DUTCH GROUP TO SELL GM ANIMAL DRUG

The first European company to produce a drug from a genetically modified animal is to begin distributing its treatment to patients in the coming weeks.

Pharming, based in the Netherlands, is set to be the world’s first pharmaceutical group to derive a medicine from transgenic rabbits, using the milk they produce as the basis for its drug to treat a rare genetic disease.

It will be only the second medicine cleared by regulators from a transgenic animal, after GTC Biotherapeutics of the US received authorisation to produce its ATryn drug, to prevent blood clots, from the milk of genetically modified goats.

After decades of research, the move could herald a new era of drugs using small, rapidly reproducing transgenic animals as “living factories”, though risking criticism from animal rights activists.

Pharming has 200 rabbits kept indoors in secured hygienic conditions. The animals have been bred with DNA modified so their mammalian glands release milk containing a protein that is then purified as the basis for the drug Ruconest.

The medicine treats hereditary angioedema, a rare genetic disease that causes uncomfortable and sometimes life-threatening swelling, pain and obstruction of the airways.

The product, approved by the European Medicines Agency in June, requires a final formal ratification from the European Commission, which is expected by the start of November. Under the brand name Rhucin, it will be submitted for US regulatory approval by the end of this year.

Production in rabbits allows high volume manufacture of the protein that is the basis of the drug. The milk is extracted using a mechanised pump, a modified version of those used by dairy farmers for cows.

Volumes can be expanded quickly through additional breeding, compared with costly and cumbersome alternative manufacturing processes using fermenters.

Pharming has refused to disclose the exact location of its factory, mindful of potential threats from animal rights activists.

Helen Wallace, head of GeneWatch UK, a research body critical of the “overuse of genetic solutions”, said: “We are concerned about the impact on animal welfare. Normally there are other ways to produce drugs. No production in this way has proved cost effective ... There are many examples of experimental attempts which have ultimately ended in failure.”

But Sijmen de Vries, chief executive of Pharming, stressed that the rabbits were well cared for and showed no evidence of side effects from genetic modification, which he said only affected the mammalian glands producing milk. “We are constantly monitoring each animal,” he said. “They are checked daily.”

He added that the production method was low cost, that there was no evidence of any disease that had passed into humans from transgenic products, and that milk was itself an immune-privileged substance that would protect its contents from infection.

The idea for the use of transgenic animals came from researchers in the 1980s at Genentech, the US biotech group now owned by Roche of Switzerland, seeking to avoid the use of biological fermenters.

SOURCE: The Financial Times, UK

AUTHOR: Andrew Jack

URL: http://www.ft.com/cms/s/06653f94-d7b2-11df-b478-00144feabdc0,Authorised=...

DATE: 14.10.2010