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

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

Cost Comparison of Efanesoctocog Alfa with Existing Factor VIII Replacement Therapies for Major Surgeries in People with Severe Hemophilia a (ASH 2024) - P2/3, P3 | "Objective To estimate total costs associated with perioperative hemostatic management in patients with severe HA treated with SHL (octocog alfa), EHL (rurioctocog alfa pegol and efmoroctocog alfa), and high-sustained (efanesoctocog alfa) FVIII replacement therapies. This is attributed to its high-sustained factor activity and reduced factor consumption during the reported perioperative period. The major limitations of the study were : the types of major surgeries varied among studies; the perioperative period data of octocog alfa were not found."

HEOR • Reimbursement • Surgery • US reimbursement • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Role of Factor VIII As a Regulator of Angiogenesis and Promoter of Endothelial Barrier Stability (ASH 2024) - "HA BOECs were treated in vitro with the rFVIII products simoctocog alfa (Nuwiq®), efmoroctocog alfa (Elocta®), rurioctocog alfa pegol (Adynovate®), damoctocog alfa pegol (Jivi®), octocog alfa (Advate®), or emicizumab (Hemlibra®). Investigating the potential extra-coagulative role of FVIII could be crucial to understanding the key molecular targets at the cellular level which impair EC function in patients with HA. Knowledge of the possible effect of different rFVIII products and non-factor therapies on EC function can be used to optimize therapeutic approaches, which in turn may result in safer and more efficient treatment of HA."

Cerebral Hemorrhage • CNS Disorders • Hematological Disorders • Hemophilia • Hemophilia A • Osteoarthritis • Rare Diseases

Rurioctocog Alfa Pegol Shows Promise for Untreated Patients With Hemophilia A (Hematology Advisor) - P3 | N=120 | NCT02615691 | Sponsor: Baxalta now part of Shire | "Rurioctocog alfa pegol (rAHF-PEG) may offer a safe and effective therapy for previously untreated patients with severe hemophilia A, according to results presented at the International Society on Thrombosis and Haemostasis 2025 Congress...The incidence of FVIII inhibitor development among participants with at least 100 rAHF-PEG exposure days was 11%, which was lower than in other studies involving untreated patients. Of the patients who did develop inhibitors, 71% had successful immune tolerance induction treatment, indicating that rAHF-PEG may help overcome inhibitor development...Prophylactic therapy with rAHF-PEG led to lower annualized bleeding rates than on-demand use. Patients rated the rAHF-PEG efficacy as excellent or good, and bleeding episodes could be effectively treated with just 1 to 2 infusions. Adverse events in the study were generally mild or moderate and unrelated to the treatment..."

P3 data • Hemophilia A

Real-world safety and effectiveness of rurioctocog alfa pegol in 338 patients with hemophilia A in South Korea: A postmarketing surveillance study. (PubMed, Thromb Res) - P | "Rurioctocog alfa pegol was effective in children and adults, with no new safety signals; most patients on prophylaxis experienced no bleeds during the study."

Clinical • Journal • P4 data • Real-world evidence • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Elucidation of the biochemical properties of efanesoctocog alpha in the coagulation reaction (ISTH 2025) - "Aims In the present study, we examined the cause of the difference between the two methods by comparing Efa to rurioctocog alfa (Ruri). In the ELISA, the antigen level of Efa was 1.32-fold higher than that of Ruri. The level of Efa 100U/mL measured by spectrophotometer was 2.5-fold higher than that of Ruri, although this might be due to the difference in molecular weight."

Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Use of FVIII- Extended half-life (EHL) in Major Orthopedic Surgery: Real-World Experience (ISTH 2025) - "In 64% of the procedures, Damoctocog Alfa Pegol was used, followed by Rurioctocog Alfa Pegol (18%) and Turoctocog Alfa Pegol (18%). Bleeding complications occurred in only one case (bilateral hip replacement) associated with a periprocedural complication. Table or Figure Upload"

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

Impact of Extended Half-Life Factor VIII on Pharmacokinetics and Bleeding Rates in Hemophilia A (ISTH 2025) - "Patients initially treated with SHL FVIII (Beriate®, Immunate®, Octanate®, Advate®, Novoeight®, Hemophil M®, Xyntha®) and later switched to EHL FVIII (Adynovate®, Jivi®) were included. A weak, non-significant negative correlation existed between spontaneous bleeding and T1/2 for both SHL (r=-0.23, IC 95% -0.51 to 0.09, p>0.05) and EHL (r=-0.24, IC 95% -0.49 to 0.05, p>0.05). A significant positive correlation was observed between AUC and T1/2 (r=0.79, IC 95% 0.7 to 0.86, p<0.05) Table or Figure Upload"

