Alprolix (recombinant Factor IX Fc fusion protein): extended half-life product for the prophylaxis and treatment of hemophilia B.

Abstract

Hemophilia B is a genetic disease caused by mutation of the gene for coagulation protein Factor IX. When severe, the disease leads to spontaneous life-threatening bleeding episodes. Current therapy requires frequent intravenous infusions of therapeutic recombinant or plasma-derived protein concentrates containing Factor IX. Alprolix™ (recombinant Factor IX Fc fusion protein), is a therapeutic Factor IX preparation that has been engineered for a prolonged half-life in circulation, has completed pivotal clinical trials and has been approved recently in the USA, Canada, Australia and Japan for use in the clinic for patients with hemophilia B. This promising therapy should allow patients to use fewer infusions to maintain appropriate Factor IX activity levels in all clinical settings, and its use may be indicated in both on demand and prophylactic treatments.

• Authors: Jonathan M Ducore, Maricel G Miguelino, Jerry S Powell
• Journal: Expert review of hematology (Expert Rev Hematol) Vol. 7 Issue 5 Pg. 559-71 (Oct 2014) ISSN: 1747-4094 [Electronic] England
• PMID: 25142322 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't)

Chemical References

• Receptors, Fc
• Recombinant Fusion Proteins
• Polyethylene Glycols
• Factor IX

Topics

• Animals
• Clinical Trials as Topic
• Dogs
• Factor IX (genetics, metabolism)
• Half-Life
• Hemophilia B (drug therapy)
• Humans
• Macaca fascicularis
• Mice
• Mice, Knockout
• Monte Carlo Method
• Polyethylene Glycols (chemistry)
• Receptors, Fc (genetics, metabolism)
• Recombinant Fusion Proteins (biosynthesis, pharmacokinetics, therapeutic use)