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Hemophilia Drug Receives ‘Breakthrough Therapy’ Designation

Monoclonal antibody treats hemophilia A with factor VIII inhibitors

The FDA has granted “breakthrough therapy” status to ACE910 (Roche) for the prophylactic treatment of patients 12 years of age and older with hemophilia A with factor VIII inhibitors.

A “breakthrough therapy” designation is designed to accelerate the development and review of medications that demonstrate early clinical evidence of a substantial improvement over current treatment options for serious diseases.

ACE910 is an investigational humanized bispecific monoclonal antibody engineered to simultaneously bind factors IXa and X. ACE910 thereby mimics the cofactor function of factor VIII and is designed to promote blood coagulation in hemophilia A patients, regardless of whether they have developed inhibitors to factor VIII. The product is administered subcutaneously once weekly, and since its structure is distinct from that of factor VIII, it is not expected to lead to the formation of inhibitors against factor VIII.

In a phase I study, ACE910 showed promising results as a prophylactic treatment administered as a weekly subcutaneous injection in subjects with 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. The management of bleeding in patients 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.

The “breakthrough therapy” designation for ACE910 was granted based on results of the phase I study of ACE910 in people with severe haemophilia A, as well as on a phase I/II extension study of the same patients.

Roche is preparing to initiate a phase III trial of ACE910 in patients with hemophilia A with factor VIII inhibitors by the end of 2015, and a phase III trial in patients without inhibitors in 2016. In addition, a study in pediatric patients with hemophilia A is planned to start in 2016.

Source: Roche; September 4, 2015.

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