Novo Nordisk announced a new analysis of phase 3 data demonstrating people with haemophilia A who had the highest annualised bleeding rate (ABR) during initial treatment with NovoEight (turoctocog alfa) showed the largest reduction in bleeding over the duration of treatment. Data for the recombinant factor VIII (rFVIII) product NovoEight were presented at the 56th Annual Meeting of the American Society of Hematology (ASH).
The post hoc analysis from the guardianT2 trial further found that, when continued on treatment with NovoEight, adults and adolescents showed reductions of 69 per cent and 63 per cent, respectively, in ABR compared with their ABRs from guardianT1. The guardianT1 trial (along with guardianT3) showed that NovoEight was efficacious in preventing and reducing bleeds with no confirmed inhibitor development in 213 previously treated patients.
"This post hoc analysis gives an indication that patients with high baseline bleed rate can benefit from prophylaxis with NovoEight, as well as patients who maintain low ABR," said Margareth Ozelo, MD, Hemocentre, IHTC, University of Campinas, Sao Paulo, Brazil, and lead investigator.
The most common adverse reactions (0.5 per cent) seen in the study were injection site reactions, increased hepatic enzymes, and fever.4
NovoEight is approved by the US Food and Drug Administration (October 2013) and the European Medicines Agency (November 2013) for the treatment and prophylaxis of bleeding in patients with haemophilia A. NovoEight is also approved in Japan (January 2014), Australia (January 2014), and Switzerland (February 2014), and applications for regulatory approvals have been submitted in a range of other countries.
The pivotal guardianT clinical programme is one of the largest and most comprehensive preregistration clinical trial programmes in haemophilia, with more than 210 patients with severe haemophilia A treated. The guardianT2 trial is an extension of the clinical programme. The post hoc analysis of the guardianT2 trial involved 166 patients who had participated in guardianT2 and either guardianT1 or 3, had 1 week of surgery treatment in guardianT1 or 3 (initial period), and had 3 months of exposure to turoctocog alfa prophylaxis during a selected time period (1 January 2012-30 June 2013) in guardianT2.
Haemophilia is a chronic, inherited bleeding disorder that primarily affects males. People with haemophilia A are either missing or have a malfunctioning factor VIII protein, which is essential for proper blood clotting. People with haemophilia A have a tendency to bleed longer than most or to bleed internally into muscles, joints or organs because they are missing this clotting factor. To manage the disease and stop bleeding, people with haemophilia A must replace the missing factor VIII protein, which is accomplished by intravenous injection of the clotting factor.
Globally, it is estimated that more than 400,000 people have haemophilia. The disease is severely underdiagnosed in developing countries.