Its approval would be a major step toward new kinds of medications made not from chemicals, but from living organisms genetically manipulated by scientists. Similar drugs could be available in the next few years for a range of human ailments, including hemophilia.
ATryn was developed by a Massachusetts biotechnology company, GTC Biotherapeutics, 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.
About 1 in 5,000 people don't produce enough of the protein, putting them at risk of developing blood clots in their veins. Such clots can be extremely painful. If they break loose and travel through the bloodstream to the lungs or the brain, the consequences can be catastrophic. Pregnant women with the disorder are at high risk of miscarriage or stillbirth, because of blood clots in the placenta.
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