The US Food and Drug Administration (FDA) has approved the first drug made using genetically engineered animals, clearing the way for a new class of medical therapies.
The drug, GTC Biotherapeutics Inc's anti-clotting therapy Atryn, is an intravenous therapy made using a human protein gathered from female goats that have been scientifically altered to produce extra antithrombin, a protein that acts as a natural blood thinner.
Atryn aims to prevent excessive blood clots in patients with a disorder known as hereditary antithrombin deficiency. The Framingham, Massachusetts-based company estimates the disorder affects anywhere from 60,000 to 600,000 people in the US.
People with hereditary antithrombin deficiency are at increased risk for venous thromboembolic events, including pulmonary embolism and deep vein thrombosis, which can be life-threatening, particularly in high risk situations.
The FDA approved Atryn to prevent blood clots from surgery or childbirth in patients with the condition, but not wider use to treat the disorder itself, the company said in a statement.
"The approval of Atryn marks a significant milestone in the development of this innovative recombinant technology and delivers a new therapeutic option to benefit hereditary antithrombin deficient patients who are undergoing surgery or childbirth procedures," said Geoffrey F Cox, GTC's chairman and chief executive officer.
The drug's approval on Friday may be the first step toward new kinds of medications made not from chemicals, but from living organisms altered by scientists.