Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / SOBI, Sanofi - LARVOL DELTA

Tailored prophylaxis in severe hemophilia A: Real-world experience with efanesoctocog alfa (ASH 2025) - "Conclusion These cases highlight the real-world benefits of once weekly efanesoctocog alfa prophylaxis in severe hemophilia A, including higher trough FVIII levels, reduced treatment burden, and improved clinical outcomes. Personalized, PK-guided care supported successful transitions and aligns with clinical trial data, reinforcing its role in modern hemophilia management."

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

Successful surgical management in severe hemophilia A patients using efanesoctocog alfa: A two-case experience undergoing orthopedic surgery (ASH 2025) - "Both patients underwent successful orthopedic surgeries with minimal perioperative exposure to efanesoctocog alfa. Each case required only one preoperative dose plus a limited number of postoperative infusions. The high and sustained FVIII levels achieved with relatively low factor consumption supported effective hemostasis throughout the perioperative period, allowing early discharge and rapid return to baseline prophylaxis."

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

Clinical experience with efanesoctocog alfa in patients with uncover needs on emicizumab (ASH 2025) - "In these patients, efanesoctocog alfa improved hemostatic control and joint status in individuals with HA and unmet needs under emicizumab, without safety concerns. Longer follow-up is necessary to confirm these findings with additional data."

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

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

Perioperative use of efanesoctocog alfa in Hemophilia A: A dual-center real-world study (ASH 2025) - No abstract available

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

Quality of life and functional improvements with efanesoctocog alfa in patients with moderate-to-severe Hemophilia A: A real-world survey (ASH 2025) - "The most recent treatments prescribed prior to Efa were standard half-life products (64.5%), extended half-life products (45.2%), emicizumab (32.3%), and others (6.5%) (groupsexceed 100% as multiple treatments were used either prophylactically or on-demand). This is the first real-world, patient-reported outcomes study of prophylactic Efa use amongpatients with moderate-to-severe HA. Most patients rated Efa more favorably for hemophilia controlcompared to previous regimens. Improvements were observed in daily functioning, participation inphysical activity, and a reduced need for preventive measures and additional prophylactic doses."

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

Real-world treatment patterns for Hemophilia A since the launch of emicizumab in the US (ASH 2025) - "Among PwHA receiving newer therapies in the real-world setting, switching fills betweenemicizumab and efanesoctocog alfa was infrequent (2% vs 4%). The majority of PwHA initiated andpersisted with emicizumab prophylaxis, supporting its real-world effectiveness as the standard of care."

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

Perioperative use of efanesoctocog alfa in Hemophilia A: A dual-center real-world study (ASH 2025) - "Perioperative Efanesoctocog alfa administration provided effective surgical hemostasis, whereby a singlepreoperative outpatient dose was sufficient for most procedures. These observations are encouragingsince Efanesoctocog alfa seems to not only facilitate but also alleviate outpatient management,advantageous to reduce management burden for patients and health care staff alike."

Clinical • Real-world • Real-world evidence • Cardiovascular • Hematological Disorders • Hemophilia • Hemophilia A • Orthopedics • Rare Diseases • Venous Thromboembolism

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

Clinical outcomes up to 4 years of once-weekly efanesoctocog alfa prophylaxis in previously treated adults, adolescents, and children with severe hemophilia A: Interim analysis of the Phase 3 XTEND-ed long-term extension study (ASH 2025) - P3 | "Overall, 60 (84.5%) participantsexperienced ≥1 TEAE; most commonly pyrexia (18.3%), upper respiratory tract infection (16.9%),arthralgia (15.5%), and cough (15.5%). Two participants had ≥1 treatment-related TEAE (asthma and postinfusion pain and headache); no treatment-related serious TEAEs or treatment discontinuations werereported.ConclusionResults from up to 4 years of the XTEND-ed study demonstrate that once-weekly efanesoctocog alfacontinues to be well tolerated, providing highly effective bleed protection with no inhibitor developmentin adults, adolescents, and children with severe hemophilia A."

