Idelvion (albutrepenonacog alfa) / CSL Behring - LARVOL DELTA

Haemodialysis in haemophilia B: a case report. Challenges and strategies from a nephrology perspective. (PubMed, J Nephrol) - "In this report, we present a case study of a 68-year-old male patient suffering from severe haemophilia B, who was administered prophylactic Idelvion® (albutrepenonacog alfa) and subsequently underwent arteriovenous fistula creation and haemodialysis initiation without encountering any bleeding complications...In addition, we put forward a series of pragmatic recommendations that are -underpinned by a systematic decision-making framework. Through meticulous multidisciplinary planning incorporating the utilisation of factor IX concentrates, meticulous surgical techniques, and tailored anticoagulation protocols, patients with haemophilia B can be deemed eligible for chronic haemodialysis with a reasonable degree of safety."

Journal • Cardiovascular • Chronic Kidney Disease • Hematological Disorders • Hemophilia • Hemophilia B • Nephrology • Rare Diseases • Renal Disease

Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Chinese Subjects With Hemophilia B Previously Treated With FIX Therapy (clinicaltrials.gov) - P3 | N=23 | Completed | Sponsor: CSL Behring | Active, not recruiting ➔ Completed | Trial completion date: Dec 2026 ➔ Jul 2026 | Trial primary completion date: Dec 2026 ➔ Jul 2026

Trial completion • Trial completion date • Trial primary completion date • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Real-World Outcomes of Prophylaxis With rIX-FP in Germany: A Prospective, Non-Interventional Study in Haemophilia B. (PubMed, Haemophilia) - "This multicentre, non-interventional study in Germany demonstrates that rIX-FP prophylaxis is effective and well tolerated in PwHB of all ages in routine clinical practice and reinforces the results of the clinical trials."

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

Presentation Theater 6 Visit the IDELVION® Presentation Theatre to discover how FIX replacement continues to shape hemophilia B car (ISTH 2026) - "Sponsored by CSL"

Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

ORPHEE study: Real-life use of rIX-FP for prophylaxis in adolescent and adult patients with hemophilia B (ISTH 2026) - P | "For the 47 patients previously treated with recombinant FIX Fc fusion protein (rFIXFc), the between-injection interval increased from 9 (5) days with rFIXFc to 11 (4) days with rIX-FP (p<0.0001), and weekly consumption decreased from 64 (29) to 47 (15) IU/kg (p<0.0001). Some patients who switched to rIX-FP were able to extend the between-injection interval and reduce FIX consumption, while maintaining adequate bleed protection. DOI*10.1016/j.rpth.2026.105974"

Clinical • 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

ORPHEE study: Real-life use of rIX-FP for prophylaxis in children with hemophilia B (ISTH 2026) - P | "Six of the 16 patients (37.5%) previously treated with recombinant FIX Fc fusion protein (rFIXFc) were able to extend the between-injection interval from 7.9 (2.1) days with rFIXFc to 9.6 (3.2) days with rIX-FP. Some children who switched to rIX-FP were able to extend the dosing interval while maintaining adequate protection against bleeding. Page 2 DOI*10.1016/j.rpth.2026.105010"

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

Pediatric Cohort Treated With rIX-FP (Idelvion): Real-World Analysis of Efficacy, Safety, PK Improvement and Resource Utilization (ISTH 2026) - "Conclusions In this pediatric real-world cohort—including both PUPs and PTPs—rIX-FP demonstrated excellent safety with no inhibitor development, marked PK improvement, and strong bleeding control. The extended half-life of rIX-FP reduces treatment burden while improving FIX exposure, supporting its role as an effective and efficient prophylactic option for children with hemophilia B. DOI*10.1016/j.rpth.2026.104913"

Clinical • HEOR • Real-world • Real-world evidence • Hematological Disorders • Hemophilia • Hemophilia B • Pediatrics • Rare Diseases

MATCHING-ADJUSTED INDIRECT COMPARISON OF RIX-FP VERSUS MARSTACIMAB FOR THE PROPHYLAXIS OF SEVERE HEMOPHILIA B (EHA 2026) - "Results were consistent in the secondary analysis. This suggests rIX-FP may be associated with reduced bleeding compared to marstacimab in patients with severe hem B."

Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Optimizing Perioperative Management of Haemophilia B With rFIX-FP: Pharmacokinetic Validation of the Hemoptidose Tool. (PubMed, Haemophilia) - "The population PK model retains valid predictive performance in surgical settings. Two to three FIX measurements enable reliable individualization. The substantial discrepancy between empirical and model-guided dosing highlights major optimization potential. Hemoptidose provides accessible MIPD implementation for perioperative rFIX-FP management."

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

A decade of Real-world Evidence with IDELVION® (ISTH 2026) - "Sponsored by CSL"

Clinical • HEOR • Real-world • Real-world evidence

A Decade of IDELVION®: From Evidence to Confidence (ISTH 2026) - "Sponsored by CSL"

Monitoring Extended Half-Life Factor VIII and IX Concentrates: Performance in Routine Clotting-and Thrombin Generation Assays. (PubMed, Haemophilia) - "Accuracy of many routine FVIII or FIX assays is insufficient, and selection of the correct test is essential for producing reliable results. The haemostatic potential of EHL may deviate from the labelled potency according to TG results."

