The availability of new drugs for hemophilia treatment

Abstract

Introduction: A number of new FVIII/IX concentrates enriched the portfolio of products available for the treatment of hemophilia A/B patients. Due to the large inter-patient variability, accurate tailoring of the therapy became essential to improve patients' adherence, clinical outcomes, and cost/effectiveness ratio. Recently, non-replacement therapies have taken the limelight and succeeded in decreasing the bleedings of patients.

Areas covered: The PK characteristics, efficacy, and safety of the new rFVIII and rFIX concentrates and of non-replacement therapy, are reported in detail in the published clinical trials.

Expert opinion: Outstanding improvements of rFIX concentrates' pharmacokinetics and pharmacodynamics have allowed to reduce the bleedings in hemophilia B patients, in order to increase their adherence to prophylaxis and quality of life. Less significant are the effects of pegylation or Fc fusion on the pharmacokinetics of the new rFVIII concentrates. The new non-replacement therapy is achieving the favor of many treaters and patients, in particular those with Factor VIII inhibitors. Great attention must be paid to the dangerous synergy of APCC and emicizumab, responsible for some fatal events during the clinical trials and compassionate use of this drug. So far, replacement therapy should be the standard of care for hemophilia patients without inhibitors or difficulties in venous access.

Keywords: Concizumab; emicizumab; fitusiran; hemophilia A/B; rFIX EHL; rFVIII EHL.