The U.S. Food and Drug Administration (FDA) assigned “Priority Review” status to ATryn®, a new drug manufactured by Framingham, MA-based GTC Biotherapeutics, Inc. The product had undergone an expedited review process when it was granted orphan drug status by the FDA in December 2007. ATryn® is designed to treat patients with hereditary antithrombin deficiency (HD), an inherited form of thrombophilia.
ATryn® is a recombinant form of the anticoagulant protein antithrombin. The drug works to enhance the clot-moderating role normally played by the antithrombin found in human plasma and has anti-inflammatory properties. In HD patients, inadequate production of the protein can increase susceptibility to excessive blood clots, which may trigger deep-vein thromboses (DVT) or a thromboembolism, a blood clot that breaks free and travels to another site in the body. ATryn’s projected uses include prophylaxis treatment for patient with DVTs, and in patients with HD who are undergoing high-risk childbirth procedures or surgery.
ATryn® is produced in the milk of goats that are genetically engineered before birth to produce the human blood protein. The milk is then purified and the human protein is extracted from it. This creates a seemingly limitless source of antithrombin, and poses new alternative for patients who depend on human plasma supplies. The goats that are used to manufacture ATryn® are “transgenic animals,” meaning that they have been inserted with a foreign gene using DNA technology to illicit the production of certain proteins. The use of transgenic animals such as cows, mice, sheep, rabbits and pigs, is a growing area of research and development, with the potential for a number of therapeutic proteins. There are no other recombinant forms of antithrombin for these patients on the market.
“We are pleased that the FDA has recognized the inherent advantage of ATryn® as the only recombinant antithrombin product that may become available to patients with hereditary antithrombin deficiency who are at risk of developing serious or potentially life-threatening venous thromboembolic events,” stated Geoffrey F. Cox, PhD, GTC’s Chairman and CEO.
The FDA’s goal for completing review of applications when assigned “Priority Review” is six months from receipt of the complete application. GTC filed the last section of the BLA on August 7, 2008. The drug is licensed to OVATION Pharmaceuticals, Inc., in Deerfield, IL, to develop and market.
Source: GTC Biotherapeutics Inc. press release dated September 4, 2008