At the International Society on Thrombosis and Haemostasis (ISTH) 2015 Congress, Novo Nordisk, a global healthcare company, announced the latest interim data from the guardian 2 extension trial, which showed that in a phase 3 trial, Novoeight (Antihemophilic Factor [Recombinant]) provided long-term efficacy and safety in the prophylaxis and treatment of bleeds in people with severe hemophilia A.

The pivotal guardian clinical programme was one of the largest and most comprehensive preregistration clinical trial programmes in hemophilia A, with more than 210 patients with severe hemophilia A treated. Interim data collected through December 31, 2013, from 451.6 patient-years on Novoeight show results consistent with previous reports.

The overall estimated median annual bleeding rate (ABR) achieved during the preventive regimen with Novoeight was 1.56. During the preventive regimen, 90 per cent of all bleeding episodes were successfully treated with 1 or 2 injections of Novoeight.

"These interim results provide an extension to the body of evidence supporting the long-term use of Novoeight," said Dr. Margareth Ozelo, IHTC Hemocentro Unicamp, University of Campinas, Sao Paulo, Brazil, and guardian 2 investigator. "For people with hemophilia A, finding treatments that are effective at preventing bleeding episodes long-term is essential."

Guardian 2 is a large, multinational extension trial of Novoeight in previously treated patients with hemophilia A from 19 countries who had been enrolled in the pivotal guardian 1 and guardian 3 trials. Patients received Novoeight in a prophylactic regimen and to treat breakthrough bleeds.

These findings now comprise more than 4 years of data and more than 450 patient- years. No inhibitors to factor VIII were detected and no safety issues were identified, supporting the findings from guardian 1 and 3 demonstrating no confirmed inhibitor development in 213 previously treated patients. The most common adverse reactions (=0.5 per cent) seen in the study were injection site reactions, increased hepatic enzymes, and fever. Novoeight is not used to treat von Willebrand Disease.

Common side effects of Novoeight include swelling or itching at the location of injection, changes in liver tests and fever.

Novoeight is a B-domain truncated recombinant human coagulation factor VIII for the treatment and prevention of bleeding in patients with hemophilia A. Novoeight has been launched in 11 countries including the United States (April 2015) and Japan (May 2014).

Hemophilia is a chronic, inherited bleeding disorder that primarily affects males. People with hemophilia A are either missing or have a malfunctioning factor VIII protein, which is essential for proper blood clotting. People with hemophilia A have a tendency to bleed longer than most or to bleed internally into joints, muscles, or organs because they are missing this clotting factor. To manage the disease and stop bleeding, people with hemophilia A must replace the missing factor VIII protein, which is accomplished by intravenous injection of the clotting factor.

Globally, it is estimated that 142,000 people have been diagnosed with hemophilia A. The disease is severely underdiagnosed in developing countries.