Solulin - Haemophilia

Haemophilia manifests itself as the inability to form stable blood clots when blood vessels are damaged leading to prolonged and recurrent bleeding. This is not only a problem in the case of obvious external damage to the body or during surgery, but also in daily life due to normal wear and tear. Such stresses are particularly common in the joints and so can lead to joint deformities and arthropathy. When inadequately treated, by bleeding into vital organs haemophilia can be fatal in childhood or early adult life.

Haemophilia is caused by a lack of certain coagulation factors, mostly Factor VIII and, less frequently Factor IX. Such deficiency results in less clotting but also increased fibrinolysis.

There are estimated to be 400,000 haemophilia sufferers world-wide, with approximately 22,000 in Europe and 20,000 in the USA. Approximately 80% of sufferers lack Factor VIII (haemophilia A) and 20% lack Factor IX (haemophilia B). As both conditions are recessive genetic conditions linked to the X chromosome it almost only affects males.

Solulin is an improved variant of the human protein thrombomodulin, an important natural regulator of the clotting system. One of the functions of thrombomodulin is to stabilize the initial fibrin clot to stop bleeding. Other than native thrombomodulin which is anchored in the wall of blood vessels, Solulin can enter the blood stream to reach its potential site of action. In low concentrations Solulin is able to stabilise blood clots and to support coagulation.

Solulin already has been successfully tested in a Phase I study which confirmed the excellent safety profile of the substance. A Phase Ib trial in patients with severe haemophilia A was initiated in August 2011. Besides haemophilia, the development of Solulin for the treatment of radiation injury could be a suitable option.