FDA Okays Anticoagulant Made from Goat's Milk

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FDA Okays Anticoagulant Made from Goat's Milk

By Kristina Fiore, Staff Writer, MedPage Today
Published: February 06, 2009
Reviewed by Zalman S. Agus, MD; Emeritus Professor
University of Pennsylvania School of Medicine. ROCKVILLE, Md., Feb. 6 -- The FDA has approved the first pharmaceutical product made from genetically engineered animals.

Antithrombin alfa (ATryn), derived from goat milk, was okayed to prevent thromboembolitic events in patients with hereditary antithrombin deficiency.

Manufacturer GTC Biotherapeutics Inc., of Framingham, Mass., received confirmation of its approval from both the FDA's Center for Biologics Evaluation and Research and its Center for Veterinary Medicine.

The Center for Biologics Evaluation and Research deemed the drug safe and effective after evaluating two studies comprising 31 patients with hereditary antithrombin deficiency who received the anticoagulant to prevent thromboemboli before, during, or after surgery or childbirth.

Only one patient developed an embolism, according to the FDA.

The most common adverse events, occurring in about 5% of patients, were hemorrhage and reactions at the infusion site.

The Center for Veterinary Medicine evaluated the safety of gene modification in the goats, and concluded that introducing the recombinant DNA (rDNA) caused no adverse health outcomes over seven generations.

The rDNA process introduces new traits into an organism by splicing together pieces of DNA to create an "rDNA construct" that gives the organism its new characteristics.

In this case, the rDNA construct causes the goats to produce human antithrombin in their milk.

Just last month, the FDA issued a final guidance for industry on the regulation of genetically engineered animals, which outlines the regulation process and provides an overview of responsibilities for manufacturers.

Because hereditary antithrombin deficiency is a rare disorder, occurring in about one in 5,000 patients in the U.S., it was given an orphan drug designation by the FDA.

The company noted that the drug is contraindicated in patients with known hypersensitivity to goat and goat milk proteins.
It is expected to be available in the second quarter of 2009.