Date: 2015-06-24
Type of information: Presentation of results at a congress
phase: 3
Announcement: presentation of results at the International Society on Thrombosis and Haemostasis (ISTH) 2015 Congress
Company: Novo Nordisk (Denmark)
Product: NovoEight® (turoctocog alfa)
Action
mechanism: blood coagulation factor. Turoctocog alfa is a third-generation recombinant coagulation factor VIII intended for prevention and treatment of bleeding in people with hemophilia A.
Disease: hemophilia A
Therapeutic area: Hematologic diseases - Genetic diseases - Rare diseases
Country:
Trial
details: Guardian™2 is a large, multinational extension trial of NovoEight® in previously treated patients with haemophilia 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.
Latest
news: * On June 24, 2015, Novo Nordisk announced the latest interim data from the guardian™2 extension trial at the International Society on Thrombosis and Haemostasis (ISTH) 2015 Congress. These data showed that in a phase 3 trial, NovoEight® (turoctocog alfa) provided long-term efficacy and safety in the prophylaxis and treatment of bleeds in people with severe haemophilia A. * On May 12, 2014, Novo Nordisk announced new phase 3 interim data from its guardianT2 trial for its recombinant coagulation factor VIII (rFVIII) product NovoEight® (turoctocog alfa), which shows that it provides long-term reduction from bleeding in people with hemophilia A when used as a preventative treatment.1 The results were presented at the World Federation of Hemophilia (WFH) World Congress and support findings from other studies within the guardianT clinical programme that found NovoEight® demonstrated good efficacy in preventing and treating bleeds without inhibitor development in previously treated patients. Phase 3 guardianT2 results: GuardianT2 is the extension of the pivotal guardianT clinical programme, one of the largest and most comprehensive pre-registration clinical trial programmes in hemophilia, with more than 210 severe haemophilia A patients treated. GuardianT2 is an open-label, multinational, single-arm extension trial involving 188 haemophilia A patients from 18 countries who had been previously enrolled in the guardianT1 and guardianT3 trials. Patients received NovoEight® in a preventative regimen and to treat breakthrough bleeds. Interim results found: . The overall estimated annual bleeding rate (ABR) achieved during preventative regimen with NovoEight® was 1.7 (median 3.1) bleeds/patient/year, ranging from 1.4 (children aged 0-5) to 1.9 for adults (median number of bleeds/patient/year)
The pivotal guardian™ clinical programme was one of the largest and most comprehensive preregistration clinical trial programmes in haemophilia A, with more than 210 patients with severe haemophilia A treated. Interim data collected through 31 December 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% of all bleeding episodes were successfully treated with one or two infusions of NovoEight®.
These findings now comprise more than four 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%) seen in the study were injection-site reactions, increased hepatic enzymes and fever.
. Preventative regimen with NovoEight® led to a decrease in ABR, followed by stabilisation at a lower level over the time period assessed.
