FDA Panel Gives Blessing to First Drug Manufactured in Bioengineered Goats

1/9/09Follow @xconomy

The first drug ever manufactured in genetically-modified animals is likely coming soon to the U.S. market. A panel of expert advisers to the FDA said today that an experimental anti-clotting product developed by Framingham, MA-based GTC Biotherapeutics looks safe and effective enough to become a marketed product.

The advisers essentially agreed with the conclusions reached by FDA staff, based on documents that were made publicly available Wednesday morning. The FDA still hasn’t made a final decision; its deadline for doing so is Feb. 7.

The product in question, a human protein called antithrombin, is isolated from the milk of goats who have been genetically modified to produce copious amounts of the protein. The drug, marketed as ATryn, has been cleared for sale in Europe since 2006. It hasn’t been a financial boon for GTC, but the company hopes that winning this FDA panel blessing will set a precedent that its manufacturing method, sometimes called “pharming,” can be used for other, more profitable medicines. GTC has signed a partnership with Deerfield, IL-based Ovation Pharmaceuticals who will now gear up in hopes of marketing the drug.

“We are very pleased with the Advisory Committee’s recommendation,” said Geoffrey Cox, CEO of GTC Biotherapeutics, in a statement after the panel’s recommendation.

Pharming’s appeal, as David Stipp wrote for us back in February, comes from its ability to churn out large quantities of protein drugs in a cheap way. Scientists already know how to make genetically engineered protein drugs through fermentation-style techniques using standard lab organisms like E. coli, yeast, or hamster cells, although these methods are more costly and time-consuming than milking a goat.

GTC was able to successfully point out that all protein drugs have to go through a rigorous purification process to separate the drug from all the gunk it brews with. Since they proved the end product is pure and consistent, who cares if it came from a modified goat?

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  • http://drughealth.blogspot.com/ Austin Lee

    This is very cool. We’ve come far along from the days of Chinese hamster ovary cells in bioreactors producting drugs for human use. I wonder how this will be received by the populace? . . . “I’m taking drugs made from what?!” Austin
    http://drughealth.blogspot.com/

  • http://amanwithaphd.wordpress.com/ Richard

    Of course, many drugs are made in yucky things like E. coli or yeast. And, truthfully, the way turkeys are raised is more disquieting than this would be. However, you never know what will get people’s dander up.

  • http://amanwithaphd.wordpress.com/ Richard

    One key thing is that these GMO are very different than many others, especially plants. No one will be eating them. The genetic material is never airborne (as pollen is) so the genes should not end up in the wrong place (like Old McDonald’s place next door).

    Care will have to be taken with waste products and such but I expect these animals to be very highly protected and regulated.

    All it would take is someone to steal a couple of these (most likely as frozen embyos) and they would be able to replicate the manufacturing process.

    Much different than today when a $100 million facility would have to be constructed. Hiding a few hundred goats somewhere in China would be very easy.