Adynovate (rurioctocog alfa pegol) / Nektar Therapeutics, Takeda - LARVOL DELTA

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

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 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

Repeated administration of efanestocog alfa does not result in a hypercoagulant condition in hemophilia A mice (ISTH 2026) - "Conclusions Administered efanesoctocog alfa over a short period demonstrated a comparable global coagulation potential to that of rurioctocog alfa and did not induce a hypercoagulant state. DOI*10.1016/j.rpth.2026.105730"

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

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

Expanding Surgical Boundaries in Severe Hemophilia A: Successful OrthognathicCorrection Using PK-Guided Extended Half-Life FVIII in a Middle-Income Country (ISTH 2026) - "He was on primary prophylaxis with EHL- rFVIII(rurioctocog alfa pegol) at 25 IU/kg three times weekly...This experience provides meaningful evidence supporting the safe performance of complex orthognathic surgery in this high-risk population. DOI*10.1016/j.rpth.2026.105105"

Hematological Disorders • Hemophilia • Hemophilia A • Orthopedics • Rare Diseases

Monitoring of factor VIII activity of rurioctocog alfa pegol: comparison of one-stage and chromogenic assays (ISTH 2026) - "These findings support suitability of these methods for monitoring of rurioctocog alfa pegol. Supported by Ministry of Health, Czech Republic – conceptual development of research organization (FNBr, 65269705) and Masaryk University - MUNI/A/1733/2025 Table or Figure Upload (1) Page 2 DOI*10.1016/j.rpth.2026.104897"

Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Low Inhibitor Rate and Assessment of Binding Antibodies in Previously Untreated Patients with Severe Hemophilia A Treated with Rurioctocog Alfa Pegol: Final Results from a Prospective Phase 3 Study (ISTH 2026) - P3 | "Methods Eligibility criteria encompassed severe HA (FVIII <1%), age <6 years, and <3 exposure days (EDs) to rAHF- PEG, octocog alfa, or plasma transfusion. Participants with specific binding antibodies. Page 2 DOI*10.1016/j.rpth.2026.104294"

Clinical • P3 data • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Individual Comparative PK Evaluation of Single-dose Octocog Alfa, Rurioctocog Alfa Pegol, and Efanesoctocog Alfa in Adults with Severe Hemophilia A. (PubMed, TH Open) - No abstract available

Journal • Hematological Disorders • Hemophilia • Hemophilia A • 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

Repeated short-interval administration of efanesoctocog alfa is not associated with increased global coagulation potential in hemophilia A mice. (PubMed, Int J Hematol) - "In HA mice, rotational thromboelastometry parameters, aPTT, TAT, and D-dimer were similar with efanesoctocog alfa and rurioctocog alfa, whereas FVIII:C by chromogenic assay was higher with efanesoctocog alfa than with rurioctocog alfa. In conclusion, global coagulation potential after short-interval administration of efanesoctocog alfa was similar to that after rurioctocog alfa under the experimental conditions."

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

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

Final data from the German AHEAD study: effectiveness and safety of octocog alfa and rurioctocog alfa pegol for hemophilia A in a real-world setting. (PubMed, Ther Adv Hematol) - "In patients with severe HA, octocog alfa prophylaxis was associated with reduced bleeding rates compared with on-demand therapy. Deutsches Register Klinischer Studien (German Clinical Trials Register): DRKS00000556."

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

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

Rurioctocog alfa pegol use in immune tolerance induction: An analysis of a phase 3 study in previously untreated patients with severe hemophilia A (THSNA 2026) - P3 | "Most participants in this small sample achieved tolerization (86%). Although SAE incidence was high, only one SAE was treatment related, and occurred before ITI initiation. This analysis supports the efficacy and safety of rAHF-PEG as ITI therapy for FVIII inhibitors in PUPs with severe hemophilia A. No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author."

