Jan. 9, 2009 -- An FDA advisory committee today recommended the approval of Atryn, which may become the first approved drug made from genetically engineered animals.
Atryn, which is given by infusion, is for people with a rare condition called hereditary antithrombin deficiency. People with hereditary antithrombin deficiency don't make enough antithrombin, an anticlotting compound. That shortfall of antithrombin puts patients at risk of life-threatening blood clots.
Current treatment involves taking blood thinners and getting infusions of human antithrombin, which is extracted from donated blood.
Atryn comes from goats that were genetically engineered to have human antithrombin III in their milk.
GTC Biotherapeutics, the company that makes Atryn, developed Atryn by injecting human antithrombin DNA into fertilized goat cells and implanting them in female goats. The genetically engineered offspring were bred for the presence of antithrombin in their milk.
Patients won't be drinking that goat milk; Atryn is made from the antithrombin harvested from the goat milk.
Atryn was tested in men and women with hereditary antithrombin deficiency who were at risk for blood clots; some of those patients were pregnant and others had recently had surgery. Atryn was safe and effective, but there isn't a lot of data yet on repeat use of Atryn, according to background information from GTC Biotherapeutics.
Today, an independent panel of experts concluded that Atryn appears safe and effective. The panel also recommended that if Atryn is approved, postmarketing safety studies should be done.
Atryn isn't going on the market yet. The FDA considers the recommendations of its advisory panels, but it's not obligated to follow that advice.