Once-weekly prophylaxis with 40 IU/kg nonacog beta pegol (N9-GP) achieves trough levels of >15% in patients with haemophilia B: Pooled data from the paradigm™ trials

Johannes Oldenburg, Manuel Carcao, Steven R. Lentz, Johnny Mahlangu, Maria Elisa Mancuso, Tadashi Matsushita, Claude Négrier, Wan Hui Ong Clausen, Silke Ehrenforth, Guy Young

Research output: Contribution to journalArticle

Abstract

Introduction: Prophylaxis with replacement factor IX (FIX) reduces bleeding frequency and improves quality of life in haemophilia B patients. With prophylaxis, the likelihood of bleeding is lowered with increasing trough levels. New products with extended half-life (EHL) can maintain high factor activity levels over prolonged periods, compared with standard FIX products. Aim: To evaluate the safety, efficacy and pharmacokinetics of the new recombinant FIX EHL product, nonacog beta pegol (N9-GP), using pooled data, with a focus on—but not limited to—prophylaxis at 40 IU/kg. Methods: N9-GP has been investigated in males with congenital haemophilia B and FIX activity ≤2% in the paradigm™ clinical trial programme. This analysis includes pooled data from five completed paradigm™ trials conducted in previously treated adults, adolescents and children, focusing on results of prophylaxis with 40 IU/kg once-weekly intravenous dosing. Results: In total, 115 previously treated patients were exposed to N9-GP. Of 54 patients (47%) treated with N9-GP 40 IU/kg once-weekly prophylaxis, 72% experienced no spontaneous bleeds over 1 year. In all patients receiving 40 IU/kg once-weekly, median overall annualized bleeding rate (ABR) was 1.03 (interquartile range 0.00; 2.89); median spontaneous ABR was 0.00 (0.00; 0.80). No patients developed inhibitors. Estimated mean steady-state trough levels with N9-GP 40 IU/kg once-weekly were ≥15% overall; 27.3% in adolescents and adults. Conclusion: N9-GP 40 IU/kg once-weekly was well tolerated and effective in preventing bleeding, maintaining mean FIX activity levels ≥15% across all age groups. N9-GP may provide a new treatment option for preventing bleeding in haemophilia B patients.

Original languageEnglish
Pages (from-to)911-920
JournalHaemophilia
Volume24
Issue number6
DOIs
Publication statusPublished - 2018

Keywords

  • factor IX
  • haemophilia B
  • nonacog beta pegol
  • prophylaxis
  • target joint
  • trough level

ASJC Scopus subject areas

  • Hematology
  • Genetics(clinical)

Fingerprint Dive into the research topics of 'Once-weekly prophylaxis with 40 IU/kg nonacog beta pegol (N9-GP) achieves trough levels of >15% in patients with haemophilia B: Pooled data from the paradigm™ trials'. Together they form a unique fingerprint.

  • Cite this

    Oldenburg, J., Carcao, M., Lentz, S. R., Mahlangu, J., Mancuso, M. E., Matsushita, T., Négrier, C., Clausen, W. H. O., Ehrenforth, S., & Young, G. (2018). Once-weekly prophylaxis with 40 IU/kg nonacog beta pegol (N9-GP) achieves trough levels of >15% in patients with haemophilia B: Pooled data from the paradigm™ trials. Haemophilia, 24(6), 911-920. https://doi.org/10.1111/hae.13608