Safety and Efficacy of Moroctocog Alfa (AF-CC) in Chinese Patients with Hemophilia A: Results of Two Open-Label Studies

Renchi Yang,1 Yongqiang Zhao,2 Xuefeng Wang,3 Jing Sun,4 Runhui Wu,5 Chenghao Jin,6 Jie Jin,7 Depei Wu,8 Pablo Rendo,9 Feifei Sun,10 Jeremy Rupon,9 Francois Huard11,†, Joan M Korth-Bradley,12 Lihong Xu,10 Binyu Luo,10 Yingxue Cathy Liu13 1Thrombosis and Hemostasis Center, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, People’s Republic of China; 2Department of Hematology, Peking Union Medical College Hospital, Beijing, People’s Republic of China; 3Department of Clinical Blood Transfusion, Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, People’s Republic of China; 4Department of Hematology, Nanfang Hospital, Southern Medical University, Guangzhou, People’s Republic of China; 5Department of Hematology, Beijing Children’s Hospital and Capital Medical University, Beijing, People’s Republic of China; 6Department of Hematology, Jiangxi Provincial People’s Hospital, Jiangxi, People’s Republic of China; 7Department of Hematology, The First Affiliated Hospital – Zhejiang University School of Medicine, Hangzhou, People’s Republic of China; 8Department of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, People’s Republic of China; 9Rare Disease, Pfizer Inc, Collegeville, PA, USA; 10 Pfizer (China) Research & Development Co, Ltd, Shanghai, People’s Republic of China; 11Clinical Pharmacology, Pfizer PIO, Paris, France; 12Pfizer Inc, Collegeville, PA, USA; 13Statistics, Pfizer (China) Research & Development Co, Ltd, Shanghai, People’s Republic of China†Dr. Francois Huard passed away on July 20, 2017.Correspondence: Renchi Yang Email rcyang65@163.comIntroduction: Moroctocog alfa albumin-free cell culture (AF-CC) increases plasma levels of factor VIII (FVIII) activity and, in China, is indicated for the control and prevention of bleeding episodes in patients with hemophilia A. This study aimed to evaluate the efficacy, safety, and recovery data of moroctocog alfa (AF-CC) in patients with hemophilia participating in two open-label studies, both conducted in China.Methods: The authorization study (clinicaltrials.gov identifier NCT00868530) enrolled patients aged ≥ 6 years, previously treated with ≥ 1 exposure day of FVIII replacement therapy. The real-world study (clinicaltrials.gov identifier NCT02492984) enrolled patients of any age who were previously untreated or requiring surgical prophylaxis. In both studies, on-demand treatment was administered over 6 months. Key assessments included response to treatment, FVIII inhibitor development, and recovery.Results: In the authorization study (N = 53; mean age, 23.2 years; severe hemophilia, 23%), response was excellent/good for 90% of infusions at 24 hours. Seven patients developed inhibitors. Mean (SD) FVIII recovery at the initial and final visits was 1.77 (0.50) and 1.67 (0.45) (IU/dL)/(IU/kg), respectively. In the real-world study (N = 85; mean age, 9.5 years; severe hemophilia, 58%), response was rated as excellent or good for most (87%) on-demand infusions and for all surgical prophylaxis patients (n = 14). Seven patients developed FVIII inhibitors. Mean (SD) FVIII recovery at the initial and final visits was 1.71 (0.50) and 1.68 (0.31) (IU/dL)/(IU/kg), respectively. No new safety signals were observed in either study.Conclusion: On-demand treatment and surgical prophylaxis with moroctocog alfa (AF-CC) is safe and effective for both previously treated and previously untreated Chinese patients with hemophilia A.Keywords: Asian, blood coagulation factor VIII, deficiency, factor VIII, ReFacto, Xyntha