Rebinyn (nonacog beta pegol) / Novo Nordisk - LARVOL DELTA

Factor IX gene variants may influence the pharmacokinetic profile of patients with hemophilia B treated with FIX-Fc or N9-GP extended half-life FIX concentrates. (PubMed, J Thromb Haemost) - "The pharmacokinetics of EHL FIX products are influenced by their ability to access the extravascular space. For products with substantial extravascular distribution, patient CRM status further modulates pharmacokinetic behaviour."

Journal • PK/PD data • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Simulation-Based Comparison of Extended Half-Life Factor IX Prophylaxis Using a Repeated Time-to-Event Model (ISTH 2026) - "Product-specific PK models and label- based regimens were applied: Refixia® (one-compartment PK, 40 IU/kg weekly) calibrated to match the PARADIGM™4 phase III extension mean trough (21.3 IU/dL); Alprolix® (three-compartment PK, 50 IU/kg weekly); and Idelvion® (two-compartment PK, 35 IU/kg weekly) (Figure 1). Limitations: This simulation-based analysis does not replace randomized trials and should be considered hypothesis-generating. Table or Figure Upload (1) Table 1 Page 2 Table or Figure Upload (2) Figure 1 DOI*10.1016/j.rpth.2026.105816 Page 3"

Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Evaluation of laboratory assays for post-infusion monitoring of nonacog beta pegol (ISTH 2026) - "In centers with a limited number of aPTT reagents, the OSA with a calibration curve constructed using each EHL product appears to be the most suitable option. Table or Figure Upload (1) Page 2 Table or Figure Upload (2) DOI*10.1016/j.rpth.2026.104870"

Gene Therapies

Nonacog Beta Pegol (N9-Gp) Prophylaxis in Pediatric Patients with Severe Hemophilia B: A Single Center Real World Experience. (PubMed, Indian J Hematol Blood Transfus) - "Mild adverse effects were noted in 20% of the patients. N9-GP prophylaxis compared to PD/r FIX replacement is a safe and effective strategy in prevention of bleeds, improving QOL and reducing joint related disability in pediatric patients with Hemophilia B."

Journal • Real-world evidence • Hematological Disorders • Hemophilia • Hemophilia B • Pain • Pediatrics • Rare Diseases

Post-marketing Surveillance (Use Result Surveillance) With Refixia® (clinicaltrials.gov) - P=N/A | N=28 | Completed | Sponsor: Novo Nordisk A/S | Enrolling by invitation ➔ Completed | N=20 ➔ 28

Enrollment change • Trial completion • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Monitoring of Nonacog Beta Pegol: One-Stage Clotting Assay With Kaolin Reagent as a Practical Alternative to Chromogenic Methods. (PubMed, Haemophilia) - "STA CK Prest and Biophen FIX showed acceptable recoveries for N9-GP, with FIX:C results in good agreement in both in vitro and post-infusion samples. This supports the reliability of STA CK Prest for FIX:C measurement."

Journal

Patient and Clinical Experience on Concizumab Clinical Trial: A Case Study (ASH 2024) - P=N/A, P3 | "The maintenance dose was initiated on week 4 based on a concizumab exposure of 97.9 ng/mL.Prior to explorer8, the patient was intensely treated with daily BeneFIX® and Rebinyn® prophylaxis during explorer6, but both FIX standard and extended half-life treatment failed to prevent bleeding episodes.Key Clinical Question : What factors should be considered when determining whether a patient should be withdrawn from a clinical trial?Clinical Approach : Throughout the clinical trial, the investigator discussed withdrawal from the study because of an increase in the number of bleeding episodes; however, the patient remained in the clinical trial as the patient and their family preferred concizumab over previous prophylaxis regimens. This case demonstrates that we may have more to learn from our patients on why they choose to participate and/or stay on clinical trials.Disclaimer : Novo Nordisk had no influence on the content of the ABSTRACT . The studies..."

Case study • Clinical • Hematological Disorders • Hemophilia • Hemophilia B • Infectious Disease • Novel Coronavirus Disease • Rare Diseases

Comparing Real-World Outcomes of Prophylaxis with Extended Half-life Factor IX (rIX-FP vs. rFIXFc and N9-GP) for Haemophilia B: An Analysis of Medical Chart Data from Germany. (PubMed, Adv Ther) - "rIX-FP prophylaxis was associated with significantly lower FIX consumption and numerically (but not significantly) lower bleeding rates compared with rFIXFc. Compared to N9-GP, prophylaxis with rIX-FP was associated with similar FIX consumption and significantly lower bleeding rates."

