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UK
21 May 2018 11:45 CET

Head-to-head trial shows Refixia® achieves greater total factor IX exposure in people with haemophilia B than recombinant factor IX-Fc fusion protein

Glasgow, UK, 21 May 2018 ­- Adults with haemophilia B who received a single dose Refixia® (nonacog beta pegol; N9-GP) achieved greater total factor IX exposure than those treated with rFIXFc (recombinant factor IX-Fc fusion protein). The head-to-head paradigm7 trial also observed a longer half-life for Refixia®. These pharmacokinetic findings were presented today at the WFH 2018 World Congress in Glasgow, UK.1

The trial showed that the total factor IX exposure of Refixia® in patient plasma after injection of a single dose (50 IU/kg) was 4.39-fold greater than for rFIXFc, as measured by the area under the curve (AUC). Seven days after injection, the factor IX activity in patients treated with a single dose of Refixia® was six-fold greater than that of patients treated with rFIXFc at the same dose.

"As a clinician, I know first-hand how challenging it can be to help people living with haemophilia B reach their treatment goals and be adequately protected from bleeding" said Dr Carmen Escuriola Ettingshausen of the Rhein-Main Haemophilia Centre (HZRM), Germany. "These data will help us better understand the different treatment options and choose the appropriate treatment for each patient."

The half-life of Refixia® in the trial was 103.2 hours, significantly longer than the half-life of rFIXFc (84.9 hours). In addition, total factor IX activity levels 30 mins after infusion were two-fold greater with Refixia® compared to rFIXFc. No patients developed inhibitors and no safety concerns were identified during the trial.

"People with haemophilia B and their clinicians want to be confident that they are protected from bleeds without worrying" said Mads Krogsgaard Thomsen, executive vice president and chief science officer of Novo Nordisk. "These head-to-head data support the strong clinical profile of Refixia® which can provide an alternative treatment option for people with haemophilia B with the convenience of a single weekly dose."

About the paradigm7 trial

Paradigm7 was a multicentre, open-label, randomised, crossover, pharmacokinetic trial in 15 previously treated adult males with congenital haemophilia B (factor IX activity <2%), conducted in three countries (US, Germany, Switzerland). Patients received single injections (50 IU/kg) of Refixia® and rFIXFc with >21 days between doses. The primary endpoint was dose-normalised area under the factor IX activity-time curve from 0 to infinity (AUC0-inf,norm).

About Refixia®
Refixia® (nonacog beta pegol; N9-GP) is an extended half-life factor IX molecule for replacement therapy in patients with haemophilia B.2 The review of Refixia® was based on the paradigm clinical programme. In the completed phase 3 trials, 115 previously treated patients had a total of more than 8,800 exposure days for up to 2.7 years of treatment with Refixia®.

About haemophilia B
Haemophilia is a chronic, inherited bleeding disorder that primarily affects males. People with haemophilia B have congenital factor IX deficiency and are either missing or have a malfunctioning factor IX protein, which is needed for proper blood clotting.3

Globally it is estimated that 30,000 people have been diagnosed with haemophilia B. The disease is severely underdiagnosed in some regions of the world.4

Further information

Media:    
Katrine Sperling +45 4442 6718 krsp@novonordisk.com
Åsa Josefsson +45 3079 7078 aajf@novonordisk.com
     
Investors:    
Peter Hugreffe Ankersen +45 3075 9085 phak@novonordisk.com
Anders Mikkelsen +45 3079 4461 armk@novonordisk.com
Christina Kjær +45 3079 3009 cnje@novonordisk.com


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References

1.       Escuriola Ettingshausen C, Hegemann I, Simpson M, et al. A head-to-head pharmacokinetic comparison of N9-GP and rFIXFc in patients with haemophilia B. Presented at the WFH 2018 World Congress, Glasgow, UK, 20-24 May. 2018
2.       EMA. Refixia®. Summary of Product Characteristics. Available at: http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Product_Information/human/004178/WC500232816.pdf. Last accessed: April 2018.
3.       Srivastava A, Brewer AK, Mauser-Bunschoten EP, et al. Guidelines for the management of hemophilia. Haemophilia. 2013;19:e1-47
4.       National Hemophilia Foundation. Fast Facts. Available at: http://www.hemophilia.org/About-Us/Fast-Facts. Last accessed: April 2018.