6^th Pharmacovigilance Congress

Biography

Biography: Aleksander Skotnicki

Abstract

Introduction: VONCENTO (CSL Behring) is a plasma-derived, high-concentration, low-volume, high-purity concentrate, which contains a high level of von Willebrand factor (VWF) high-molecular-weight multimers and a  VWF/factor VIII (FVIII) ratio of -2.4:1, similar to Haemate P (CSL Behring).

Methods: The pharmacokinetic, efficacy and safety profiles of VONCENTO were investigated in this multicentre double-blind, randomised study. Subjects aged _>12 years with haemophilia A who required treatment of non-surgical bleeds, treatment during surgical events or who were receiving prophylaxis were included. Pharmacokinetics were investigated with a single dose of 50 IU) FVIII/kg body weight of either VONCENTO or BIOSTATE reference product (Biostate-RP) (Day 1: Day 8 (n = 16), repeated on Day 180 [VONCENTO only; n = 15J).Efficacy and safety analyses were performed either during on-demand treatment (n = 52) or prophylaxis (n = 29)for ≥6 months and ≥50 exposure days, respectively.

Results: Besides the confirmation of bioequivalence between VONCENTO and Biostate-RP, which displayed comparable PK profiles, haemostatic efficacy was rated by the investigators as either "excellent" or " good" in 96.4% of all bleeding events (96.5% spontaneous, 96.6% traumatic. 96.9% joint bleeds) as well as in 80% of  major and 100% of minor surgical procedures at discharge. The median number of annualised bleeding events per subject (range) was significantly lower in the prophylaxis group (2.0 (0.0-34.6)) than in the on-demand group (14.0 (0.0-87.8), p = 0.0013).VONCENTO was well tolerated and no inhibitory antibodies were identified during the study period.

Conclusions: : This study demonstrated the bioequivalence of VONCENTO to Biostate-RP, and its excellent efficacy and safety profile in haemophilia A subjects.