Journal • Review

Optimization of hemophilia B treatment via population PK modeling of rIX-FP, including a 3-week regimen. (PubMed, Front Pediatr) - "rIX-FP supports individualized prophylaxis with extended intervals up to 3 weeks in patients ≥12 years while younger children typically require more frequent dosing due to higher weight-normalized clearance. These PK-based insights can guide shared decision-making to balance protection, treatment burden, and patient preference across pediatric and adolescent age groups."

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

Cost-Savings Analysis of Fitusiran Prophylaxis: Reducing Breakthrough Bleeding Treatment Expenditure in the Kingdom of Saudi Arabia (ISPOR-EU 2025) - P3 | "For people with haemophilia (PwH) A without inhibitors, episodic treatments comprised octocog alfa, efmoroctocog alfa and rurioctocog alfa pegol for PwH A and nonacog alfa, albutrepenonacog alfa and eftrenonacog alfa for PwH B. Treatments included for PwH with inhibitors were factor VIII inhibitor bypassing activity (FEIBA) and eptacog alfa. In the KSA, fitusiran AT-DR prophylaxis may considerably reduce breakthrough bleed management costs in PwH versus CFC/BPA prophylaxis. Cost savings are predicted to be more substantial in PwH with inhibitors than in those without inhibitors."

HEOR • Hematological Disorders • Hemophilia • Rare Diseases

Cost-Savings Assessment of Fitusiran Prophylaxis in Minimizing Breakthrough Bleeding Treatment Expenses in the United Arab Emirates (ISPOR-EU 2025) - P3 | "The episodic treatments included were efmoroctocog alfa, octocog alfa and rurioctocog alfa pegol for people with haemophilia (PwH) A without inhibitors and albutrepenonacog alfa, nonacog alfa and eftrenonacog alfa for PwH B without inhibitors...A scenario analysis examined the impact of vial sharing. Among PwH without inhibitors, fitusiran AT-DR enabled per-bleed savings ranging from UAE Dirham (AED) 4,625 (efmoroctocog alfa) to AED 11,521 (rurioctocog alfa pegol) in PwH A and from AED 8,935 (nonacog alfa) to AED 30,053 (albutrepenonacog alfa) in PwH B. In PwH with inhibitors, fitusiran AT-DR usage generated per-bleed savings of AED 71,846 (FEIBA) to AED 90,761 (eptacog alfa). In the UAE, fitusiran AT-DR prophylaxis may considerably reduce costs associated with episodic treatments for breakthrough bleeds in PwH compared with CFC/BPA prophylaxis. PwH with inhibitors might have larger cost savings than those without inhibitors."

HEOR • Hematological Disorders • Hemophilia • Rare Diseases

Safety, Efficacy and Treatment Patterns of rIX-FP in Previously Untreated Paediatric Haemophilia B Patients: A Retrospective Chart Review in Japan. (PubMed, Haemophilia) - "This study provides real-world insights into rIX-FP use in paediatric PUPs, demonstrating its favourable safety, effective prophylaxis, and lower IVR in infants."

Journal • Retrospective data • Hematological Disorders • Hemophilia • Hemophilia B • Pediatrics • 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

Real-World Prophylaxis Outcomes with rIX-FP and rFIXFc for Males with Hemophilia B: Pooled Analysis of Medical Chart Data from Germany and Italy. (PubMed, Adv Ther) - "rIX-FP prophylaxis was associated with reduced FIX consumption versus rFIXFc and offered equally effective or potentially improved bleed protection. Additionally, PwHB who switched to rIX-FP achieved significant decreases in FIX consumption, ABR, and AjBR compared with their prior FIX product."

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

Relationship between Dose, Factor IX Activity Levels and Bleeding Probability for rIX-FP Prophylaxis in Hemophilia B: A Repeated Time-to-Event Analysis. (PubMed, Clin Pharmacol Ther) - "Variability in bleeding hazard between individuals with similar FIX activity levels was substantial (182%). Simulations showed that targeting trough FIX activity levels to 20 IU/dL resulted in a median annual bleeding rate (ABR) of zero (range: 0-3)."

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

Performance of a Drug-Specific Calibration Curve for Monitoring Treatment With Albutrepenonacog Alfa: A Multicenter Study in Argentina. (PubMed, Int J Lab Hematol) - "Idelvion-specific calibration curve showed better performance and lower CV rates independently of the aPTT reagent or the platform used. Calibration using this specific standard might allow more laboratories to obtain acceptable FIX values when processing NC and LC levels and patients' plasmas."

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

Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Chinese Subjects With Hemophilia B Previously Treated With FIX Therapy (clinicaltrials.gov) - P3 | N=23 | Active, not recruiting | Sponsor: CSL Behring | Recruiting ➔ Active, not recruiting

Enrollment closed • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Surgical use of rIX-FP in patients with haemophilia B: French real-world data (EAHAD 2025) - No abstract available

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

Long-term, real-world use of rIX-FP in adult patients with haemophilia B in Italy: outcomes of more than 4 years of retrospective-prospective follow-up (IDEAL Part A+B) (EAHAD 2025) - No abstract available

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

An update on rIX-FP prophylaxis use in paediatric patients with haemophilia B: French real-world data (EAHAD 2025) - No abstract available

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