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

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
• blood coagulation factor VIII
• deficiency
• factor VIII
• ReFacto
• Xyntha

Acknowledgments

The authors thank all of the clinical personnel and patients who participated in these studies.

Abbreviations

ABR, annualized bleeding rate; AE, adverse event; AF-CC, albumin-free cell culture; BU, Bethesda unit; CI, confidence interval; ED, exposure day; FVIII, factor VIII; IV, intravenous; LETE, less-than-expected therapeutic effect; SAE, serious adverse event; SD, standard deviation; WFH, World Federation of Hemophilia.

Data Sharing Statement

Upon request, and subject to certain criteria, conditions, and exceptions (see https://www.pfizer.com/science/clinical-trials/trial-data-and-results for more information), Pfizer will provide access to individual de-identified participant data from Pfizer-sponsored global interventional clinical studies conducted for medicines, vaccines, and medical devices (1) for indications that have been approved in the US and/or EU or (2) in programs that have been terminated (ie, development for all indications has been discontinued). Pfizer will also consider requests for the protocol, data dictionary, and statistical analysis plan. Data may be requested from Pfizer trials 24 months after study completion. The de-identified participant data will be made available to researchers whose proposals meet the research criteria and other conditions, and for which an exception does not apply, via a secure portal. To gain access, data requestors must enter into a data access agreement with Pfizer.

Author Contributions

All authors made substantial contributions to the conception and design, acquisition of data, or analysis and interpretation of data; participated in drafting the article or revising it critically for important intellectual content, with the support of a medical writer provided by Pfizer Inc; gave final approval of the version to be published; and agree to be accountable for all aspects of the work.

Disclosure

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.

Additional information

Funding

This study was sponsored by Pfizer Inc. Medical writing and editorial support were provided by Bina J. Patel, PharmD, and Michelle McDermott, PharmD, of Peloton Advantage, LLC, an OPEN Health company, and were funded by Pfizer Inc.