NovoEight® provides long-term reduction of bleeding in people with haemophilia A

Today, Novo Nordisk announced new phase 3 interim data from its guardian™2 trial for its recombinant coagulation factor VIII (rFVIII) product NovoEight^® (turoctocog alfa), the first new rFVIII molecule in over a decade, which shows that it provides long-term reduction from bleeding in people with haemophilia A when used as a preventative treatment.¹ The results were presented at the World Federation of Haemophilia (WFH) World Congress and support findings from other studies within the guardian™ clinical programme that found NovoEight^® demonstrated good efficacy in preventing and treating bleeds without inhibitor development in previously treated patients.^2,3

Commenting on the results, Dr Margareth Ozelo, Hemocentre, IHTC, University of Campinas, Sao Paulo, Brazil and guardian™2 investigator said, “As a physician treating haemophilia A, having another FVIII product available is good news and can lead to improved treatment outcomes and quality of life for our patients. Reducing the number of bleeding episodes is a key consideration as they are very painful and frightening for patients and caregivers and can lead to severe long-term joint damage or arthropathy.”

NovoEight^® has been carefully designed using advanced protein and purification technology to provide a reliable and portable treatment option for people with haemophilia A combined with a good safety profile.⁴ NovoEight^® offers convenient and flexible storage requirements and can be stored at 30°C/86°F for 6 monthsgiving patients the freedom to administer whenever andwherever needed.^5,6

Phase 3 guardian™2 results

Guardian™2 is the extension of the pivotal guardian™ clinical programme, one of the largest and most comprehensive pre-registration clinical trial programmes in haemophilia, with more than 210 severe haemophilia A patients treated. Guardian™2 is an open-label, multinational, single-arm extension trial involving 188 haemophilia A patients from 18 countries who had been previously enrolled in the guardian™1 and guardian™3 trials. Patients received NovoEight^® in a preventative regimen and to treat breakthrough bleeds. Interim results found:¹

• 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)
• Preventative regimen with NovoEight^® led to a decrease in ABR, followed by stabilisation at a lower level over the time period assessed.

References

1. Ozelo M et al. Lowering in annualized bleeding rates over time with turoctocog alfa prophylaxis: 3-year interim results of the guardian™2 extension trial. Oral presentation (FP-M-01) at WFH 2014, 12 May 2014.
2. Lentz SR et al. Results from a large multinational clinical trial (guardian™1) using prophylactic treatment with turoctocog alfa in adolescent and adult patients with severe haemophilia A: safety and efficacy. Haemophilia 2013; 19(5):691–7.
3. Kulkarni R. et al. Results from a large multinational clinical trial (guardian™3) using prophylactic treatment with turoctocog alfa in paediatric patients with severe haemophilia A: safety, efficacy and pharmacokinetics. Haemophilia 2013; 19(5):698–705.
4. Thim et al. Haemophilia 2010; 16:349–59.
5. Viuff et al. Haemophilia 2011; 17:695–702.
6. Targeted room temperature storage.