GTC's ATryn granted orphan status by FDA

USA-based GTC Biotherapeutics, a specialist in the development and commercialization of therapeutic proteins, says that its candidate product ATryn has been designated as an Orphan Drug for hereditary antithrombin deficiency by the Food and Drug Administration. The compound is a recombinant form of human antithrombin, a plasma protein that has anticoagulant and anti-inflammatory properties.

The drug, which is currently being examined in a Phase III comparative study in HD patients at risk of developing deep vein thrombosis, is produced in goats milk using GTC's unique transgenic technology. The firm said that this method allows for the creation of antithrombin supplies irrespective of plasma availibility, which would otherwise limit production.

The drug, which was co-developed by GTC and Danish partner Leo Pharma AS, has received European Medicines Agency (EMEA) approval for the treatment of HD patients undergoing surgery, and has been granted similar authorizations in Canada and the Middle East. GTC's share price jumped 19.6% to $1.10 in pre-market trading on the day of the announcement, December 10.