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U.S. Department of Health and Human Services

Animal & Veterinary

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FDA Approves First Human Biologic Produced by GE Animals

FDA Veterinarian Newsletter 2008 Volume XXIII, No VI

When people think of life-saving products approved by the Food and Drug Administration for use in human medicine, goats don’t usually come to mind. But for a person affected by a rare clotting disorder called hereditary antithrombin (AT) deficiency, a group of goats living in Framingham, MA, may be a true life saver.

These goats are genetically engineered (GE) to produce human AT in their milk. After the human AT is purified from the goats’ milk, the biological product, called ATryn, is used as an anticoagulant to prevent blood clots in patients with hereditary AT deficiency. People living with this disorder are at high risk of developing life-threatening blood clots, especially during pregnancy, surgery, or prolonged bed rest. The approval of ATryn provides patients with hereditary AT deficiency a new and reliable source of the anticoagulant.

FDA approved ATryn on February 6, 2009, and it is the first approval of a human biological product produced by GE animals in the United States. In a joint effort between the FDA’s Center for Biologics Evaluation and Research (CBER) and Center for Veterinary Medicine, the manufacturer of ATryn, GTC Biotherapeutics, Inc., in Framingham, MA, received two approvals: one from CBER for the human anticoagulant and one from CVM for the recombinant DNA (rDNA) construct in the GE goats.

CBER approved the human anticoagulant based on its safety and effectiveness in humans, while CVM approved the rDNA construct based on its safety and ability to consistently produce the human AT over seven generations of the GE goats. The rDNA construct is a segment of DNA that, when introduced and expressed in the goats, gives them the ability to produce the human anticoagulant in their milk.

CVM determined that the introduction and expression of the rDNA construct in the goats do not pose any health risk to the animals and that the GE goats do not significantly impact the environment.

Neither the GE goats nor any products derived from them are intended to be consumed as food, and CVM made sure that there are adequate procedures in place to prevent the GE goats and their products from entering the food supply. In addition, CVM validated GTC’s method for identifying the rDNA construct in both animals and their products and concurred with GTC’s durability plan for post-approval monitoring of the rDNA construct and its expression.