Clinical • P3 data • Hematological Disorders • Hemophilia • Hemophilia A • Immunology • 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

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

Chronic Disease Outcomes As Predictors of Quality of Life in Patients with Hemophilia Α: Data from the Real-World AHEAD International Study (ASH 2023) - P | "The international Antihemophilic factor Hemophilia A outcome Database (AHEAD) study (NCT02078427) is an ongoing prospective, non-interventional, multicenter study evaluating the long-term safety and effectiveness of FVIII replacement in patients with HA receiving either octocog alfa or rurioctocog alfa pegol (ADVATE®; ADYNOVATE® [US] / ADYNOVI™ [Europe]; Baxalta US Inc. In this Mixed Effects Model, chronic pain level and number of PJs both had a statistically significant negative association with QoL, highlighting the importance of managing these chronic outcomes with effective treatment."

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

A Prospective, Observational, Multi-Center Study of Rurioctocog Alfa Pegol Pharmacokinetic-Guided Prophylaxis Using Mypkfit in Severe Hemophilia a Patients in Taiwan (ATTRACT-HA Study): An Interim Analysis (ASH 2023) - "ConclusionThe ATTRACT-HA study is the first prospective, multi-center, observational study currently ongoing in Taiwan to provide real-world evidence of the effectiveness of rurioctocog alfa pegol PK-guided prophylaxis using myPKFiT app in patients with severe hemophilia A. In this interim report, we described the clinical features and factors of the study participants at the beginning of this real-world study. Between the two trough levels, we observed a higher percentage of patients experiencing episodic bleeding prior to PK-guided prophylaxis and lower baseline HEAD-US scores in the <3% trough level group, which may be in part due to the younger median age in this group."

Clinical • Observational data • PK/PD data • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases • Rheumatology

Impact of Recombinant Factor VIII and Platelet Interaction on Platelet Functionality and Hemophilia a Treatment (ASH 2023) - "Simoctocog alfa demonstrated higher binding to activated platelets in vitro compared with efmoroctocog alfa, rurioctocog alfa pegol or damoctocog alfa pegol, resulting in an increased phenotype shift of platelets from the pro-aggregatory to the pro-coagulant state. The increased binding of simoctocog alfa was associated with a phenotypic shift in platelets as evidenced by increased exposure of PS on the platelet membranes. The binding of simoctocog alfa to platelets was disrupted when integrin αIIbβ3 activation was inhibited, suggesting a role of integrin αIIbβ3 signaling following binding of FVIII to platelets."

Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases • ANXA5

Impact of Variable Recombinant Factor VIII Binding on Platelet Functions (ASH 2024) - "rFVIII-platelet binding : Activated platelets were incubated with simoctocog alfa (Nuwiq®), efmoroctocog alfa (Elocta®), rurioctocog alfa pegol (Adynovate®) or damoctocog alfa pegol (Jivi®). The rFVIII products explored in this study bound to platelets with varying strength, with simoctocog alfa demonstrating the highest amount of platelet binding, as well as the highest interaction with pro-aggregatory platelets. These findings indicate that variations in platelet binding may influence the efficacy of rFVIII products in the treatment of HA."

Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases • ANXA5

Indirect Comparison of the Efficacy and Therapy-Related Costs of a Pharmacokinetic and Individualized Prophylaxis Regimen with Simoctocog Alfa Versus Other Extended-Half Life Factor VIII Concentrates (ASH 2024) - "For comparison, aggregated data was obtained from the following trials with EHL concentrates : pathfinder2 (turoctocog alfa pegol, N = 175), A-LONG (efmoroctocog alfa, N = 117), PROTECT FVIII (damoctocog alfa pegol, N = 110), PROPEL (rurioctocog alfa pegol 1–3% and 8–12%; N = 57 and 58), and XTEND-1 (efanesoctocog alfa, Group A, N = 133). Conclusion : Albeit at a generally higher weekly dose, a PK-guided, individualized prophylaxis regimen with simoctocog alfa offered comparable or significantly improved zero bleed rates and significantly lower or comparable ABRs than prophylactic regimens with EHL rFVIII concentrates. Nevertheless, the estimated annual cost of a simoctocog alfa-based regimen is 20–55% lower than with the other concentrates."

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