Alprolix (eftrenonacog alfa) / SOBI, Sanofi - LARVOL DELTA

Women and girls with haemophilia: Australian registration rates in an international context (ISTH 2026) - "A total of seven females with moderate or severe haemophilia received prophylaxis with either emicizumab for haemophilia A or eftrenonacog alfa for haemophilia B. There were five cases with a history of an inhibitor. These findings provide important foundational data, serving both as a surrogate for prevalence and to inform targeted strategies to improve identification, classification and registration of WGWH. Table or Figure Upload (1) Page 2 DOI*10.1016/j.rpth.2026.104343"

Clinical • Hematological Disorders • Hemophilia • Hemophilia A • 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

AI-Assisted TIHR Framework with Baseline-Adjusted Recovery for Individualized Hemophilia B Prophylaxis: A Transparent Proof-of-Concept (ISTH 2026) - "Methods Retrospective analysis of nine HB patients (2 severe, 7 moderate) receiving EHL-FIX prophylaxis (albutrepenonacog, eftrenonacog, nonacog beta; n=3 each) with exact trough sampling and complete clinical data...Findings are exploratory, warranting external validation. Table or Figure Upload (1) Table 1 Page 2 Table or Figure Upload (2) Table 2 Page 3 DOI*10.1016/j.rpth.2026.105849"

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

Multidisciplinary care pathway in a girl with two X-linked disorders: severe hemophilia B revealing Turner syndrome (ISTH 2026) - "Treatment with intravenous eftrenonacog alfa was initiated, followed by secondary prophylaxis...The cumulative burden of comorbidities and long-term therapies may significantly impact quality of life and affect self-esteem, emphasizing the need for comprehensive and continuous follow-up. DOI*10.1016/j.rpth.2026.104970"

Cardiovascular • Endocrine Disorders • Genetic Disorders • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases • Turners Syndrome

Final Surgery Data from B-MORE: A 24-Month, Prospective, Non-Interventional Study of the Real-World Effectiveness and Usage of rFIXFc in Haemophilia B (ISTH 2026) - P | "Funding: Sobi. Table or Figure Upload (1) Page 2 DOI*10.1016/j.rpth.2026.104856"

Clinical • Observational data • Real-world • Real-world effectiveness • Real-world evidence • Surgery • Hematological Disorders • Hemophilia • Hemophilia B • Orthopedics • Rare Diseases

EARLY IMPACT OF GENE THERAPY (GT) ON JOINT HEALTH IN HEMOPHILIA B: AN N-OF-1 STUDY WITH COUNTERFACTUAL MODELING (ISTH 2026) - "GT with etranacogene dezaparvovec (ED) achieves sustained FIX expression and has demonstrated significant bleeding reduction in 5-year HOPE-B trial...Methods N-of-1 observational study in patient with established arthropathy (baseline HEAD-US=7), and prior prophylaxis with eftrenonacog alfa 60 IU/kg weekly (FIX trough 4–5%)...Bayesian modeling supports that GT could modify natural history of joint disease, not only bleeding. Table or Figure Upload (1) Figure 1 Page 2 Table or Figure Upload (2) Table 1 DOI*10.1016/j.rpth.2026.104792"

Gene therapy • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia B • Musculoskeletal Diseases • Rare Diseases

GENE THERAPY IN 1ST INFUSED SPANISH HEMOPHILIA B PATIENT: COMPARATIVE ANALYSIS OF CLINICAL EFFICACY AND COST-EFFECTIVENESS (ISTH 2026) - "Background Extended half-life FIX (EHL-FIX) such as rFIXFc enable prolonged dosing intervals (7-14 days), improving adherence and quality of life...Long-term follow-up essential for durability validation. Table or Figure Upload (1) Figure 1 Page 2 Table or Figure Upload (2) Table 1 DOI*10.1016/j.rpth.2026.104790"

Clinical • Cost effectiveness • Gene therapy • HEOR • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

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

Etranacogene dezaparvovec in haemophilia b: Report of 1ST patient receiving HEMGENIX® in Spain (ASH 2025) - "RESULTS A 41-year-old male with moderately severe HB (baseline FIX 1.8%) was on prophylaxis with eftrenonacog alfa (50 IU/kg weekly)...In our case, the patient's evolution follows the expected pattern: good initial response, mild and reversible liver enzyme elevation with use of prednisone, and satisfactory FIX levels. Immunosuppression is being adapted to clinical and laboratory evolution. We expect to present updated 6-month data at the ASH 2025 Congress."

Clinical • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia B • Hepatitis C • Infectious Disease • Rare Diseases • Rheumatology

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

Assessing factor consumption and bleeding outcomes of prophylaxis with 3 commonly prescribed factor IX products for hemophilia B: A retrospective patient medical record analysis in the United States. (PubMed, J Manag Care Spec Pharm) - "Mean ABR was also significantly reduced after switching to rIX-FP (3.2 vs 1.7; P = 0.0009). In this retrospective study, rIX-FP prophylaxis was associated with lower FIX consumption and potentially improved protection against bleeds compared with prophylactic treatment with rFIXFc and rFIX."

