Roche Hemophilia Drug Gets FDA Breakthrough Therapy StatusBy
The US Food and Drug Administration has granted breakthrough therapy designation to Roche’s 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 either not present in sufficient amounts or is defective.
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.
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. 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. ACE910 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. ACE910 was created by Chugai Pharmaceutical Co., Ltd. and is being co-developed by Roche.