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FDA Approves ATryn®, First Drug from Genetically Engineered Animals

On Saturday, February 6, 2009, the U.S. Food and Drug Administration (FDA) approved ATryn® , an anticoagulant produced by genetically engineered goats, to prevent blood clots in people with hereditary antithrombin deficiency (HD), a rare disorder. People with HD are prone to blood clot developing during surgery and childbirth. ATryn® is the first recombinant human antithrombin product available in the U.S. It is manufactured by GTC Biotherapeutics, Inc., of Framingham, MA, and will be launched commercially by OVATION Pharmaceuticals, Inc., of Deerfield, IL.

In HD patients, inadequate production of the protein antithrombin can increase susceptibility to excessive blood clot formation, which may trigger VTE, either deep-vein thrombosis (DVT) or pulmonary thromboembolism (PE). DVT occurs when a clot forms deep in veins, usually in the lower extremities (legs, thighs or hip area), often causing pain and swelling. When a clot breaks free, it can travel through the bloodstream and lodge in the lungs, causing a pulmonary embolism. This is a serious complication that can result in permanent damage to the lungs and, in some cases, death.

ATryn® is produced in the milk of goats that are genetically engineered before birth to produce antithrombin. 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. The goats used to manufacture ATryn® are called “transgenic animals,” meaning they have received altered genetic material inserted with DNA technology to elicit 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.

Clinical studies in 31 HD patients showed that 30 of them experienced prevention of clots before, during and after childbirth or surgery. Side effects, experienced by 5% of the patients, included hemorrhage and a reaction at the infusion site.

ATryn® was previously granted orphan drug status by the FDA. This designation accelerates the process by which manufacturers produce products for relatively small populations of affected people. HD is a rare condition; estimates of its occurrence range from one in 2,000 to one in 5,000 people in the U.S.

“This product offers an important new treatment option for patients with hereditary antithrombin deficiency, preventing life-threatening clots that otherwise frequently occur during high-risk situations,” said Jesse Goodman, MD, MPH, Director of the FDA’s Center for Biologics Evaluation and Research., which approves human biologics based on safety and efficacy.

Source: FDA press release dated February 6, 2009, Medical News Today press release, dated February 9, 2009