Eloctate (efraloctocog alfa) / UCB, Sanofi, SOBI - LARVOL DELTA

A-MORE: A 48-Month Study to Evaluate Long-Term Effectiveness of Elocta on Joint Health (clinicaltrials.gov) - P=N/A | N=427 | Completed | Sponsor: Swedish Orphan Biovitrum | Active, not recruiting ➔ Completed

Trial completion • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Evaluating Costs and Efficacy of rFVIII Prophylaxis Using Matching-Adjusted Indirect Comparisons in Hemophilia A. (PubMed, TH Open) - "Methods Matching-adjusted indirect comparisons (MAICs) were performed to compare simoctocog alfa with efanesoctocog alfa (ALTUVIIIO) and turoctocog alfa pegol (Esperoct), and results were integrated with a previously published MAIC comparing simoctocog alfa with efmoroctocog alfa (ELOCTATE), damoctocog alfa pegol (JIVI), and rurioctocog alfa pegol (ADYNOVATE). In comparison with efanesoctocog alfa (Group A), no significant differences were observed in the proportion of individuals with zero bleeds, whereas treated total annualized bleeding rate (ABR) and treated spontaneous ABR favored efanesoctocog alfa. Conclusion This indirect comparison suggests that personalized prophylaxis with simoctocog alfa may offer economic advantages versus EHL rFVIII products in individuals with severe hemophilia A. Clinical outcomes were broadly comparable across comparators, although treated total and spontaneous ABRs were significantly lower with efanesoctocog alfa (Group A)."

Journal • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Prevalence of Inhibitor Development in Hemophilia A Patients Receiving Coageight: A Multicenter Cross-sectional Observational Study (clinicaltrials.gov) - P=N/A | N=262 | Not yet recruiting | Sponsor: AryoGen Pharmed Co.

New trial • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Post Hoc Analyses of A-SURE and PREVENT Confirm the Effectiveness of rFVIIIFc Prophylaxis Across All Ages, BMIs, Severities, and Inhibitor Histories. (PubMed, TH Open) - P | "Injection frequencies ( n injections/week) were comparable across subgroups (range of medians, 2.0-2.3), and factor consumption was generally similar (range of medians, 68.3-100.0 international units/kg/week), although with slightly higher factor consumption in pediatric PwHA and lower consumption in the overweight or obese BMI groups. Conclusion These post hoc analyses support the effectiveness and, therefore, use of rFVIIIFc prophylaxis in PwHA across all ages, BMI categories, severities, and for those with a history of inhibitors."

Journal • Retrospective data • Hematological Disorders • Hemophilia • Hemophilia A • Obesity • Pediatrics • Rare Diseases

Extended Half-Life Recombinant Factor VIII Conjugated with Modifying Substances Does Not Affect Fibrin Clot Formation or Stability in Haemophilia A Blood Samples. (PubMed, Haemophilia) - "People with haemophilia A require treatment with factor VIII (FVIII) to prevent or control bleeding. Some FVIII products are designed to remain active in the body for a longer time, which can reduce the number of injections needed. This extended half-life is achieved by chemically or biologically modifying FVIII, for example by attaching polyethylene glycol (PEG) or the Fc portion of immunoglobulin G. These treatments are known as extended half-life FVIII (EHL-FVIII) products. However, it has not been fully established whether these modifications affect how blood clots form and remain stable. In this study, we compared three EHL-FVIII products with two standard FVIII products using FVIII-deficient blood and plasma. We evaluated clot formation and stability using several laboratory techniques, including whole-blood assays and scanning electron microscopy. We also examined potential differences in anticoagulant and fibrinolytic properties. At comparable FVIII activity..."

