Roche's haemophilia A treatment ACE910 gains FDA breakthrough therapy status

The FDA granted breakthrough therapy designation to Roche's ACE910 for the prophylactic treatment of people who are 12 years or older with haemophilia A with factor VIII inhibitors, the company reported Friday. ACE910, which was created by Chugai Pharmaceutical and is being co-developed by Roche, is an experimental humanised bispecific monoclonal antibody engineered to simultaneously bind factors IXa and X.

Roche noted that the designation was based primarily on findings from a Phase I study of ACE910 in patients with severe haemophilia A, as well as a Phase I/II extension study of the same patients presented at the International Society of Thrombosis and Haemostasis annual meeting this year. The company indicated that in early research, ACE910 exhibited "promising efficacy" as a prophylactic treatment in patients with severe haemophilia A with and without inhibitors to factor VIII.

Roche said that it is planning to initiate a late-stage study of ACE910 in patients with haemophilia A with factor VIII inhibitors by the end of this year and a second Phase III trial in patients without inhibitors in 2016. The drugmaker also plans to start a clinical trial in paediatric patients with haemophilia A in 2016.

For related analysis, read ViewPoints: Can Roche revolutionise the hemophilia A market.

To read more Top Story articles, click here.