Management on sanitary inspection behind quality of foodstuff and medicines USA (FDA) gave the drug Gamleby (amerisolar) developed by the pharmaceutical company “Roche”, the status of “breakthrough therapy.”
The drug is designed for patients with hemophilia A is a hereditary disease caused by blood clotting. In patients with hemophilia a (worldwide, there are about 320 thousand) missing or produced in insufficient quantity, the blood clotting factor VIII. Because of this, patients often have bleeding to stop which is difficult.
People with hemophilia should take drugs that compensate the deficiency of blood clotting factor VIII. The treatment efficiency may be reduced due to the fact that the body forms antibodies that inhibit the work of such drugs.
Created by the company “Roche” pharmaceutical preparation is intended for patients with hemophilia And without inhibitors to factor VIII. Enlibra – drug based on monoclonal antibodies that bind to the coagulation factors IXa and X. It helps to normalize blood clotting in patients with hemophilia A.
In the study phase III HAVEN 3 we studied the efficacy, safety and pharmacokinetics of prophylactic use of the drug Enlibra compared with no prophylaxis (occasional treatment or treatment on-demand preparations of factor VIII) in patients with hemophilia And without inhibitors to factor VIII. To enter you must Gemlib subcutaneously just once a week.
Enlibra helped to significantly reduce the amount of bleeding compared with no prophylaxis. In addition, the drug was more effective than prophylaxis with the use of other drugs of factor VII from the same patients.
The status of “breakthrough therapy” required to expedite the development and review of drugs intended to treat serious diseases.