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

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

[Translated article] Pharmacokinetic-guided switching from standard half-life factor VIII to extended half-life pegylated factor VIII in haemophilia A therapy in clinical practice. (PubMed, Farm Hosp) - "The pharmacokinetically guided switch from standard half-life FVIII to pegylated FVIII demonstrated significant clinical benefits with reduced bleeding rates and improvements in joint health. Additionally, improvements in pharmacokinetic parameters were observed, allowing for reduced treatment burden by decreasing administration frequency, as well as lower consumption and costs."

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

Cost-Efficacy Analysis of Rurioctocog Alfa Pegol (PROPEL) vs. Efanesoctocog in Haemophilia A using Artificial Intelligence (AI) (EAHAD 2025) - No abstract available

Clinical • HEOR • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Effectiveness of PK-Guided Personalized Recombinant FVIII Treatment in Patients with Hemophilia A: Clinical Case Experiences Based on an Observational Study. (PubMed, J Blood Med) - "The results from this study in a small number of patients suggest that PK-guided regimen adjustment with myPKFiT may support optimization of the individual prophylactic administration of the FVIII products octocog alfa and rurioctocog alfa pegol. UMIN000044800."

Clinical • Journal • Observational data • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

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

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

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

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

Efanesoctocog Alfa versus Standard and Extended Half-Life Factor VIII Prophylaxis in Adolescent and Adult Patients with Haemophilia A without Inhibitors. (PubMed, Adv Ther) - P3 | "Efanesoctocog alfa was associated with significantly lower ABRs (any, spontaneous and joint) compared with EHL or SHL prophylaxis therapies. Patients had, on average, 2.2 and 3.6 fewer bleeds per year versus EHL and SHL therapies, respectively."

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

A Study of PEGylated Recombinant Factor VIII (BAX855) in Previously Untreated Young Children With Severe Hemophilia A (clinicaltrials.gov) - P3 | N=120 | Completed | Sponsor: Baxalta now part of Shire | Active, not recruiting ➔ Completed

Trial completion • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases • CD4

Pharmacokinetic-guided switching from standard half-life factor VIII to extended half-life pegylated factor VIII in the therapy of hemophilia A in clinical practice. (PubMed, Farm Hosp) - "The pharmacokinetically guided switch from standard half-life FVIII to pegylated FVIII demonstrated significant clinical benefits with reduced bleeding rates and improvements in joint health. Additionally, improvements in pharmacokinetic parameters were observed, allowing for reduced treatment burden by decreasing administration frequency, as well as lower consumption and costs."

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

Real-world insights into the management of hemophilia A in Italy: treatment patterns and healthcare resource utilization. (PubMed, Blood Res) - "These findings revealed an extensive use of EHL FVIII products, suggesting growing efforts from clinicians to optimize prophylactic strategies and achieve better bleeding protection."

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

A Study of Adynovate in Previously Treated Chinese Teenagers and Adults With Severe Hemophilia A (clinicaltrials.gov) - P3 | N=37 | Completed | Sponsor: Takeda | Active, not recruiting ➔ Completed

Trial completion • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases • CD4

Evaluation of FVIII pharmacokinetic profiles in Korean hemophilia A patients assessed with myPKFiT: a retrospective chart review. (PubMed, Blood Res) - "This study confirmed significant interpatient variation in the PK of FVIII among Korean patients with hemophilia A. To achieve optimized prophylaxis, personalizing the regimen based on the PK profile of each individual patient is essential."

Journal • PK/PD data • Retrospective data • Review • Hematological Disorders • Hemophilia • Rare Diseases

Damoctocog Alfa Pegol, a PEGylated B-domain Deleted Recombinant Extended Half-life Factor VIII for the Treatment of Hemophilia A: A Product Review. (PubMed, Drugs R D) - P1 | "The PK of damoctocog alfa pegol was shown to be improved as compared with that of sucrose-formulated rFVIII (rFVIII-FS, Kogenate®), and was also demonstrated to be non-inferior to and, for some variables, more favorable than rFVIII-Fc fusion protein, efmoroctocog alfa (Elocta®; NCT03364998), rurioctocog alfa pegol (BAX 855, Adynovate®/Adynovi®; NCT04015492), and antihemophilic factor (recombinant) plasma/albumin-free method (rAHF-PFM, Advate®; NCT02483208). Efficacy for perioperative hemostasis has been demonstrated. Low bleeding rates were achieved across the studies, with twice weekly, every 5-day and every 7-day prophylaxis offering patients ≥ 12 years and their clinicians the chance to tailor treatment to individual needs and lifestyles, while maintaining long-term protection from bleeds and their consequences."

Journal • Review • Hematological Disorders • Hemophilia • Rare Diseases

INDIVIDUAL PHARMACOKINETIC EVALUATION OF FIXED-SEQUENCE SINGLE-DOSE OCTOCOG ALFA, RURIOCTOCOG ALFA PEGOL, AND EFANESOCTOCOG ALFA IN ADULTS WITH SEVERE HEMOPHILIA A (EHA 2024) - P1 | "Consistent superiority regarding time over 40% FVIII activity, half-life, clearance, and FVIII exposure wereobserved with efanesoctocog alfa compared with SHL and EHL FVIII in all patients. No serious TEAEs orinhibitor development were reported for efanesoctocog alfa. Study funded by Sanofi and Sobi."