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

Investigating the Impact of FIX Fc Fusion on Its Extravascular Distribution and Hemostatic Properties (ASH 2024) - "A single dose of 50 IU/kg of recombinant FIX Fc fusion (rFIX-Fc; Alprolix) was administered retro-orbitally to each mouse...In sum, our data demonstrates the localization of rFIX-Fc in seemingly identical spatial regions as Col IV, and this appears to be the result of a specific interaction between these two proteins as confirmed in vitro in our binding assay. Overall, this suggests that the extension of FIX half-life via Fc-fusion technology does not impair its extravascular distribution and Col IV binding."

Hematological Disorders • Hemophilia • Hemophilia B • Musculoskeletal Diseases • Rare Diseases

HAEMOPHILIA B, CHRONIC KIDNEY DISEASE MANAGEMENT AND ACUTE ISCHAEMIC STROKE (WSC 2025) - "She had alprolix (factor IX) administration before ILR implantation. Roxadustat was exchanged for darbepoetin to lower thrombotic risk, and aspirin 300mg was changed to clopidogrel 75mg on discharge. The patient recovered well and experienced no clinically significant haemorrhage. The patient recovered well and experienced no clinically significant haemorrhage. This case required a cautious balance between the risks of haemorrhage and thrombosis. It highlights the complexities associated with both treatment and workup of stroke in patients with bleeding disorders."

Anemia • Ataxia • Atrial Fibrillation • Cardiovascular • Chronic Kidney Disease • Diabetes • Hematological Disorders • Hemophilia • Hemophilia B • Hypertension • Ischemic stroke • Metabolic Disorders • Movement Disorders • Nephrology • Rare Diseases • Renal Disease • Thrombosis • Type 1 Diabetes Mellitus

Orthopaedic Surgery Outcomes in Patients With Haemophilia A or B Treated With Extended Half-Life Recombinant Factor VIII and IX Fc Fusion Proteins: A Multicentre Prospective Study. (PubMed, Haemophilia) - "RFVIIIFc and rFIXFc provide effective haemostasis during orthopaedic surgery in patients with haemophilia A and B, with a favourable safety profile. Further multicentre studies are warranted to confirm these results and refine perioperative management guidelines."

Journal • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Musculoskeletal Diseases • Orthopedics • Rare Diseases • Rheumatology

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

BIOPAL: Dosage and PD Study of Eftrenonacog-alfa (clinicaltrials.gov) - P=N/A | N=15 | Completed | Sponsor: Assistance Publique - Hôpitaux de Paris | Not yet recruiting ➔ Completed

Trial completion • 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

Safety and efficacy of Fc fusion factor VIII and IX in major orthopedic surgery (ISTH 2025) - "Conclusion(s) rFVIIIFc and rFIXFc provide effective hemostasis during orthopedic surgery in hemophilia A and B, with a favorable safety profile...Tranexamic acid was administered in 93.3% (n=14) of cases for a median duration of 3 days (range:2–8)...Median hospital stay was 7 days (range:3–10). Table or Figure Upload"

Clinical • Surgery • Anemia • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Orthopedics • Rare Diseases

Real-World Study of Eftrenonacog alfa use in Hemophilia B at 8 Hemophilia Centers in Spain (ISTH 2025) - "The results are presented in the following table. Table or Figure Upload"

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

Real-World Effectiveness and Usage of Recombinant Factor IX Fc: Final Data from the B-MORE Study (ISTH 2025) - P | "An intrapatient comparison was conducted in 92 patients switching from prior standard half-life (SHL) FIX PPX to rFIXFc PPX (mean [range] duration: 11.5 [6.4–12.0] vs 40.6 [9.8–83.3] months). In these patients, median (IQR) change in ABR was 0.0 (-1.0–0.4), change in IF was -52.7 (-56.6– -38.4), and change in FC was -1,379.9 (-2,594.4– -264.3; n=91) IU/kg/year.Overall, most physicians (88.5%; 69/78) and patients (90.0%; 90/100) were satisfied/highly satisfied with rFIXFc PPX, at last assessment.No inhibitor development or serious adverse events related to rFIXFc were reported."

Clinical • Real-world • Real-world effectiveness • Real-world evidence • Hematological Disorders • Hemophilia • Hemophilia B • 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

Press Release: Q4 sales growth of 10.3%, 2024 business EPS guidance exceeded, and strong business EPS rebound expected in 2025 (GlobeNewswire) - "Lantus sales were €439 million and increased by 63.4%. US sales were €193 million...Toujeo sales were €290 million and increased by 6.5%, mainly driven by Europe (+10.1%) and Rest of World (+7.4%) where Toujeo continued to increase its basal insulin market share...Alprolix sales were €169 million and increased by 19.0%, benefiting from an increase in US market share and supply sales to Sobi. Myozyme/Lumizyme sales were €132 million and decreased by 17.0% due to the aforesaid shift to exviazyme/Nexviadyme. Thymoglobulin sales were €125 million and increased by 15.2%, reflecting increased sales in the US and in Rest of World.Praluent sales were €110 million and decreased by 6.8%, reflecting lower sales in Rest of World, specifically in China."

Sales • Aplastic Anemia • Familial Hypercholesterolemia • Hemophilia A • Hemophilia B • Pompe Disease • Type 2 Diabetes Mellitus