Federal officials approved for the first time a drug made from milk of genetically engineered goats. The Center for Biologics Evaluation and Research (CBER) approved the human biologic based on its safety and efficacy, and the Center for Veterinary Medicine (CVM) approved the rDNA construct in the goats that produce ATryn.
The drug is designed to prevent fatal blood clots in people who suffer from a rare condition, called hereditary antithrombin deficiency. These people are at high risk during surgeries and childbirth and the drug will be used to untangle blood clots in patients with this condition. Current treatment involves taking blood thinners and infusions of human antithrombin, extracted from donated blood.
The same drug, Atryn, was approved in Europe three years ago but it has not been widely prescribed. The drug is made by GTC Biotherapeutics and comes from goats whose DNA was altered to produce a drug needed by those who suffer from this rare blood disorder.
“The mammary gland is designed by nature to make proteins for offspring in a substance that we call milk, so all we have done is provide the extra bit of coding so it makes this particular protein,” explained Thomas E. Newberry, a vice president at GTC Biotherapeutics. Human DNA for antithrombin and goat DNA is combined in a way so that the goat’s milk glands express hum antithrombin.
GTC Biotherapeutics (NASDAQ: GTCB) and partner Ovation Pharmaceuticals of Deerfield, IL hope they will be able to introduce the drug to the market before the end of June.
“This product offers an important new treatment option for patients with hereditary antithrombin deficiency, preventing life-threatening clots that otherwise frequently occur during high risk situations,” Jesse Goodman, M.D., M.P.H., CBER director, was quoted as saying.
“The approval of ATryn marks a significant milestone in the development of this innovative recombinant technology,” the company said in a statement.
This rare blood deficiency affects a small population, approximately 1 in 5,000 people in the United States.
The Center for Biologics Evaluation and Research looked at two studies that included 31 patients diagnosed with hereditary antithrombin deficiency. The participants received ATryn to prevent thromboemboli (TE) before, during or after surgery or childbirth. Almost all patients had a history of at least one TE. These problems are likely to reappear in high-risk situations if they are left untreated. The study found that only one in 31 patients treated with the drug developed a TE, or thromboemboli. The adverse reactions, which were reported in about five percent of patients, were hemorrhage and reactions in the infusion site.
Earlier this year, the FDA issued a final guidance for industry on the regulation of genetically engineered (GE) animals. The final guidance includes a set of recommendations to producers of GE animals to help them successfully complete the obligations and responsibilities under the law.
