CSL Behring submits MAA to EMA for recombinant factor VIII single-chain (rVIII-SingleChain) to treat haemophilia A

CSL Behring has announced that the company has submitted its Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for its novel investigational recombinant factor VIII single-chain (rVIII-SingleChain) for the treatment of haemophilia A. In the pivotal clinical trial, rVIII-SingleChain met all primary endpoints. The MAA is based on the AFFINITY clinical development programme, which includes a phase II/III open-label, multi-centre trial examining safety and efficacy. The pharmacokinetics of rVIII-SingleChain compared with recombinant human factor VIII (octocog alfa) was also studied. Study design details for rVIII-SingleChain are available at clinicaltrials.gov (NCT01486927).

Results from the phase II/III study presented earlier in 2015 at the International Society on Thrombosis and Haemostasis (ISTH) congress in Toronto showed that patients treating prophylactically had a median annualized bleeding rate (ABR) of 1.14 and a median annualized spontaneous bleeding rate (AsBR) of 0.00. The data also showed that, of 848 bleeds treated in the study, 94% were controlled with no more than two infusions of rVIII-SingleChain, with 81% controlled by one infusion. Moreover, haemostatic control of a bleeding event treated with rVIII-SingleChain was assessed by the investigator as excellent or good 94% of the time (835 assessed bleeding events).