FDA Designates ACE910 Breakthrough Therapy for Hemophilia A

Genentech announced that the Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to ACE910 for the prophylactic treatment of hemophilia A patients ≥12 years old with factor VIII inhibitors.

The Breakthrough Therapy designation was granted based on results of a Phase 1 study in people with severe hemophilia A and a Phase 2/3 extension study of the same patients. In the Phase 1 study, ACE910 showed promising results as a prophylactic treatment administered as a weekly SC injection in people with severe hemophilia A with and without inhibitors to factor VIII.

Genentech is preparing to initiate two Phase 3 trials for ACE910; one in hemophilia A patients with factor VIII inhibitors by the end of 2015, and the second in patients without inhibitors in 2016. Additionally, a trial in pediatric patients with hemophilia A is planned to commence in 2016.

ACE910 is an investigational humanized bispecific monoclonal antibody engineered to simultaneously bind factors IXa and X. It thereby mimics the cofactor function of factor VIII and is designed to promote blood coagulation in hemophilia A patients, regardless of development of factor VIII inhibitors. As it is distinct in structure from factor VIII, it is not expected to lead to the formation of inhibitors against factor VIII.

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