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

COMPARATIVE EVALUATION OF QUALITY OF LIFE IN HEMOPHILIA A PATIENTS UNDER PROPHYLAXIS: A 4-YEAR PERSPECTIVE (EHA 2024) - "Consequently, 20 patients previously under prophylaxis with standard half-life (SHL) products (INN-octocog alfa, octocog alfa, moroctocog alfa), transitioned to prophylactic treatment utilizing extended half-life(EHL) products (rurioctocog alfa pegol, efmoroctocog alfa, damoctocog alfa pegol), while 4 remained on SHL. A comprehensive analysis of 4 years' worth of data reveals a significant improvement across all quality-of-lifedimensions, underscoring the imperative for improved and personalized prophylactic interventions to achieveoptimal efficacy. Tailoring treatment regimens to align with the emotional and physical well-being of patientsholds promise for the implementation of more successful clinical approaches."

Clinical • HEOR • CNS Disorders • Depression • Hematological Disorders • Hemophilia • Pain • Psychiatry • Rare Diseases • Sexual Disorders

Effect of extended half-life products and emicizumab on routine coagulation and thrombin generation tests (ISTH 2024) - "Except for SynthaSIL reagent, all Adynovi measurements from mid to high range (10-150%) overestimated FVIII activity. In the low range all reagents overestimated FVIII activity with more than 20% except for the BioPhen FVIII and CK Prest reagent. For Elocta, only the SynthaSIL reagent measured FVIII activity accurately in the mid to high range."

Hematological Disorders • Hemophilia • Rare Diseases

Real-world Utilization of Extended Half-life Factor VIIIs in the Treatment of Hemophilia A: A Retrospective Single Center Study in Korea (ISTH 2024) - "Rurioctocog alfa pegol was used in 16(42.1%), Efmoroctocog alfa in 22 patients(57.9%). 25 patients(65.8%) were under 19 years old at the time of EHL use. Two patients started each drug as an additional choice to control bleeding, combined with emicizumab or another clinical trial drug."

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

Factor VIII is a regulator of angiogenesis and a promoter of endothelial barrier stability (ISTH 2024) - "HA BOECs were treated in vitro with simoctocog alfa, efmoroctocog alfa, rurioctocog alfa pegol, damoctocog alfa pegol, or octocog alfa. Impaired tubulogenesis, migration and permeability of HA ECs vs healthy ECs were observed. A significant enhancement of EC functionality was demonstrated by treating HA BOECs with rFVIII products, with a higher positive effect for simoctocog alfa. Moreover, in NSG-HA mice treated with different rFVIII concentrates, and subsequently injected with Evans Blue dye, we showed a significant reduction of dye extravasation with a complete correction in mice treated with simoctocog alfa compared with other rFVIII products."

Hematological Disorders • Hemophilia • Rare Diseases

Investigation of genetic factors associated with the half-life of infused factor VIII in hemophilia A patients (ISTH 2024) - "We analyzed the half-life of 33 PwHA and found three patients with a longer half-life than the others. Patients 1 and 2 had a half-life of 19.5 and 25.5 hours for rurioctocog alfa, respectively, and patient 3 had a half-life of 31.75 hours for rurioctocog alfa pegol. The blood groups and VWF:Ag of each patient were type A 131%, type O 66%, and type B 187%, respectively."

Clinical • Hematological Disorders • Hemophilia • Rare Diseases • HSPG2 • LRP1 • MRC1 • MUC4 • SIGLEC5

Real-World Assessment of rVIII-SingleChain for Prophylactic Treatment in Severe Hemophilia A Patients in Taiwan (ISTH 2024) - "Our cohort encompassed 39 PwHA (38 male, 1 female), with 14 PwHA in rVIII-SingleChain group (mean observation period 198.1, SD 63.4 days) and 25 in the compared group (Efmorocotocog alfa or Rurioctocog alfa pegol, mean 345.3, SD 62.9 days). Demographics and other baseline characteristics are comparable between two groups. Of the rVIII-SingleChain group, median annualized bleed rates (ABRs), annualized spontaneous bleed rates (AsBRs), and annualized joint bleed rates (AjBR) were comparable with other rFVIII group (0.0 vs 2.0, 0.0 vs 1.0, 0.0 vs 0.0, respectively) without statistical differences."

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

Physical activity engagement and health-related quality of life in patients with hemophilia A receiving either octocog alfa or rurioctocog alfa pegol versus other FVIII replacement therapies (ISTH 2024) - "The analysis included 500 patients (Table 1). Based on the ATE, patients in the treatment cohort were 16.4% (p < 0.01) more likely to engage in sport/activity compared with control (Table 2); however, no significant difference was observed between cohorts for the prophylaxis subgroup. No significant difference in EQ-5D index scores was observed between the treatment and control cohorts in either the total or prophylaxis subgroup (Table 2)."

Clinical • HEOR • Hematological Disorders • Hemophilia • Pediatrics • Rare Diseases