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Efficacy, safety and bioequivalence of the human-derived B-domain-deleted recombinant factor VIII TQG202 for prophylaxis in severe haemophilia A patients

Department of Hematology, The First Hospital of Lanzhou University, Lanzhou, Gansu Province, China. Hemophilia Information Management Center of Jiangxi Province, Jiangxi Provincial People's Hospital, Nanchang, China. State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital,Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China. Department of Hematology, The Cancer Hospital affiliated to Zhengzhou University, Zhengzhou, China. Department of Hematology, Henan Provincial People's Hospital, People's Hospital of Zhengzhou University, Zhengzhou, China. Department of Hematology, Affiliated Drum Tower Hospital, Medical School of Nanjing University, Nanjing, Jiangsu, China. Department of Hematology, The Second Affiliated Hospital, Chongqing Medical University, Chongqing, China. Department of Hematology, Xiangya Hemophilia Diagnosis and Treatment Center, Xiangya Hospital, Central South University, Changsha, Hunan Province, China. Department of Hematology, The First Affiliated Hospital of Guangxi Medical University, Nanning, Guangxi Province, China. Department of Hematology, The Affiliated Provincial Hospital of Anhui Medical University, Hefei, China. R&D Institute, Chia Tai Tianqing Pharmaceutical Group Co. Ltd., Nanjing, Jiangsu Province, China.

Haemophilia : the official journal of the World Federation of Hemophilia. 2022
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Abstract
INTRODUCTION Current treatment of severe haemophilia A includes prophylaxis with factor VIII (FVIII) replacement. The supply of plasma-derived FVIII is short in China. PURPOSE To evaluate the efficacy and safety of a new B-domain deleted (BDD) recombinant FVIII (TQG202) produced by human-derived cells for prophylaxis in severe haemophilia A patients and compare the bioequivalence with Xyntha. METHODS This multicentre, clinical trial consisted of an open-label, randomized, two-period cross-over trial assessing single-dose pharmacokinetics (PK), and a single-arm clinical trial evaluating the efficacy and safety of 24 weeks of TQG202 prophylaxis, and repeated PK were assessed after prophylaxis phase. The single-dose was 50 IU/kg in PK assessment, and the initial dose was 30 ± 5 IU/kg for prophylaxis. The primary endpoints of prophylaxis were the annualized bleeding rate (ABR) and the incremental recovery rate of the first administration. Adverse events (AEs) were recorded. RESULTS Twenty-six participants were enrolled in the PK assessment and 81 participants in the prophylaxis phase. Mean age was 25.9 ± 10.8 years and all participants were male. The results of PK assessment showed TQG202 is bioequivalent to Xyntha. The total ABR was 2.0 (95% CI: 1.2-2.9) in prophylaxis phase. The mean incremental recovery rate of the first administration was .027 (95% CI: .026-.028) (IU/ml)/(IU/kg). AEs occurred in 42 participants, with an incidence of 51.9%. One severe AE not related to TQG202 occurred. No participants developed FVIII inhibitors. CONCLUSION TQG202 shows bioequivalence with Xyntha. The promising efficacy and tolerability in the severe haemophilia A prophylaxis support the use of TQG202in clinical practice.
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Language : eng
Credits : Bibliographic data from MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine