GTC Therapeutics says it's been told by the EMEA that its application for marketing approval for Atryn has been turned down. Atryn is a recombinant form of human antithrombin, a protein in human plasma with both anticoagulant and anti-inflammatory uses. GTC, which is based in Framingham, MA, says the committee determined that researchers did not have enough patients enrolled in the trial to support their application. GTC went on to say that its drug partner, LEO Pharma, would continue to work toward gaining approval for an acquired deficiency indication for Europe. And GTC says it is proceeding with plans to seek U.S. marketing approval in the first half of next year.
The therapy has received considerable scrutiny, as it would have been the first drug developed from a biotech animal. The therapy is a human protein extracted from the milk of genetically modified goats. Researchers modified goat DNA with a human gene that promotes anticoagulants.