Lonoctocog alfa (rVIII-SingleChain) for the treatment of haemophilia A

ABSTRACT

Introduction: 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.

KEYWORDS:

• Haemophilia
• factor VIII
• single-chain

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties. One referee was the lead author for the Lonoctocog pivotal study in adults,

Box 1. Drug summary.

Additional information

Funding

This manuscript has not been funded.