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

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

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

Cost-Effectiveness of Emicizumab Vs Efanesoctocog Alfa, Standard Half Life (SHL) and Other Extended Half Life (EHL) FVIII Products for Prophylaxis in People with Severe Hemophilia a without Inhibitors (ASH 2023) - "Advate and Eloctate data were used to represent SHL and EHL, respectively. Findings in this cost-effectiveness analysis suggest that emicizumab is less costly and more effective (i.e., dominant) over a lifetime compared with available FVIII prophylaxis in pediatrics and adult PwHA in the US."

Cost effectiveness • HEOR • Hematological Disorders • Hemophilia • Hemophilia A • Pediatrics • Rare Diseases

The Correlation of Muscle Mass and Walking Distance with Joint Health and Functional Status in Hemophilic Arthropathy Among People with Hemophilia a (PwHA) (ASH 2023) - " At the time when the 29 patients receiving this survey of joint health, there were 28 on rFVIIIFc treatment for an average of 31.2 months (mean: 33.5, range 19.5-43), among whom 26 severe-type patients had rFVIIIFc prophylaxis and 2 moderate-type patients had on-demand therapy with rFVIIIFc... For the first time, limbs muscle thickness and the distance of 6MWT were linked to assessment of joint health, quality of life and activity participation in PwHA. Ultrasound measurement of muscle thickness appears to be a reliable tool for the assessment of muscle mass in PwHA. The 6MWT also appears to be useful tool for assessment of physical capacity and endurance in PwHA."

Hematological Disorders • Hemophilia • Hemophilia A • Muscular Atrophy • Musculoskeletal Diseases • 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

Influencing Factors and Predictors for Bleeding Outcomes in Severe-Type People with Hemophilia a (PwHA) on Extended Half-Life rFVIII-Fc Prophylaxis (ASH 2024) - "Introduction : In the past, we had ever reported that real-world severe-type people with hemophilia A (PwHA) got improved bleeding outcomes when they shifted to extended half-life (EHL) recombinant FVIII with Fc-fusion protein (rFVIIIFc) prophylaxis...Many clinical factors, dosing regimen, and pharmacokinetic factors were proved to be unable to independently influence bleeding outcomes. Only ratio of VWF : activity/VWF : Ag was proved as a predictor for ABR."

Hematological Disorders • Hemophilia • Hemophilia A • Hepatitis C • Infectious Disease • Rare Diseases • Rheumatology

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

Factor VIII (FVIII) Trough Levels of Real-World Severe-Type People with Hemophilia a (PwHA) on rFVIII-Fc Prophylaxis: Clinical Correlates and Effects on Perceivable Bleeding Outcomes (ASH 2024) - "Conclusion : Our study showed that for real-world PwHA on rFVIIIFc prophylaxis, trough levels variated hugely, and that trough levels were significantly higher in PwHA with non-O group and longer rFVIIIFc half life, and significantly correlated to endogenous VWF levels, age, and BMI. Meanwhile, our data indicated that between PwHA with non-zero and zero bleeding, the proportion of trough <1% was not significantly different; meanwhile, trough level was not correlated to perceivable bleeding frequency, including ABR and AJBR."

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

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

Preventing Bleeds in Pediatric Patients With Hemophilia A: Which Factor Replacement Therapy Offers the Best Protection and at What Cost? (ISPOR-EU 2025) - "Second lowest number of bleeds was achieved with efanesoctocog alfa (Altuviiio/Altuvoct) with 3.90 bleeds, following Efmoroctocog alfa (Elocta), turoctocog alfa pegol (Esperoct), turoctocog alfa (NovoEight), simoctocog alfa (Nuwiq), octocog alfa (Kovaltry), Afstyla (lonoctocog alfa) and octocog alfa (Advate) with 9.80, 9.85, 9.85, 11.85, 16.85, 18.45 and 18.75 bleeds, respectively. Prevention of bleeds is of utmost importance when treating pediatric patients with hemophilia A. Choosing a treatment with the lowest possible bleeding rates can support the physical development of the patient. This analysis showed that the lowest number of bleeds and lowest costs are estimated to be reached by damoctocog alfa pegol (Jivi)."

