Copenhagen, Denmark, 12 May, 2014/PRNewswire/– Today, Novo Nordisk announced new phase 3 interim data from its guardianTM2 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 guardianTM clinical programme that found NovoEight® demonstrated good efficacy in preventing and treating bleeds without inhibitor development in previously treated patients.2,3
Read more Read more

Commenting on the results, Dr Margareth Ozelo, Hemocentre, IHTC, University of Campinas, Sao Paulo, Brazil and guardianTM2 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 months giving patients the freedom to administer whenever and wherever needed.5,6

Phase 3 guardianTM2 results:

GuardianTM2 is the extension of the pivotal guardianTM 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. GuardianTM2 is an open-label, multinational, single-arm extension trial involving 188 haemophilia A patients from 18 countries who had been previously enrolled in the guardianTM1 and guardianTM3 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 3.1 (median 1.7) 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.

About NovoEight®

NovoEight® has been approved by the FDA (October 2013) and EMA (November 2013) for the treatment and prophylaxis of bleeding in patients with haemophilia A. NovoEight® has also been approved in Japan and Australia (January 2014) and in Switzerland (February 2014) and applications for regulatory approvals have been submitted in a range of other countries.

Novo Nordisk in Haemophilia

In addition to NovoEight®, Novo Nordisk has developed a comprehensive portfolio of molecules aimed at improving haemophilia treatment. Marketed products include NovoSeven® for the treatment of spontaneous bleeds and for coverage during surgery in patients with haemophilia and inhibitors (antibodies) against factor VIII (FVIII) and factor IX (FIX) (haemophilia A and B with inhibitors) and NovoThirteen® for FXIII congenital deficiency, a very rare and serious bleeding disorder with limited treatment options. Molecules in the clinical development pipeline include N8-GP, designed to provide a longer half-life and the potential to reduce the treatment burden for people with haemophilia A and N9-GP, a long-acting recombinant factor IX for haemophilia B that has the potential to significantly reduce bleeding with weekly injections.

About Haemophilia A

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 420,000 people have haemophilia.7 The disease is severely under-diagnosed in developing countries.

Headquartered in Denmark, Novo Nordisk is a global healthcare company with 90 years of innovation and leadership in diabetes care. The company also has leading positions within haemophilia care, growth hormone therapy and hormone-replacement therapy. Novo Nordisk employs approximately 40,000 employees in 75 countries, and markets its products in more than 180 countries. For more information, visit novonordisk.com.

Further information
Media:

Katrine Sperling, +45 3079 6718, krsp@novonordisk.com
Ken Inchausti (US), +1 609 514 8316, kiau@novonordisk.com

Investors:

Kasper Roseeuw Poulsen, +45 3079 4303, krop@novonordisk.com
Jannick Lindegaard Denholt, +45 3079 8519, jlis@novonordisk.com
Lars Borup Jacobsen, +45 3075 3479, lbpj@novonordisk.com
Daniel Bohsen, +45 3079 6376, dabo@novonordisk.com
Frank Daniel Mersebach (US), +1 609 235 8567, fdni@novonordisk.com

References

  1. Ozelo M. et al. Lowering in annualized bleeding rates over time with turoctocog alfa prophylaxis: 3-year interim results of the guardianTM2 extension trial. Oral presentation (FP-M-01)at WFH 2014, 12th May 2014.
  2. Lentz SR. et al.Results from a large multinational clinical trial (guardianTM1) using prophylactic treatment with turoctocog alfa in adolescent and adult patients with severe haemophilia A: safety and efficacy. Haemophilia2013;19(5):691-7.
  3. Kulkarni R. et al.Results from a large multinational clinical trial (guardianTM3) using prophylactic treatment with turoctocog alfa in paediatricpatients 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
  7. Internal calculation, data on file