Liposomal approach towards the development of a longer-acting factor VIII

Abstract

Prevention of spontaneous bleeding in patients with severe haemophilia A usually requires therapeutic infusions every 2-3 days because of the short half-life of factor VIII (FVIII). Longer-acting FVIII products that require less frequent infusions would be beneficial and might obviate the need for central catheters in most patients. Liposomal formulation can enhance the efficacy of some therapeutic products. The incorporation of high-molecular weight polyethylene glycol (PEG) can extend the circulatory half-life of the liposome. These combined approaches led to the development of BAY 79-4,980, a PEG-containing liposomal version of Kogenate FS (rFVIII-FS). Results from preclinical models and early clinical trials have shown that BAY 79-4,980 prolongs the time to the next bleed. Further clinical evaluation of the efficacy and long-term safety of BAY 79-4,980 are planned.