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

Renchi Yang, Yongqiang Zhao, Xuefeng Wang, Jing Sun, Runhui Wu, Chenghao Jin, Jie Jin, Depei Wu, Pablo Rendo, Feifei Sun, Jeremy Rupon, Francois Huard, Joan M Korth-Bradley, Lihong Xu, Binyu Luo, Yingxue Cathy Liu, Renchi Yang, Yongqiang Zhao, Xuefeng Wang, Jing Sun, Runhui Wu, Chenghao Jin, Jie Jin, Depei Wu, Pablo Rendo, Feifei Sun, Jeremy Rupon, Francois Huard, Joan M Korth-Bradley, Lihong Xu, Binyu Luo, Yingxue Cathy Liu

Abstract

Introduction: 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; ReFacto; Xyntha; blood coagulation factor VIII; deficiency; factor VIII.

Conflict of interest statement

R. Yang, Y. Zhao, X. Wang, J. Sun, R. Wu, C. Jin, J. Jin, and D. Wu have no interests that might be perceived as posing a conflict or bias. J. Rupon, J. M. Korth-Bradley, and B. Luo, are employees of Pfizer Inc and may own stock/options in the company. F. Huard, P. Rendo, F. Sun, L. Xu, and Y. C. Liu were employees of Pfizer Inc at the time of this study. The authors report no other conflicts of interest in this work.

© 2020 Yang et al.

Figures

Figure 1
Figure 1
Patient disposition in the authorization study (A) and in the real-world study (B). *Two patients rolled over from the surgical prophylaxis group to the on-demand group and were counted in each group, but were included only once in the overall population.

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