Clinical • Clinical data • P3 data • P3 data: top line • Asthma • CNS Disorders • Cough • Hematological Disorders • Hemophilia • Hemophilia A • Immunology • Infectious Disease • Influenza • Musculoskeletal Pain • Novel Coronavirus Disease • Rare Diseases • Respiratory 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

Real-world experience of efanesoctocog alfa in Hemophilia A patients in the US: A retrospective analysis (ASH 2025) - P3 | "The study included adult and pediatric patients with a diagnosis of hemophilia A whoswitched from a prior therapy (standard half-life FVIII [SHL]; extended half-life FVIII [EHL] or non-factortherapy [emicizumab]) to efanesoctocog alfa in routine clinical practice. Patientsexperienced improved bleed protection, especially joint bleeds, and required fewer injections comparedto SHL/EHL. Comparisons before and after the switch to efanesoctocog alfa showed a meaningfulreduction in ABRs, highlighting that clinical outcomes in real world use were consistent with thoseobserved in the clinical trial program."

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

Assay of Efanesoctocog Alfa in 200 Centres: Data Form Collaborative NEQAS BC (United Kingdom) and ECAT (Netherlands) Exercise Autumn 2024. (PubMed, Haemophilia) - "The NEQAS BC and ECAT exercise confirmed the field study: over-estimation for Actin FS OSA and all CA assays at all EFA levels plus an under-estimation for SynthasIL OSA at EFA levels of: 20, 50 and 100 IU/dL. Centres using Actin FSL on a Siemens/Sysmex or Stago analyser plus centres using Synthafax on a Werfen analysers had median recoveries within ±25% compared to the assigned potency for samples at EFA levels of: 20; 50 and 100 IU/dL. Centres using CK Prest and Pathromtin had median recoveries within ±25% compared to the assigned potency for samples at an EFA levels of 100 IU/dL."

Journal

Sobi to Showcase Scientific advances and Commitment to Haematology at ASH 2025 (PRNewswire) - "Sobi's innovative portfolio including data from efanesoctocog alfa, pegcetacoplan, avatrombopag, emapalumab, and pacritinib....Overview of treatment advances with complement Inhibitors in patients with Paroxysmal Nocturnal Haemoglobinuria; Optimising PNH treatment with the complement inhibitor Pegcetacoplan: A case report; User experience with Pegcetacoplan on-body Injector in patients with Paroxysmal Nocturnal Hemoglobinuria."

Clinical data • Aplastic Anemia • Chronic Myelomonocytic Leukemia • Hemophagocytic lymphohistiocytosis • Hemophilia A • Immune Thrombocytopenic Purpura • Myelofibrosis • Paroxysmal Nocturnal Hemoglobinuria

In Silico Assessment of Limited Blood Sampling Strategies for Individualised Pharmacokinetic-guided Dosing of Efanesoctocog Alfa in Haemophilia A Patients. (PubMed, Haemophilia) - "Several LSSs demonstrated adequate predictive performance for PK-guided dosing of efanesoctocog alfa."

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

Efanesoctocog Alfa Improves Outcomes for Patients With Hemophilia A (DocWire) - "Patients received efanesoctocog alfa for a median of 14.0 months at a median dose of 50 IU/kg, and most received a once-weekly injection....In the prophylaxis cohort, the annualized bleeding rate reduced from 1.5 to 1.2 in the SHL group, from 2.2 to 0.6 in the EHL group, and from 1.6 to 0.7 in the emicizumab group. The longer half-life of efanesoctocog alfa allowed for less frequent injections and greater protection against joint bleeds among patients who switched from SHL (n=12) or EHL therapies (n=12) to efanesoctocog alfa for prophylaxis or on-demand treatment. Similarly, patients who switched from emicizumab to efanesoctocog alfa had improved control of joint bleeds and improved bleed protection during exercise. Patients did not require blood transfusions or experience unexpected bleeding events."

Real-world • Hemophilia A

Matching-Adjusted Indirect Comparison of Personalized Prophylaxis with Simoctocog Alfa Versus Standard Prophylaxis with Efanesoctocog Alfa in Adults with Severe Hemophilia a (ASH 2023) - "An indirect comparison analysis demonstrated that a PK-guided, personalized prophylaxis with simoctocog alfa in adult PTPs resulted in zero bleed rates and ABRs that are not statistically different from those with efanesoctocog alfa. The higher total weekly dose observed with simoctocog alfa is to be expected based on the dosing recommendations for each respective FVIII replacement product. This MAIC analysis provides important comparative efficacy and utilization data, which can help guide patients and physicians in making decisions regarding product choice for prophylaxis regimens."

