WASHINGTON U.S. health officials on Friday approved the first drug made using genetically engineered animals despite lingering concerns over health and environmental implications.
The drug, GTC Biotherapeutics Inc's anti-clotting therapy Atryn, is an intravenous therapy made using a human protein gathered from female goats specially bred to produce it in their milk.
It aims to prevent excessive blood clots in patients with a disorder known as hereditary antithrombin deficiency. The company estimates the condition affects anywhere from 60,000 to 600,000 people in the United States.
"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.
But 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.
The FDA last month, under the Bush administration, issued final guidelines on regulating animals with altered DNA. It has yet to approve such an animal for human consumption.
Agency officials have said they aimed to make the regulatory process more open, but critics argue they did not go far enough and are calling on President Barack Obama's administration to review the policy.
"This is a backdoor way to approve genetically engineered animals," said Jaydee Hanson, a policy analyst for the nonprofit group Center for Food Safety.
In a statement, the FDA said it had "determined that the manufacturer, GTC, has adequate procedures in place to ensure that food from these goats does not enter the food supply." It also said GTC must properly dispose of any dead goats by burying or burning.
Gregory Jaffe, director of the Center for Science in the Public Interest's Biotechnology Project, said even with those precautions little is known about the impact of such genetic changes long-term.
"Humans are fallible and animals are living creatures. Even with the best intentions, we still have that chance that they will escape or be eaten. So I think it's imperative that FDA determine whether there are any adverse impacts," he told Reuters.
GTC spokesman Tom Newberry said the Framingham, Massachusetts-based company has numerous precautions in place to ensure its goats stay in place, including two fenced-in areas and special transponders to help track each animal.
"The Massachusetts area we're in does not have native goats so its pretty obvious if there's a goat around it's most likely ours," he said.
GTC's goats are bred using cells injected with human DNA in a process that it says is a cost effective way to produce human antithrombin, a natural protein to prevent blood from clotting. The company has a herd of about 200 at its Massachusetts facility that it says is otherwise normal and healthy.
The FDA looked at the impact of goats as they aged and reproduced. "We have looked carefully at seven generations of these (generically engineered) goats; all of them are healthy and we haven't seen any adverse effects," said Bernadette Dunham, head of the FDA's Center for Veterinary Medicine.
The FDA approved Atryn to prevent blood clots from surgery or childbirth in patients with the condition. GTC earlier predicted the drug could generate $40 million to $50 million in annual sales in the United States in its first five years on the market.
The drug is licensed to Ovation Pharmaceuticals Inc in the United States and should be available by the second quarter this year, the company said. It is already approved in Europe to prevent clots during surgery in patients with the inherited disorder.
GTC's Newberry said the companies planned to seek additional approval for non-hereditary antithrombin deficiency, which can occur after heart surgery and other instances.
Shares of the company closed off more than 14.6 percent at 70 cents on Nasdaq.
(Editing by Tim Dobbyn, John Wallace and Bernard Orr)