FDA grants Breakthrough Therapy designation for Roche hemophilia drug

The US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for ACE910 (ROG: SIX) in patients with hemophilia A with factor VIII inhibitors.

The Phase I study of ACE910 showed promising results as a prophylactic treatment administered as a weekly subcutaneous injection in severe hemophilia A with and without inhibitors to factor VIII. The development of inhibitors is a serious complication of hemophilia A treatment regardless of disease severity, making it difficult, if not impossible, to achieve a level of factor VIII sufficient to control bleeding with traditional replacement therapies. Management of bleeding in people with hemophilia A who have inhibitors to factor VIII is a major challenge, and there remains a need for additional treatment options for these patients.

Sandra Horning, Roche’s chief medical officer and head of global product development, said: “People with hemophilia A may require regular and frequent infusions of replacement clotting factor to reduce the risk of dangerous bleeding, and they can develop inhibitors that make replacement ineffective. We are pleased that the FDA has granted Breakthrough Therapy designation for ACE910, recognising an unmet need for patients with inhibitors and the promise of these early data. Roche has been developing antibody treatments for people with blood disorders for over 20 years, and we are excited to expedite the development of a potential new treatment for hemophilia A.”