Clinical • Hematological Disorders • Hemophilia • Hemophilia A • 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

Treatment of Bleeding Episodes with Efanesoctocog Alfa in Children with Severe Hemophilia A in the XTEND-Kids Phase 3 Study (ASH 2023) - P3 | "Once-weekly efanesoctocog alfa provided efficient prophylaxis with no bleeds in 64% of participants, and consistently low ABRs among all types and locations of bleeds. Efanesoctocog alfa provided effective treatment and prevention of bleeds in previously treated children <12 years of age with severe hemophilia A."

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

Experiences with Efanesoctocog Alfa: Exit Interviews with Caregivers of Previously Treated Patients with Hemophilia A from the XTEND-Kids Phase 3 Clinical Trial (ASH 2023) - P3 | "Conclusion Caregivers of children with severe hemophilia A reported major impacts at study baseline on both daily life and functioning with prior treatments. Efanesoctocog alfa prophylaxis provided improvements in both children and their caregiver's daily life and functioning, and all caregivers preferred efanesoctocog alfa to their child's previous treatment."

Clinical • Interview • P3 data • Hematological Disorders • Hemophilia • Hemophilia A • Musculoskeletal Pain • Osteoarthritis • Pain • Rare Diseases

Once-Weekly Efanesoctocog Alfa Prophylaxis Provided High Sustained Factor VIII Activity Levels, Independent of Blood Group, in Children <12 Years of Age with Severe Hemophilia A (ASH 2023) - P3 | "At steady state (Week 26), FVIII activity levels were maintained in the normal to near-normal range (>40 IU/dL) for 3 days and remained >10 IU/dL for ~7 days following a 50 IU/kg dose of efanesoctocog alfa. Once-weekly prophylaxis with efanesoctocog alfa provided high sustained FVIII levels across the week for children <12 years of age in the XTEND-Kids study."

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

Equitable Care for Severe Hemophilia_A: Distributional Cost-Effectiveness of Prophylactic Weekly Efanesoctocog Alfa Versus Standard-Care Factor VIII in Patients with Severe Hemophilia_A in the United States (ASH 2023) - "As compared to standard-care factor VIII and at VA-FSS pricing, efanesoctocog alfa prophylaxis is conventionally cost-ineffective for patients with severe hemophilia A. Prophylaxis with efanesoctocog alfa does not meet commonly used distributive equity standards in the United States at this time."

Clinical • Cost effectiveness • HEOR • Cerebral Hemorrhage • CNS Disorders • Hematological Disorders • Hemophilia • Hemophilia A • Osteoarthritis • Rare Diseases • Rheumatology

Experience with Accelerometer Physical Activity Tracking in Adults and Adolescents with Hemophilia A: Tool Validation Using Results from the XTEND-1 Efanesoctocog Alfa Phase 3 Trial (ASH 2023) - P3 | "Analyses of these results revealed limitations in the psychometric properties of the assessed outcomes. To further understand the personal motivation and barriers for improving physical activity, addition of goal-setting could be considered in future clinical studies."

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

Clinical Outcomes over 2 Years of Once-Weekly Efanesoctocog Alfa Treatment in Children with Severe Hemophilia A: Second Interim Analysis from the Phase 3 XTEND-ed Long-Term Extension Study (ASH 2024) - P3 | "None of the TEAEs led to death or treatment discontinuation. There were no thrombotic events.ConclusionThe results from over 2 years in previously treated children with severe hemophilia A show that once-weekly efanesoctocog alfa continues to be well tolerated and provides highly effective bleed protection with no FVIII inhibitors reported."

Clinical • Clinical data • P3 data • P3 data: top line • CNS Disorders • Cough • Epilepsy • Hematological Disorders • Hemophilia • Hemophilia A • Musculoskeletal Pain • Pain • Rare Diseases • Respiratory Diseases

Real-World Use of Efanesoctocog Alfa in Adult Patients with Hemophilia a Including Those with a History of Factor VIII Inhibitor (ASH 2024) - "Eighteen PwHA used extended half-life recombinant Factor VIII products, one used standard half life products, and four used emicizumab-kxwh. Our cost analysis is based on estimates, actual costs may differ. Future studies should aim to evaluate long-term comprehensive outcomes in patients on Efa prophylaxis and its impact on health care system utilization."

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