SOUTH SAN FRANCISCO (dpa-AFX) - Genentech, a member of the Roche Group (RHHBY), Friday said the U.S. Food and Drug Administration has granted breakthrough therapy designation to ACE910 (RG6013, RO5534262) for the prophylactic treatment of people who are 12 years or older with hemophilia A with factor VIII inhibitors.
Hemophilia A, a rare genetic disorder, occurs when an essential blood clotting protein called factor VIII is not present in sufficient amounts or is defective. People with severe hemophilia A can be susceptible to uncontrolled or difficult to control bleeding including internal bleeding.
Breakthrough therapy designation is designed to accelerate the development and review of medicines that demonstrate early clinical evidence of a substantial improvement over current treatment options for serious diseases.
In a Phase I study, ACE910 showed promising results as a prophylactic treatment administered as a weekly subcutaneous injection in people with severe hemophilia A with and without inhibitors to factor VIII.
Copyright RTT News/dpa-AFX