Lonoctocog alfa (rVIII-SingleChain) for the treatment of haemophilia A.
(2018) Expert Opinion on Biological Therapy — Vol. 18, n° 1, p. 87-94 (2018)
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- Abstract
- The administration of factor VIII (FVIII) concentrates on-demand or on long-term prophylaxis is the effective and safe standard of care of patients with hemophilia A (HA). Development of neutralizing antibodies against exogenous FVIII and the short half-life of the current available products remain major challenges. There is currently a great interest towards newer FVIII products with the goal of reducing the inhibitor risk and increasing the half-life. Area covered: In this review, the authors describe the efficacy and safety of rVIII-SingleChain (Lonoctocog alfa), the first and only single chain recombinant FVIII (rFVIII) molecule developed for the prevention and treatment of bleeding episodes in HA patients. The pre-clinical and clinical studies of rVIII-SingleChain as well as the results of the AFFINITY trial program in previously treated patients both adults and pediatric are presented and discussed. Expert opinion: The results from PTP studies document the efficacy and safety profile of the rVIII-SingleChain. However, even if rFVIII-SingleChain presents advantageous pharmacokinetic features compared to conventional rFVIII, it should not be considered as an EHL-FVIII while its immunogenicity is currently being studied in PUPs. The slightly better PK profile of rFVIII-SingleChain could however allow a small number of selected patients to be treated with a less intensive regimen.
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Raso, S., & Hermans, C. (2018). Lonoctocog alfa (rVIII-SingleChain) for the treatment of haemophilia A. Expert Opinion on Biological Therapy, 18(1), 87-94. https://doi.org/10.1080/14712598.2018.1416088 (Original work published 2018)