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

Analysis of Pooled Real-World Utilization and Outcomes Data of rVIII-Single Chain Compared With Standard and Extended Half-Life FVIII Products for Prophylaxis of Hemophilia A in France, Germany, and Italy (ISPOR-EU 2025) - "Prophylaxis with rVIII-SingleChain may reduce consumption and improve bleed protection compared with SHL FVIII products, and may provide comparable protection to EHL FVIII products."

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

Cost-effectiveness of emicizumab for the treatment of hemophilia A: a systematic review. (PubMed, Front Public Health) - "In addition, rFVIIIFc and valoctocogene roxaparvovec were more cost-effective than emicizumab for people with HA without inhibitors. Cost-effectiveness analyses with more accurate cost estimations of different countries should provide more convincing evidence for clinical decision-making. Identifier CRD 42023429349, https://www.crd.york.ac.uk/PROSPERO/view/CRD42023429349."

HEOR • Journal • Review • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

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

Clinical and Surgical Experiences with Efmoroctocog Alfa in Pediatric Population with Hemophilia A. (PubMed, Clin Appl Thromb Hemost) - "P18 had refixation surgery due to a left arm radius and ulna bone fracture, and a cast was applied. All three surgical operations were performed under treatment of recombinant FVIII Efmoroctocog alfa (Ealfa/Elocta®) successfully.ConclusionEfmoroctocog alfa seems to be effective in EHL FVIII replacement therapy and surgical management of hemophilia A. Compared with standard half-life (SHL) FVIII products, EHL FVIII products such as Ealfa have the potential to optimize prophylactic outcomes by decreasing the burden of treatment or increasing the level of bleed protection."

Journal • Hematological Disorders • Hemophilia • Hemophilia A • Musculoskeletal Diseases • Orthopedics • Pediatrics • Rare Diseases

Press Release: Q2: double-digit sales and solid business EPS growth. 2025 sales guidance is now high single-digit growth, at upper end of range (GlobeNewswire) - "ALTUVIIIO (hemophilia A) sales were €291 million of which 82% were in the US. Growth was driven by continued patient switches from older plasma-derived and recombinant factor medicines and to a lesser extent from non-factor treatments. Rest of World sales of €53 million benefited from the launch in Japan and supply sales to the collaborator Sobi. The hemophilia A franchise (ALTUVIIIO and Eloctate combined) sales were €356 million and increased by 41.1%, primarily driven by ALTUVIIIO’s strong performance of €291 million, while Eloctate contributed €65 million...Rezurock (chronic graft-versus-host disease, third line) sales were €132 million and increased by 21.1%, driven by launches gaining further momentum in Europe (sales of €14 million) and in Rest of World (sales of €11 million), including in China...Cablivi (acquired thrombotic thrombocytopenic purpura) sales were €69 million...Qfitlia (hemophilia A and B) sales were €1 million..."

Sales • Chronic Graft versus Host Disease • Hemophilia • Hemophilia A • Hemophilia B • Thrombocytopenic Purpura

Switching hemophilia a patients to an extended half-life agent on a prophylactic basis: an economic appraisal. (PubMed, Expert Rev Pharmacoecon Outcomes Res) - "However, there is a statistically significant reduction in cost across treatment styles. The analysis confirms the advantages offered by prophylaxis with rFVIIIFc from an economic standpoint for the Greek healthcare system, in accordance with other studies."

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

A-SURE: intra-patient comparison of prophylactic effectiveness of a recombinant factor VIII Fc fusion protein versus standard half-life factor VIII in hemophilia A. (PubMed, Hematology) - P | "These trends were consistent across age groups. This intra-patient comparison demonstrates switching from SHL FVIII to rFVIIIFc prophylaxis reduces frequency of bleeds, injection frequency, and factor consumption, complementing previously reported data from A-SURE.Trial registration:ClinicalTrials.gov identifier: NCT02976753."

Clinical • Journal • Hematological Disorders • Hemophilia • Hemophilia A • 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

Long-term observational joint health study in patients initiating efanesoctocog alfa: baseline data (ISTH 2025) - P | "Prior prophylaxis (n=44) included efmoroctocog alfa (34%), emicizumab (24%), and octocog alfa (22%). Baseline AjBR and annualized bleed rates (ABRs) prior to starting efanesoctocog alfa are shown in Table 2; overall pre-switch ABR was >3. Table or Figure Upload"

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