New Era in Animal Biotechnology: FDA Approves Therapy Produced with Genetically Engineered Goats
The U.S. Food and Drug Administration granted the first-ever U.S. approval of a product derived from a genetically engineered animal. The product is a protein, developed to treat hereditary antithrombin deficiency, a rare clotting disorder associated with severe complications for which there are few treatment options. To give you more specifics, I’ll quote from the news release that we issued today:
“ATryn®, a recombinant form of human antithrombin developed by GTC Biotherapeutics, was approved by the FDA for the prevention of peri-operative and peri-partum thromboembolic events in hereditary antithrombin deficient patients. It is not indicated for treatment of thromboembolic events in hereditary antithrombin deficient patients. ATryn® is the first ever transgenically produced therapeutic protein and the first recombinant antithrombin approved in the United States. Along with the approval of ATryn®, the FDA’s Center for Veterinary Medicine also approved GTC’s New Animal Drug Application, the first of its kind to regulate genetically engineered animals. This is now required for a recombinant technology used to develop transgenic animals, such as the goats that produce recombinant antithrombin. GTC has granted OVATION the right to market ATryn® in the United States and pursue further clinical development.”
This has been a long road, and I am thrilled because this product will make a difference in the lives of these patients. It also means that the U.S. government regulatory process for genetically engineered animals is effective. The regulatory process was clarified in final guidance released by FDA on January 15, 2009 (link to BIO’s press release) The process ensures the products made available through various applications of genetic engineering will go through a rigorous and transparent review process before being approved for the marketplace. In short, there’s nowhere to go, but up! Biotechnology does indeed heal the world.