The US Food and Drug Administration (FDA) on Friday cleared the first medicine made from genetically engineered animals.

 

The drug is called ATryn and belongs to a Massachusetts biotechnology company, GTC Biotherapeutics. It was obtained by altering the genes of goats so they would produce milk rich in antithrombin, a protein that in humans acts as a natural blood thinner.

 

The drug is designed to help people with a rare hereditary disorder that makes them vulnerable to life-threatening blood clots. It is used during surgery and childbirth.

 

The FDA approval follows a January 9 recommendation by outside advisers to the FDA that ATryn be approved for people with hereditary lack of antithrombin. Its use will be limited to about 100,000 patients for the beginning. However, the approval marks the foundation for broader use of genetically engineered animals in medicine.

 

The FDA approval “really takes away one of the biggest issues that have always been on the table, which is how do regulatory agencies view this kind of technology,” said Samir Singh, president of the American operations of Pharming, another company using such technology.

 

“Advancing this novel technology from the early days of demonstrating its capability to the daily practice of producing a safe and efficacious product for the U.S. and the European Union, is a testament to the persistence and capability of our employees,” Geoffrey Cox, GTC’s chairman and CEO, said in a statement.

 

According to estimates of GTC, about 1 in 2,000 to 5,000 people in the US have an inherited antithrombin deficiency. The condition is treated with blood thinners and infusions of human antithrombin extracted from donated human blood.

 

The antithrombin approved by the FDA is produced by a herd of 200 bioengineered goats living under carefully controlled conditions on a farm in central Massachusetts.

 

GTC is also planning studies to test the drug in patients at risk for clots in non-hereditary conditions such as coronary bypass surgery, which if approved later by the FDA could boost sales, said Geoffrey Cox, chairman of GTC.