Journal • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases • Thrombosis

Real World Experience of Efanesoctocog Alfa in Irish Children with Severe Haemophilia A and High Intensity Sporting Activities (ISTH 2026) - "Five children switched from emicizumab, five switched from elocta and two were on both emicizumab and elocta. Conclusions Efanesoctocog alfa offers effective protection with good trough levels for older children with severe haemophilia A and high intensity sporting activities. DOI*10.1016/j.rpth.2026.105037"

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

Spontaneous Antopol-Goldman Lesion in Severe Hemophilia A: Successful Resolution Followed by Emicizumab Prophylaxis (ISTH 2026) - "The most frequent causes are trauma, NSAIDs, anticoagulants, chemotherapy, amyloidosis, and exceptionally, bleeding diatheses, including Hemophilia A. Aims To describe a suburothelial hemorrhage or Antopol-Goldman lesion episode in a patient with severe Hemophilia A (basal FVIII 0.2%) on prophylaxis with Efmoroctocog alfa 5000 UI every 5 days but null adherence, including management and clinical evolution. Abdominal MRI; coronal view. DOI*10.1016/j.rpth.2026.106559"

Amyloidosis • Hematological Disorders • Hemophilia • Hemophilia A • Inflammation • Mood Disorders • Rare Diseases • Renal Calculi

Real-World Assessment of rVIII-SingleChain for Prophylactic Treatment in People With Severe Hemophilia A in High-Resource Settings. (PubMed, Haemophilia) - "rVIII-SingleChain prophylactic treatment demonstrated effective bleeding control in PwSHA in Taiwan, with low dosing frequency (≤2 times/week) in most PwSHA."

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

Interim analysis of the A-MORE real-world study: 4-year treatment outcomes with a recombinant factor VIII Fc in the overall and non-severe populations (ISTH 2026) - P | "Conclusions The fifth interim analysis demonstrates that rFVIIIFc prophylaxis provides sustained protection against bleeding and supports maintenance of joint status for PwHA in the overall and non-severe populations of A-MORE (sponsored by Sobi). Table or Figure Upload (1) Table 1: Baseline characteristics Page 2 Table or Figure Upload (2) Table 2: Outcomes across the 48-month observation period a Page 3 DOI*10.1016/j.rpth.2026.105943"

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

AFSTYLA® (rVIII-SingleChain) demonstrates high adherence, effective bleed control,and superior weight-adjusted cost efficiency in prophylaxis for hemophilia A in realworldclinical practice (ISTH 2026) - "Methods A retrospective analysis was conducted in 29 patients with moderate or severe hemophilia A receiving prophylactic treatment with AFSTYLA, ADYNOVI, ELOCTA, ESPEROCT, REFACTO, or NUWIQ. Conclusions In real-world clinical practice, AFSTYLA® shows excellent adherence, effective bleed control, and the best weight-adjusted cost–efficiency profile among the evaluated FVIII products. These findings support AFSTYLA as a solid, effective, and sustainable long-term prophylactic option for patients with hemophilia A. Table or Figure Upload (1) No Table or Figure Upload (2) No DOI*10.1016/j.rpth.2026.105746"

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

Time in Hemostatic Range (TIHR) Provides Harmonized Regimen-Level Assessment of FVIII Prophylaxis in Real-World Hemophilia A cohort (ISTH 2026) - "Methods Retrospective single-center analysis of 15 hemophilia A patients (11 severe, 4 moderate) receiving prophylaxis with seven FVIII products: efanesoctocog (n=6), simoctocog (n=3), efmoroctocog (n=2), Turoctocog (n=1), rurioctocog (n=1), fandhi (n=1), and octanate (n=1). AI-assisted transparent workflow demonstrates reproducible pharmacometric methodology. Table or Figure Upload (1) Table 1 Page 2 Table or Figure Upload (2) Table 2 Page 3 DOI*10.1016/j.rpth.2026.105835"

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

Evaluation of the effect of direct oral anticoagulants on thrombin generation in haemophilia A (ISTH 2026) - "Methods FVIII deficient plasma was spiked with 0 to 100IU/dL efmoroctocog alfa: concentrations: 0 to 100IU/dL). Table or Figure Upload (1) Results of TGT for FVIII spiked samples at peak and trough apixaban and rivaroxaban levels. NR=not reported Page 2 DOI*10.1016/j.rpth.2026.106024"

