Novo Nordisk's Long-Acting Factor IX Product Refixia (N9-GP) Demonstrates Significant Reduction in Annualized Bleeding Rate in Pediatric Hemophilia B Patients

July 09, 2019/BioValleyBIOON/-- Danish pharmaceutical giant Novo Nordisk recently presented interim analysis data from two pediatric clinical studies (Paradigm-5 and Paradigm-6) of its long-acting coagulation factor IX product, Refixia/Rebinyn (nonacog beta pegol, N9-GP), at the 27th Annual Congress of the International Society on Thrombosis and Haemostasis (ISTH 2019) held in Melbourne, Australia. The results demonstrated that N9-GP treatment in pediatric patients with hemophilia B was associated with a low annualized bleeding rate and was well tolerated, further reinforcing the long-term safety and efficacy established in previous studies.

The 5-year interim analysis of the Paradigm-5 study demonstrated low bleeding rates in previously treated pediatric patients (≤12 years) with hemophilia B (median annualized bleeding rate [ABR]: overall ABR 0.66, spontaneous bleeding ABR 0.0, traumatic bleeding ABR 0.47). Bleeding rates decreased after 5 years of treatment compared with 1 year of treatment. Twenty percent of children experienced no bleeding episodes, and 64% had no spontaneous bleeding throughout the study. No inhibitory antibodies were detected in any pediatric patient, and no new safety signals were identified.

Ludovic Helfgott, Executive Vice President of Novo Nordisk’s Biopharm Operations, stated: “Reducing and managing bleeding episodes in children with hemophilia B is particularly challenging for their families. We are pleased that these results further support the favorable efficacy of N9-GP in the prevention and treatment of bleeding episodes in children with hemophilia B.”

The efficacy and safety of N9-GP are further supported by preliminary interim results from ≥20 patients who completed 50 exposure days (ED). Previously untreated children (<6 years of age) receiving once-weekly prophylactic therapy reported low bleeding rates and favorable resolution of bleeds, with median annualized bleeding rates (ABRs) of 0.0 for total, spontaneous, and trauma-related bleeds. The incidence of inhibitory antibodies was within the expected range, affecting 2 out of 33 patients (6.1%). No unexpected safety signals were identified.

N9-GP is an extended half-life factor IX product used as replacement therapy for the treatment of hemophilia B. Compared with standard coagulation factor IX products, N9-GP has a half-life that is up to five times longer. This drug is a glycoPEGylated recombinant coagulation factor IX with significantly improved pharmacokinetic (PK) properties. (Bioon.com)

Original Source:New data show nonacog beta pegol (N9-GP) is effective and well tolerated for the prevention and treatment of bleeding in children with haemophilia B