Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A

Author:

Mahlangu Johnny1,Powell Jerry S.2,Ragni Margaret V.3,Chowdary Pratima4,Josephson Neil C.5,Pabinger Ingrid6,Hanabusa Hideji7,Gupta Naresh8,Kulkarni Roshni9,Fogarty Patrick10,Perry David11,Shapiro Amy12,Pasi K. John13,Apte Shashikant14,Nestorov Ivan15,Jiang Haiyan15,Li Shuanglian15,Neelakantan Srividya15,Cristiano Lynda M.15,Goyal Jaya15,Sommer Jurg M.15,Dumont Jennifer A.15,Dodd Nigel15,Nugent Karen15,Vigliani Gloria15,Luk Alvin15,Brennan Aoife15,Pierce Glenn F.15

Affiliation:

1. Haemophilia Comprehensive Care Centre, Faculty of Health Sciences, University of the Witwatersrand and National Health Laboratory Service, Johannesburg, South Africa;

2. University of California at Davis, Davis, California;

3. University of Pittsburgh and the Hemophilia Center of Western Pennsylvania, Pittsburgh, PA;

4. Katharine Dormandy Haemophilia Centre and Thrombosis Unit, Royal Free Hospital, London, United Kingdom;

5. Puget Sound Blood Center, Seattle, WA;

6. Universitätsklinik für Innere Medizin I, Medizinische Universität Wien, Wien, Austria;

7. Department of Hematology, Ogikubo Hospital, Suginami-ku, Tokyo, Japan;

8. Haemophilia Centre and Department of Medicine, Maulana Azad Medical College & Associated Lok Nayak Jai Prakash Narayan Hospital, New Delhi, India;

9. Department of Pediatrics and Human Development, Michigan State University, East Lansing, MI;

10. Penn Comprehensive Hemostasis and Thrombosis Program, Hospital of the University of Pennsylvania, Philadelphia, PA;

11. Cambridge Haemophilia & Thrombosis Centre, Addenbrooke’s Hospital, Cambridge, United Kingdom;

12. Indiana Hemophilia and Thrombosis Center, Department of Hematology, Indianapolis, IN;

13. Royal London Haemophilia Centre, Barts and the London School of Medicine and Dentistry, London, United Kingdom;

14. Sahyadri Hospital, Department of Hematology, Mahara, India; and

15. Biogen Idec Hemophilia, Cambridge, MA

Abstract

Key Points A novel recombinant factor VIII with prolonged half-life, rFVIIIFc, was developed to reduce prophylactic injection frequency. rFVIIIFc was well-tolerated in patients with severe hemophilia A, and resulted in low bleeding rates when dosed 1 to 2 times per week.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

Reference29 articles.

1. Fc-fusion proteins and FcRn: structural insights for longer-lasting and more effective therapeutics [published online ahead of print October 24, 2013].;Rath

2. National Hemophilia Foundation MASAC recommendation concerning prophylaxis (regular administration of clotting factor concentrate to prevent bleeding). Available at:http://www.hemophilia.org/NHFWeb/MainPgs/MainNHF.aspx?menuid=57&contentid=1007. Accessed July 18, 2013

3. Improvements in factor concentrates.;Lillicrap;Curr Opin Hematol,2010

4. Barriers to compliance with prophylaxis therapy in haemophilia.;Hacker;Haemophilia,2001

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