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Liposomal approach towards the development of a longer‐acting factor VIII
Author(s) -
POWELL J. S.
Publication year - 2007
Publication title -
haemophilia
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.213
H-Index - 92
eISSN - 1365-2516
pISSN - 1351-8216
DOI - 10.1111/j.1365-2516.2007.01502.x
Subject(s) - medicine , bleed , liposome , haemophilia , clinical trial , haemophilia a , peg ratio , polyethylene glycol , pharmacology , intensive care medicine , surgery , materials science , nanotechnology , finance , chemical engineering , engineering , economics
Summary. 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 79‐4980, a PEG‐containing liposomal version of Kogenate ® FS (rFVIII‐FS). Results from preclinical models and early clinical trials have shown that BAY 79‐4980 prolongs the time to the next bleed. Further clinical evaluation of the efficacy and long‐term safety of BAY 79‐4980 are planned.