The U.S. Food and Drug Administration (FDA) issued its first approval for a biological product produced by genetically engineered (GE) animals. The approval is for ATryn, an anticoagulant used for the prevention of blood clots in patients with a rare disease known as hereditary antithrombin (AT) deficiency. These patients are at high risk of blood clots during medical interventions, such as surgery, and before, during and after childbirth.
ATryn is a therapeutic protein derived from the milk of goats that have been genetically engineered by introducing a segment of DNA into their genes (called a recombinant DNA or rDNA construct) with instructions for the goat to produce human AT in its milk. AT is a protein that naturally occurs in healthy individual and helps to keep blood from clotting in the veins and arteries.
Because hereditary AT deficiency occurs in a small population (approximately 1 in 5,000 people in the United States), the FDA granted ATryn an orphan drug designation. The orphan drug designation system encourages the development of medications for patients with a rare disease or condition.