CSL Behring files for approval of hemophilia therapies in Europe and Japan

Germany-based biotherapeutics firm CSL Behring has submitted its Marketing Authorization Application to the European Medicines Agency for its novel investigational recombinant factor VIII single-chain (rVIII-SingleChain) for the treatment of hemophilia A. In the pivotal clinical trial, rVIII-SingleChain met all primary endpoints.

“Our focused research and development and strong relationship with the bleeding disorders community led us to seek further advancements in the care and treatment of hemophilia,” said Andrew Cuthbertson, chief scientific officer and director of R&D, at Australian parent company CSL Limited (ASX: CSL). “Today, we have the only recombinant single-chain factor VIII product in late-stage development for the management of hemophilia A. We look forward to, upon approval, providing this innovative specialty biotherapy to patients in the EU and European Economic Area,” he added.

The MAA is based on the AFFINITY clinical development program, which includes a Phase I/III open-label, multicenter trial examining safety and efficacy. The pharmacokinetics of rVIII-SingleChain compared with recombinant human antihemophilic factor VIII (octocog alfa) was also studied.