Von Willebrand disease product approved in Europe

The safety analysis included 32 patients who received rVWF-rFVIII. There were no thrombotic events, serious adverse events, or new cases of inhibitors to VWF or FVIII in these patients.

The pharmacokinetic analysis included 19 patients. The researchers said the pharmacokinetics of rVWF ristocetin cofactor activity, VWF antigen, and collagen-binding activity were similar with rVWF-rFVIII and pdVWF-pdFVIII.

FVIII levels were higher after infusion with rVWF-rFVIII than with pdVWF-pdFVIII, even after 72 hours. These results were published in Blood (2013 Aug 1;122[5]:648-57).The phase 3 nonsurgical study (NCT01410227) included 49 patients with VWD who received vonicog alfa with or without rFVIII.

All participants had successful treatment of bleeding episodes. Nearly all (96.9%) treated bleeds were given an “excellent” efficacy rating and most (81.8%) were resolved with a single infusion of vonicog alfa; the treatment had a mean half-life of 21.9 hours.

There were eight adverse events considered related to vonicog alfa, two of which were serious. There were no thrombotic events, no treatment-related binding or neutralizing antibodies against VWF, and no neutralizing antibodies against FVIII. These results were published in Blood (2015 Oct 22;126[17]:2038-46).

The phase 3 surgical trial (NCT02283268) enrolled 15 adults with severe VWD who were undergoing elective surgical procedures.