ATryn: Biotech Trailblazer
A little over a year ago, GTC Biotherapeutics successfully won orphan drug status for a new product they were developing called ATryn. The drug is intended to treat people suffering from a rare disease called “Hereditary Antithrombin Deficiency,” 
a genetic disorder leading to poorly functioning or diminished levels of antithrombin, a condition that can lead to excessive clotting.” (1) Obviously, the disease can be serious and life-threatening because of its ability to spontaneously cause blood clots that can block normal organ operation or put fetuses at serious risk in pregnant women. However, because of its rarity, few but the smallest drug companies could undertake the task of coming up with a viable, sustainable method for producing a drug that could treat the disease. An article in Scientific American entitled, “Atryn, on Old MacDonald’s Pharm,” estimated a market of about $50 million, small by comparison to the multi-billion dollar drugs typified by big pharma’s pipeline. But $50 million is $50 million, and GTC thought they could develop a solution.
On February 9th, 2009, the FDA granted approval to the partnership of Ovation Pharmaceuticals and GTC Biotherapeutics to bring ATryn to market for treatment of the disease. This is a pretty standard procedure for bringing a drug to market. This drug is special, however, because it’s the “first ever transgenically produced therapeutic protein and the first recombinant antithrombin approved in the U.S.” (2)
Transgenics probably strikes at the heart of what biotechnology is and its promise to bring better medications into market more efficiently. In the case of the partnership between Ovation and GTC, goats and chickens were genetically altered to produce the protein for the drug in their milk and eggs, respectively, and at far less of a cost than traditional biotechnology methods using mamillian cells grown in a controlled environment. They essentially intervene when the animal is an embryo and allow nature to take its course from there to develop a viable and consistent carrier of the necessary protein. The biggest expense is maintenance of the farm where the animals are brought up, and the feed necessary in order to keep them nourished — embryonic modification and protein extraction from the milk apparently are not. Scientific American estimates the actual cost using transgenic methodology at about tens of millions of dollars, as opposed to the hundreds of millions necessary to follow the traditional methodology. (3)
There are some counterindications that have appeared in clinical studies, such as a possibility of allergic reaction in those people who have a hypersensitivity to goat’s milk, but according to the press release more information will be necessary through close studies of patients who receive repeated doses over a longer period of time.
Genetic modification is a scary thing, and it’s tough not to consider the moral implications of the territory in which we are about to embark. However, I do believe that we are entering a really exciting time in the medical industry thanks in large part to the ideas pioneered by biotechnology, and it seems a normal evolutionary step in the development of our collective knowledge of how things work and how we can get them to work for us. As evidenced in my post last week, “Superbugs Rising,” traditional approaches are starting to show their age, and we must be nimble in order to ensure we stay ahead of what ails us.
What do you think?
(1) www.ATIII.com, a resource for information on Hereditary Antithrombin Deficiency, home page.
(2) See, “ATryn® (Antithrombin [Recombinant]) Approved by the FDA,” press release, , February 6, 2009.
(3) See, “ATryn, on Old MacDonald’s Pharm,” Charles Choi, Scientific American, January 10, 2009.
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