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June 2023 - Katarina D. Kovačević


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Mag.pharm. Katarina Kovačević, PhD


Von Willebrand factor (VWF)-binding aptamer called Rondoraptivon pegol (previously BT200) increased plasma levels of VWF/FVIII in healthy volunteers. In the awarded publication, its safety, pharmacokinetics, and pharmacodynamics were investigated in hemophilia A. Nineteen adult patients with hemophilia A received subcutaneous injections of rondoraptivon pegol. All patients tolerated rondoraptivon pegol well. The geometric mean half-life of rondoraptivon pegol was [5].4 days and rondoraptivon pegol significantly increased VWF levels. In severe hemophilia A, rondoraptivon pegol increased the half-lives of FVIII products from a median of 10.4 hours to 31.1 hours. Median FVIII increased from 22% to 48% in mild hemophilia A and from 3% to 7.5% in moderate hemophilia A. Rondoraptivon pegol is a first-in-class prohemostatic molecule that extended the half-life of substituted FVIII approximately 3-fold and increased endogenous FVIII levels approximately 2-fold in hemophilia patients.

Selected Literature

  1.  Ay C, Kovacevic KD, Kraemmer D, et al. von Willebrand Factor-binding aptamer rondoraptivon pegol as treatment for severe and non-severe hemophilia A. Blood. 2022.

  2. White GC, 2nd, Rosendaal F, Aledort LM, Lusher JM, Rothschild C, Ingerslev J. Definitions in hemophilia. Recommendation of the scientific subcommittee on factor VIII and factor IX of the scientific and standardization committee of the International Society on Thrombosis and Haemostasis. Thrombosis and haemostasis. 2001;85(3):560.

  3. Sriv astava A, Santagostino E, Dougall A, et al. WFH Guidelines for the Management of Hemophilia, 3rd edition. Haemophilia : the official journal of the World Federation of Hemophilia. 2020;26 Suppl 6:1-158.

  4. Pipe SW, Montgomery RR, Pratt KP, Lenting PJ, Lillicrap D. Life in the shadow of a dominant partner: the FVIII-VWF association and its clinical implications for hemophilia A. Blood. 2016;128(16):2007-2016.

  5. Tomeo F, Mariz S, Brunetta AL, Stoyanova-Beninska V, Penttila K, Magrelli A. Haemophilia, state of the art and new therapeutic opportunities, a regulatory perspective. Br J Clin Pharmacol. 2021.

  6. Callaghan MU, Negrier C, Paz-Priel I, et al. Long-term outcomes with emicizumab prophylaxis for hemophilia A with or without FVIII inhibitors from the HAVEN 1-4 studies. Blood. 2021;137(16):2231-2242.

  7. Weyand AC, Pipe SW. New therapies for hemophilia. Blood. 2019;133(5):389-398.

  8. Gelbenegger G, Schoergenhofer C, Knoebl P, Jilma B. Bridging the Missing Link with Emicizumab: A Bispecific Antibody for Treatment of Hemophilia A. Thrombosis and haemostasis. 2020;120(10):1357-1370.

  9. Versloot O, Iserman E, Chelle P, et al. Terminal half-life of FVIII and FIX according to age, blood group and concentrate type: Data from the WAPPS database. Journal of thrombosis and haemostasis : JTH. 2021;19(8):1896-1906.

  10.  Peyvandi F, Tavakkoli F, Frame D, et al. Burden of mild haemophilia A: Systematic literature review. Haemophilia : the official journal of the World Federation of Hemophilia. 2019;25(5):755-763.

  11.  Scott MJ, Xiang H, Hart DP, et al. Treatment regimens and outcomes in severe and moderate haemophilia A in the UK: The THUNDER study. Haemophilia : the official journal of the World Federation of Hemophilia. 2019;25(2):205-212.

  12.  Lim MY, Cheng D, Recht M, Kempton CL, Key NS. Inhibitors and mortality in persons with nonsevere hemophilia A in the United States. Blood Adv. 2020;4(19):4739-4747.

  13.  Abdi A, Eckhardt CL, van Velzen AS, et al. Treatment-related risk factors for inhibitor development in non-severe hemophilia A after 50 cumulative exposure days: A case-control study. Journal of thrombosis and haemostasis : JTH. 2021;19(9):2171-2181.

Mag.a pharm. Katarina D. Kovačević, PhD

Medizinische Universität Wien
Universitätsklinik für Klinische Pharmakologie
Währinger Gürtel 18-20
1090 Wien

T: +43 (0)1 40400-73749