of them are likely to suffer thrombosis before 25; one study indicated up to 85 percent may suffer a thromboembolic event by 50.

Last month, GTC announced shareholder approval of three proposals, relating to a $15 million financing agreement with French firm LFB Biotechnologies. This included a proposal for LFB to purchase $15 million in GTC common stock and convertible debt.