4 September 2015 - Roche today announced that the US Food and Drug Administration (FDA) has granted breakthrough therapy designation to ACE910 (RG6013, RO5534262) for the prophylactic treatment of people who are 12 years or older with haemophilia A with factor VIII inhibitors. Haemophilia A, a rare genetic disorder, occurs when an essential blood clotting protein called factor VIII is either not present in sufficient amounts or is defective. People with severe haemophilia A can be susceptible to uncontrolled or difficult to control bleeding including internal bleeding, especially into the joints, which can lead to the need for joint replacements.
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 haemophilia A with and without inhibitors to factor VIII. The development of inhibitors is a serious complication of haemophilia 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 haemophilia A who have inhibitors to factor VIII is a major challenge, and there remains a need for additional treatment options for these patients.

For more details, go to: http://www.roche.com/media/store/releases/med-cor-2015-09-04.htm