Recombinant long-acting glycoPEGylated factor IX in hemophilia B: a multinational randomized phase 3 trial

Author:

Collins Peter W.1,Young Guy2,Knobe Karin3,Karim Faraizah Abdul4,Angchaisuksiri Pantep5,Banner Claus6,Gürsel Türkiz7,Mahlangu Johnny8,Matsushita Tadashi9,Mauser-Bunschoten Eveline P.10,Oldenburg Johannes11,Walsh Christopher E.12,Negrier Claude13

Affiliation:

1. Arthur Bloom Haemophilia Centre, Institute of Infection and Immunity, School of Medicine, Cardiff University, Cardiff, United Kingdom;

2. Hemostasis and Thrombosis Center, Children's Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA;

3. Haemophilia R&D Portfolio, Novo Nordisk A/S, Soeborg, Denmark;

4. Haemophilia Centre, National Blood Centre, Kuala Lumpur, Malaysia;

5. Division of Hematology, Department of Medicine, Faculty of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand;

6. Haemophilia Clinical Operations, Novo Nordisk A/S, Soeborg, Denmark;

7. Gazi University Medical School, Ankara, Turkey;

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

9. Department of Transfusion Medicine, Nagoya University Hospital, Nagoya, Japan;

10. Van Creveldkliniek, University Medical Centre, Utrecht, The Netherlands;

11. Institute of Experimental Haematology and Transfusion Medicine, University Clinic Bonn, Bonn, Germany;

12. Division of Hematology and Medical Oncology, Mount Sinai Medical Center, New York, NY; and

13. Hôpital Edouard Herriot, University Claude Bernard Lyon 1, Lyon, France

Abstract

Key Points Nonacog beta pegol, a recombinant glycoPEGylated FIX with extended half-life, was developed to improve care for patients with hemophilia B. Weekly prophylaxis with nonacog beta pegol was well tolerated and was associated with low bleeding rates and an improved quality of life.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

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