Cardiovascular • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Real-World Experience with Efanesoctocog Alfa in Hemophilia A. (ISTH 2026) - "All patients were previously on secondary prophylaxis: 9 with efmoroctocog alfa, 1 with turoctocog alfa, and 1 with plasma-derived FVIII/vWF concentrate. These real-world data suggest that efanesoctocog alfa may improve quality of life, although larger studies are warranted to confirm these findingswit. DOI*10.1016/j.rpth.2026.104919"

Clinical • Real-world • Real-world evidence • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases • Renal Calculi

Comparison of Coagulation and Chromogeic Methods in Pharmacokinetics with Extended Half-life Factor VIII (ISTH 2026) - "Chromogenic substrate assays (CSA) with Coamatic FactorVIII Results A sufficient number to compare results between methods was reached only for the pharmacokinetics with 4 concentrates: 3 FVIII-EHL (Efmoroctocog Alfa (Elocta) - Turoctocog Alfa (Esperoct) - Damoctocog Alfa Pegol (Jivi), all B domain deleted) and a single-chain FVIII (Lonoctocog Alfa (Afstyla). Table or Figure Upload (2) Figure2. DOI*10.1016/j.rpth.2026.104886"

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

Optimization of pre analytical heat treatment in samples containing Extended or Ultra-Long Half-Life Factor VIII concentrate (ISTH 2026) - "Extended half-life (EHL) FVIII: efmoroctocog alfa and ultra-long half-life (ULHL) FVIII: efanesoctocog alfa were spiked into FVIII deficient plasma at serial dilutions. Post-treatment patient samples containing SHL: octocog alfa, EHL-FVIII or ULHL-FVIII were evaluated...Although, no differences were seen using a heat-block or water-bath for SHL-FVIII, longer PHT times were needed for the heat-block for EHL & ULHL-FVIII concentrates. Laboratories should verify PHT conditions based on FVIII concentrates used in clinical care prior to inhibitor testing DOI*10.1016/j.rpth.2026.104341"

Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases

Cost comparison of efanesoctocog alfa vs conventional factor VIII therapies for major surgeries in patients with severe hemophilia A. (PubMed, Blood Vessel Thromb Hemost) - "The median total factor consumption per major surgery involving octocog alfa (SHL), rurioctocog alfa pegol (EHL), efmoroctocog alfa (EHL), and efanesoctocog alfa was 910, 629, 493, and 163 IU/kg, respectively. Efanesoctocog alfa resulted in $81 022 to $38 947 savings vs SHL/EHL therapies per surgery. The perioperative management with efanesoctocog alfa was estimated to be markedly more economical than that with SHL and EHL therapies due to its high-sustained factor activity."

HEOR • Journal • Reimbursement • US reimbursement • Hematological Disorders • Hemophilia • Hemophilia A • 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

POCUS: Hemostatic Potential and Joint Health in Patients With Severe Hemophilia A on Novel Replacement Therapies (clinicaltrials.gov) - P4 | N=0 | Withdrawn | Sponsor: University of Texas Southwestern Medical Center | N=28 ➔ 0 | Recruiting ➔ Withdrawn

Enrollment change • Trial withdrawal • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

In Vitro Stability Study Supports the Use of Efmoroctocog Alfa and Rurioctocog Alfa Pegol for Continuous Infusion in Hemophilia A. (PubMed, Semin Thromb Hemost) - "Efmoroctocog alfa and rurioctocog alfa pegol demonstrated stability for 96 hours. This suggests that CI would be feasible for these EHL products."

Journal • Preclinical • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Analysis of Soluble and Humoral Immunological Parameters During Re-exposure with rFVIIIFc after History of Inhibitors. (PubMed, Hamostaseologie) - "Additionally, PMBCs showed an elevated production of IL-23 and TNF-α when stimulated with FVIII.A prospective analysis of the described parameters might help to identify the onset of an immune response. Even though the number of analyzed patients is low, the detailed analyses may help to further understand the development of tolerance to FVIII and to define starting points for further interventions against treatment complications through inhibitor development in HA."