Journal • Real-world evidence • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Performance Evaluation of Different FIX Activity Assays for Determining Nonacog Beta Pegol (N9-GP, Refixia®) Activity Using Reagent-/Platform-Specific Conversion Factors. (PubMed, Hamostaseologie) - "However, we recommend that every laboratory ascertain its own conversion factor. When measuring real patient samples, we encourage simultaneous measurement of N9-GP spiked control material with known concentrations to ensure the validity of the current back-calculation."

Journal • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

REAL-WORLD EXPERIENCE WITH NONACOG BETA PEGOL IN HEMOPHILIA B AFTER 30 MONTHS OF FOLLOW-UP (EHA 2025) - "A retrospective review of the medical records of two patients who switched from SHL to EHL FIX was conducted to assess their clinical outcomes and therapeutic response.Patient 1 (46 years old, 72 kg): Previously on prophylaxis with Nonacog Alfa (SHL) at 4,000 IU twice weekly. Prophylactic therapy is highly effective in reducing bleeding episodes and their long-term complications, especially in patients with severe factor deficiency or a bleeding phenotype.Treatment should be individualized, considering the risk/benefit ratio alongside patient values and preferences, to determine the optimal timing and choice of prophylaxis.Recombinant factor products are a safe and effective treatment option for patients with .The availability of extended half-life factor IX products offers substantial prolongation of half-life, improving patient quality of life, reducing infusion frequency, and enhancing treatment adherence.Significant economic impact is observed, with a reduction in..."

Clinical • Real-world • Real-world evidence • Cerebral Hemorrhage • CNS Disorders • Gastroenterology • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Osteoarthritis • Rare Diseases

Association between FIX levels & bleeding rates in hemophilia B patients receiving rFIXFc or N9-GP (ISTH 2025) - "However, these estimates were highly imprecise and statistically non-significant. Table or Figure Upload"

Clinical • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

The Indian Experience With Nonacog Beta Pegol for the Treatment of Hemophilia B in Children and Adolescents: A Safety and Efficacy Evaluation. (PubMed, Cureus) - "None of them developed any adverse drug reactions or thromboembolic events during the entire course of treatment. None of the patients developed inhibitors.  N9-GP has been shown to be well tolerated, safe, and efficacious in the treatment and prevention of bleeding episodes in people with hemophilia B."

Journal • Cardiovascular • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Growing up with N9-GP: Safety and efficacy of prophylaxis for up to eleven years in children with haemophilia B (EAHAD 2025) - No abstract available

Clinical • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Erratum: TKR in Hemophilic Arthropathy: A Combination of Special Surgical Considerations and Novel Nonacog Beta Pegol: A Case Report. (PubMed, JBJS Case Connect) - No abstract available

Journal • Rheumatology

Pharmacokinetic model-based assessment of factor IX prophylaxis treatment regimens in severe hemophilia B. (PubMed, Sci Rep) - "A population pharmacokinetic (popPK) model-based assessment comparing the performance of FIX products (rFIX, rIX-FP, rFIXFc, N9-GP) was developed. N9-GP use in adolescents and adults was associated with lower consumption compared with rIX-FP. Insights from this study may be utilized by clinicians to inform decision-making, by considering the model-generated estimated optimal posologies alongside multiple clinical factors and patient preferences."

Journal • PK/PD data • Hematological Disorders • Hemophilia • Rare Diseases

TKR in Hemophilic Arthropathy: A Combination of Special Surgical Considerations and Novel Nonacog Beta Pegol: A Case Report. (PubMed, JBJS Case Connect) - "Hemophilias commonly result in end-stage hemophilic arthropathy of the joints at a young age that may warrant joint replacement surgeries. This case report illustrates the surgical protocol of total knee arthroplasty in a patient who received a long-acting factor IX preparation."

Journal • Hematological Disorders • Hemophilia • Musculoskeletal Diseases • Orthopedics • Pain • Rare Diseases • Rheumatology

Paradigm9: A Research Study Investigating Nonacog Beta Pegol (N9-GP) for Treatment and Prevention of Bleedings in Chinese People With Haemophilia B (clinicaltrials.gov) - P3 | N=30 | Completed | Sponsor: Novo Nordisk A/S | Active, not recruiting ➔ Completed

Trial completion • Hematological Disorders • Hemophilia • Rare Diseases

Haemophilia B therapy: impact of the reimbursability of a long-acting recombinant factor on pharmaceutical expenditure in Italy (PubMed, Epidemiol Prev) - "this predictive spending assessment may be useful in evaluating the economic impact from new treatment options, such as etranacogene dezaparvovec gene therapy already approved by the European Medicines Agency and the Food and Drug Administration, and to improve pharmaceutical governance."

