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

• DOI: 10.1111/hae.14652
• Publication Date: 2022-08-23T03:30:27Z
• Authors: Yaming Xi; Chenghao Jin; Wei Liu; Hu Zhou; Zhen Wang; Rongfu Zhou; Shifeng Lou; Xielan Zhao; Fangping Chen; Peng Cheng; Zimin Sun; Haifei Jia; Lei Zhang
• Keywords: Bioequivalence; Tolerability; Haemophilia B

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