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ATryn® - a safe drug without use of donor blood


It concerns patients who, due to a genetic defect, cannot produce enough of the protein antithrombin III or whose antithrombin III is not sufficiently efficient.

Antithrombin is a protein that is a key part of managing the coagulation process in the bloodstream.

This hereditary fault (Hereditary antithrombin Deficiency, HD) means that the patients e.g. when in surgery can be in danger of dying from thrombosis if not treated for the lacking antithrombin III.

With ATryn®, the HD patients now get access to a new, safe and technological remarkable treatment without the use of donor blood. ATryn® is the world’s first approved drug which is produced in transgenic goats. Human antithrombin is expressed in the milk of these goats and then processed into a highly purified product.

GTC Biotherapeutics

In 2005, LEO Pharma and the American GTC Biotherapeutics Inc. concluded a development and marketing agreement for Europe, Canada and the Middle East concerning ATryn®.

LEO is responsible for the marketing and sales in the mentioned areas for the HD indication as well as further developing ATryn® for new indications. GTC is responsible for supplying the product.

Clinical phase II study

In August, LEO Pharma began a clinical phase II study in which patients with DIC (Disseminated Intravascular Coagulation) are treated with ATryn®. DIC is serious condition during which the blood coagulates in the blood vessels and occurs in connection with serious sepsis. In the EU and Canada, the number of patients stricken by sepsis and DIC each year is estimated to be about 125,000 – 30-60% of these ends fatally.

“ATryn® is a new, exciting drug with interesting perspectives if it can be used for the treatment of other indications. This could e.g. be for the treatment of DIC at patients with severe sepsis. ATryn® fits well with our existing anticoagulation business which among other things includes Innohep®, Heparin LEO® and Protaminsulfat LEO Pharma®,” says Ernst Lunding, CEO of LEO Pharma.

The ATryn® phase II study concerning DIC will prove the drug’s safety, effect and optimal dose against future phase III study.


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