Journal • Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases

FIX Post infusion monitoring surveys: Results and analysis from the updated UK NEQAS BC Haemophilia programmes 2024 (ISTH 2024) - "Data was returned from 47 centres. Centres performed either one stage assays (OSA), chromogenic assays (CA) or both. OSA Alprolix Median value, 40 IU/dL with CV = 8.7%; Benefix Median value, 14.3 IU/dL with CV = 13.6%; Idelvion Median value, 20.4 IU/dL with CV = 14.5% and Refixia Median value, 15.4 IU/dL with CV = 18.2% CA Alprolix Median value, 35.7 IU/dL; Benefix Median value, 11.7 IU/dL; Idelvion Median value, 24.8 IU/dL and Refixia Median value, 13.4 IU/dL."

Hematological Disorders • Hemophilia • Rare Diseases

Evaluation of recovery of factor IX replacement therapy with HemosIL Chromogenic Factor IX (ISLH 2024) - "HemosIL Chromogenic Factor IX demonstrated the ability to correctly measure FIX activity in plasma pools with 3 FIX replacement therapies: Alpha Nine SD, BeneFIX, and REBINYN. Results with albutrepenonacog alpha (recombinant FIX fused with recombinant human albumin; Idelvion) demonstrated over recovery of 59% when compared to the theoretical value based on the potency assignment by the manufacturer. In 2019 Rosén et al."

Hematological Disorders • Hemophilia • Rare Diseases

Real-world outcomes of prophylaxis with extended half-life factor IX products for hemophilia B: a pooled analysis of medical chart data from Germany and Italy (ISTH 2024) - "Overall, 225 PwHB were included (mean age 34.1 years; 66% with severe hemophilia B; Table 1). Pooled results for consumption and bleeding rates are presented in Table 2. PwHB treated with rIX-FP prophylaxis had significantly lower FIX consumption compared to those treated with rFIXFc: mean 42.4 IU/kg/week vs."

Real-world • Real-world evidence • Retrospective data • Hematological Disorders • Hemophilia • Rare Diseases

Pharmacokinetic study of nonacog beta pegol with differents models (ISTH 2024) - "4 patients treated with Nonacog beta pegol from 2 Spanish hospitals were recruited. Median age was 40,7 years, median weight 83Kg. The mean activity-time points per patient were 3,2."

PK/PD data • Hematological Disorders • Hemophilia • Rare Diseases

A Canadian Roundtable Discussion of the Peri-operative Use of Nonacog beta pegol (N9-GP) for Persons with Moderate to Severe Hemophilia B (ISTH 2024) - "The panel favored dosing for higher target FIX perioperative (~120 IU/dL) and trough levels (~80 IU/dL) than WFH guidelines recommendations, for bleed prevention and facilitation of post-operative physiotherapy/rehabilitation. They emphasized individualization taking into consideration patient- and surgical-factors, and post-operative rehabilitation needs. Conclusion(s) : The prolonged N9-GP half-life enables maintenance of higher trough levels and potential reduction in associated healthcare systems and patient-related costs."

Hematological Disorders • Hemophilia • Orthopedics • Rare Diseases

Interim results from a non-interventional, post authorisation safety study of nonacog beta pegol as routine prophylaxis in patients with haemophilia B (ISTH 2024) - "At interim cut-off (31/03/2022), 41 patients were enrolled; mean (range) age: 34 years (9–74); disease severities: 2% mild, 27% moderate, 66% severe, 5% missing. Four patients withdrew (one withdrawal of consent; three on physician decision [one moved from centre, two due to bleedings]). Cumulative N9 GP exposure time: 40 patient-years."

Clinical • Cardiovascular • Gastroenterology • Hematological Disorders • Hemophilia • Hepatology • Immunology • Pain • Peptic Ulcer • Rare Diseases

Clinical outcomes of people with hemophilia B treated with N9-GP in real-world settings: the Adelphi Disease Specific Programme™ hemophilia study (ISTH 2024) - "119 PwHB receiving N9-GP prophylaxis were included in the analysis (characteristics shown in Table 1). 91% (105/116) received N9-GP weekly, with 88% (102/116) taking a 40 U/kg dose, as per label recommendations. 42% (n=50) PwHB receiving N9-GP prophylaxis were judged to have ever had hemophilia-related joint problems, of which most (n=33, 80.5%) had ≥1 target joint."

Clinical • Clinical data • Real-world • Real-world evidence • Hematological Disorders • Hemophilia • Rare Diseases