February 7, 2009

US Approves Drug From Genetically Altered Goats

On Friday, U.S. health officials approved the first drug made using genetically engineered animals in the midst of concerns about genetic implications.

ATryn, made by GTC Biotherapeutics, is a drug that is manufactured using milk from goats that have been scientifically altered to produce extra antithrombin, a protein that acts as a natural blood thinner.

The drug aims to prevent excessive blood clots in patients with a disorder known as hereditary antithrombin deficiency.  The company estimates that this disorder affects anywhere from 60,000 to 600,000 people in the United States.

The drug was approved by the Food and Drug Administration to help prevent blood cots from surgery or childbirth in patients with the condition, but no wider use to treat the disorder itself, the company said in a statement.

The company had been looking for approval for both uses, and earlier predicted the drug could generate $40 million to $50 million in annual sales in the U.S. in the first five years on the market.

"The approval of Atryn marks a significant milestone in the development of this innovative recombinant technology," GTC Chairman and CEO Geoffrey Cox said in a statement.

According to GTC, about 1 in 5,000 people do not produce enough antithrombin protein.  As a result, their blood is more likely to stick together, occasionally causing clots that can travel to the lungs or brain, causing death.  Half of patients with the disorder experience their first life threatening clot before age 25.

Women that are pregnant and have the disorder are at higher risk of miscarriage or stillbirth, because of blood clots in the placenta.

Currently, patients suffering from hereditary antithrombin deficiency are prescribed conventional blood thinners, such as Plavix from Bristol-Myers and Sano-Aventis. 

Some genetic safety and animal advocates have raised doubts about using so-called transgenic animals to make pharmaceuticals, saying the FDA needs to provide more information about genetically engineered animals.

Last month, the FDA issued final guidelines on its plans to regulate animals who's DNA had been altered.  It has yet to show a genetically engineered animals for human consumption.  Agency officials said they aimed to make the regulatory process more transparent, but some critics said they did not go far enough.

A policy analyst for the nonprofit group Center for Food Safety, Jaydee Hanson, said treating GTC's goats as a drug rather than as an animals left many questions unanswered, such as what would happen to the goats that die.  Dead animals are sometimes processed for pet food.

"This is a backdoor way to approve genetically engineered animals," Hanson told Reuters.

The goats that GTC uses are bred using cell injected with human DNA.  The company has a herd of about 200 at its Massachusetts facility.  GTC also says that the heard is otherwise normal and healthy.

The company said that the drug is licensed to Ovation Pharmaceuticals Inc. in the U.S. and should be available by the second quarter this year.


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