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

Balancing the thrombotic and haemorrhagic risk of non severe haemophilia A in major surgery (ISTH 2025) - "The patient received rurioctocog-alfa-pegol preoperatively (Table 1)...The patient underwent adjuvant chemotherapy with capecitabine...We performed perioperative prophylaxis with turoctocog-alfa-pegol (Table 2)...Case 2:The postoperative course was complicated by bleeding from the surgical site on day 3, requiring a single packed red blood cell transfusion, with subsequent clinical recovery. Table or Figure Upload"

Surgery • Anemia • Atherosclerosis • Cardiovascular • Dyslipidemia • Genetic Disorders • Hematological Disorders • Hemophilia • Hemophilia A • Hepatitis C • Infectious Disease • Metabolic Disorders • Nephrology • Obesity • Oncology • Pulmonary Arterial Hypertension • Rare Diseases • Renal Disease

Advancing Hemophilia A Management with the Portability of Turoctocog Alfa Pegol (N8-GP) (ISTH 2025) - "Results Six patients (mean age: 33.7 years, range 27–43) included three with moderate and three with severe hemophilia A. Before switching to N8-GP, half were treated with rurioctocog alfa pegol, while the rest used damoctocog alfa pegol, moroctocog alfa, or plasma-derived FVIII equally. The results of the satisfaction survey are presented in Figures 1 and 2. Table or Figure Upload"

Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases • Rheumatology

Efmoroctocog alfa and Rurioctocog alfa pegol demonstrate FVIII stability during in vitro testing (ISTH 2025) - "Results The in vitro FVIII did not decrease over the 96 hours following reconstitution. The average in vitro FVIII activity for Efmoroctocog alfa was 1.03 +/- 0.17U/mL and the average in vitro FVIII for Rurioctocog alfa pegol was 1.06 +/- 0.12U/mL compared to baseline activity."

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

Use of Generative AI to analyze pegylated FVIII Concentrates (ISTH 2025) - "Methods Adynovi, Jivi and Esperoct are compared, according to data reported in EMA's technical sheets (Table 1). ChatGPT4o concludes that if the objective is to maximize cost-effectiveness efficiency under the same unit cost, Esperoct® 50 IU/Kg each 4 days is the best option followed by JIVI® at 60 IU/kg each 7 days. Table or Figure Upload"

Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Phase 3 Study of Rurioctocog Alfa Pegol in Previously Untreated Patients with Severe Hemophilia A (ISTH 2025) - No abstract available

Clinical • Late-breaking abstract • P3 data • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Role of factor VIII as a regulator of angiogenesis and promoter of endothelial barrier stability (ISTH 2025) - "HA BOECs were treated in vitro with simoctocog alfa, efmoroctocog alfa, rurioctocog alfa pegol, damoctocog alfa pegol, octocog alfa, efanesoctocog alfa or non-factor therapy, emicizumab. Furthermore, simoctocog alfa in vivo rescue was greater compared to efmoroctocog alfa or efanesoctocog alfa. These effects on ECs may be mediated by the binding and signaling of simoctocog alfa to the ECs surface."

Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Excitement builds for ISTH 2025 Congress in Washington, D.C. as late-breakthrough abstracts are announced (PRNewswire) - "The International Society on Thrombosis and Haemostasis (ISTH) prepares to welcome nearly 5,000 attendees from the global thrombosis and hemostasis community to the ISTH 2025 Congress in Washington, D.C....A key highlight of the ISTH 2025 Congress, taking place from June 21-25, 2025, is the presentation of late-breakthrough abstracts, offering new data from major clinical trials and breakthrough studies, giving attendees a first look at transformative findings."

Clinical data • Hemophilia A • Immune Thrombocytopenic Purpura • Venous Thromboembolism

Efficacy and Safety of Recombinant Factor VIII in Previously Untreated and Previously Treated Children with Hemophilia A: A Systematic Review. (PubMed, Adv Ther) - "Our analysis revealed that both octocog alfa and rurioctocog alfa pegol showed low inhibitor development, with octocog alfa having few treatment-related AEs. Regular monitoring for inhibitors during rFVIII therapy is important."

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

POCUS: Hemostatic Potential and Joint Health in Patients With Severe Hemophilia A on Novel Replacement Therapies (clinicaltrials.gov) - P4 | N=28 | Recruiting | Sponsor: University of Texas Southwestern Medical Center | Trial completion date: Jul 2025 ➔ Jul 2027 | Trial primary completion date: Jul 2025 ➔ Jul 2027

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

A Study of Adynovate in Previously Treated Chinese Teenagers and Adults With Severe Hemophilia A (clinicaltrialsregister.eu) - P3 | N=37 | Sponsor: Baxalta US Inc.

New P3 trial • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases • CD4