Journal • Hematological Disorders • Hemophilia • Hemophilia A • Immunology • Inflammatory Arthritis • Rare Diseases • CD4 • IL23A • TNFA

Real-world treatment patterns and outcomes of switching to efanesoctocog alfa in children with severe and moderate haemophilia a: A single-centre experience from the United Arab Emirates (ASH 2025) - "Switching from efmoroctocog alfa to once weekly efanesoctocog alfa prophylaxis demonstrated highly effective bleeding prevention and a favourable safety profile in paediatric patients. These findings highlight efanesoctocog alfa's potential and long-term clinical outcomes in routine paediatric haemophilia A management, warranting further exploration through larger, prospective studies."

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

Lentiviral vector transduced autologous CD34+ cells with FVIII transgene for gene therapy of Hemophilia A with history of inhibitors (ASH 2025) - "He was then started on immune toleranceinduction (ITI) with 25 IU/kg of extended half-life FVIII (Eloctate®) three times a week in December, 2022.For a trauma related oral bleeding in January, 2023, he was given one dose of emicizumab 3mg/kg withwhich his bleeding stopped within 24 hours...The conditioning protocol for the HSCT consisted ofmyeloablative doses of treosulfan along with fludarabine followed by infusion of the transduced CD34+gene therapy product cryo-preserved from November, 2022...This case demonstrates the first successful sustained expression of measurable FVIII activity in plasmaafter gene therapy for hemophilia A with history of inhibitors which had resolved after immune toleranceinduction. Gene therapy with LV transduced autologous CD34+ cells technology holds promise forhemophilia A patients with inhibitors and needs to be further explored."

Gene therapy • Viral vector • Bone Marrow Transplantation • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia A • Hypotension • Infectious Disease • Inflammation • Mood Disorders • Mucositis • Neutropenia • Rare Diseases • Respiratory Diseases • Septic Shock • Tuberculosis • CD34

Real-world factor VIII trough levels of severe-type people with Hemophilia A (PwHA) on efanesoctocog alfa prophylaxis: Interpersonal variation, clinical correlates and predictors (ASH 2025) - "Introduction:Efanesoctocog alfa (EA) is a high-sustained FVIII, designed to decouple FVIII from endogenous vonWillebrand factor (VWF). In our study, there were apparent interpersonal variation in trough levels of PwHA on EA prophylaxis inreal-world setting. The trough level by OSA with PTT-A might be over-estimated and strongly correlatedwith that by OSA with Actin FSL. Pre-switching rFVIIIFc trough level was significantly correlated to post-switch EA trough level."

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

Patient characteristics, treatment patterns, and bleeding in people with Hemophilia A without inhibitors initiating efanesoctocog alfa in the US: An administrative claims analysis (ASH 2025) - "The mean (SD) Charlson Comorbidity Index score among all patients was 0.49 (1.28).Within the 180-day pre-index period, 81.3% of patients received any prophylaxis (FVIII or emicizumab),5% used on-demand FVIII therapy only, and 13.7% patients were not treated with FVIII or emicizumab.The most commonly used products as on-demand or prophylactic prior to index were efmoroctocog alfa(30.0%), octocog alfa (17.5%), emicizumab (15.0%), and rurioctocog alfa pegol (11.3%). This retrospective claims analysis showed that bleed rates among PwHA receiving Efa werelow, consistent with outcomes observed in clinical trials. Most patients transitioning to Efa werepreviously treated with EHLs. Majority of patients continued treatment during the follow-up period, withrelatively low switching or discontinuation of treatment."

Clinical • Cardiovascular • Genetic Disorders • Hematological Disorders • Hemophilia • Hemophilia A • Hypertension • Immunology • Obesity • 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