GTC Biotherapeutics and Ovation Pharmaceuticals, Inc. have announced hat they are on the verge of receiving the US FDA approval for ATryn to treat hereditary antithrombin deficiency, a rare clotting disorder.
An expert panel voted overwhelmingly Friday that it is safe and effective. Atryn will become the first drug from a genetically engineered animal to be approved in the U.S. The agency is expected to make a final decision in early February.
GTC is raising genetically engineered goats on a farm in central Massachusetts to make a human protein in their milk that prevents dangerous blood clots from forming. The company extracts the protein and turns it into Atryn to fight strokes, pulmonary embolisms and other life-threatening conditions.
“We are very pleased with the Advisory Committee’s recommendation in support of the safety and efficacy of ATryn,” said Geoffrey F. Cox, GTC’s Chairman and Chief Executive Officer. “ATryn is the first transgenically produced therapeutic to achieve approval in Europe and undergo review by the FDA. ATryn is also a testament to our established strength in recombinant technology, and has the potential to provide an important new treatment option for patients with hereditary antithrombin deficiency.”
"The committee’s recommendation takes us a step closer to making ATryn available to people in the U.S. with hereditary antithrombin deficiency, a rare clotting disorder associated with severe complications for which there are few treatment options," said Jeffrey S. Aronin, Ovation President and Chief Executive Officer. “Consistent with our overall focus on addressing unmet medical needs of small patient populations, bringing ATryn to market would give us the opportunity to make a meaningful difference in the lives of people suffering from this rare disorder.”
