GTC’s ATryn receives FDA orphan designation

ATryn is GTC’s recombinant form of human antithrombin, a protein with anticoagulant and anti-inflammatory properties that is normally present in human plasma.

Orphan drug designation provides GTC with seven years of marketing exclusivity upon product licensure and automatic waiver of the FDA’s application user fee.

Geoffrey Cox, GTC’s chairman and CEO, said: “ATryn is the only recombinant human antithrombin product being developed to address the needs of this rare patient population who are at risk of developing serious and potentially life-threatening venous thromboses. The orphan drug designation is recognition of the importance of our developing a unique therapeutic for this rare patient population.

“We are close to completion of the Phase III comparative study to treat hereditary antithrombin deficiency patients at risk for developing deep vein thrombosis or thromboembolism while undergoing surgery or childbirth. We plan to have top-line results of the comparative study available in late December to early January, depending on final patient scheduling. The FDA recently granted fast track designation for ATryn and provided permission for a rolling Biologics License Application, or BLA, submission. We will complete the submission after all clinical data is gathered, analyzed and available for the BLA, which is planned to be in the first half of 2008.”