The U.S. Food and Drug Administration (FDA) has granted orphan drug status to a new product designed to treat patients with an inherited form of thrombophilia known as hereditary antithrombin deficiency (HD). The drug, called ATryn®, is manufactured by GTC Biotherapeutics, Inc., based in Framingham, MA.
ATryn® is a recombinant form of the anticoagulant protein antithrombin, which works by performing the clot-moderating role normally played by the antithrombin found in human plasma. 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 where it lodges--a serious and sometimes fatal complication.
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 a new alternative for patients who depend on human plasma supplies. The goats 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.
“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,” said 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.”
The FDA’s Office of Orphan Products Development grants orphan drug status, which means the drugs go through an expedited review process. The designation allows for accelerated approval, so that much-needed drugs or biologics become available to patients with rare, life-threatening diseases who need effective treatments. More than 1,400 drugs and biologics have been designated as orphan drugs. Since 1983 more than 250 of them have been approved for marketing.
Source: GTC Biotherapeutics, Inc. press release dated December 10, 2007