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

Comparative Effectiveness of Valoctocogene Roxaparvovec and Efanesoctocog Alfa in the Treatment of Severe Hemophilia A: A Matching-Adjusted Indirect Comparison of Bleeding Frequency. (PubMed, Adv Ther) - "This MAIC suggests that valoctocogene roxaparvovec provides a greater likelihood of patients experiencing zero treated bleeds compared with efanesoctocog alfa during the first year following treatment initiation."

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

UTILITI: Understanding Treatment Outcomes and Immunologic Mechanisms in Altuviiio Immune Tolerance Induction (clinicaltrials.gov) - P4 | N=15 | Not yet recruiting | Sponsor: Versiti

New P4 trial • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Long-Term Anticoagulation in a Severe Hemophilia A Patient Receiving Efanesoctocog Alpha Prophylaxis: A Case Report. (PubMed, Semin Thromb Hemost) - No abstract available

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

Turning up the heat: optimizing Bethesda assays for efanesoctocog alfa monitoring. (PubMed, Res Pract Thromb Haemost) - No abstract available

Journal

Post hoc analysis of bleeding episodes and clinically relevant pharmacokinetic parameters among children <12 years old with severe haemophilia A receiving once-weekly efanesoctocog alfa prophylaxis in the XTEND-Kids phase 3 multinational trial. (PubMed, J Thromb Haemost) - P3 | "Once-weekly prophylaxis with efanesoctocog alfa provided high-sustained FVIII levels and highly effective bleed treatment and prevention in previously treated children <12 years of age with severe haemophilia A."

Journal • P3 data • PK/PD data • Retrospective data • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

A Phase 1, Open-Label Study to Assess the Pharmacokinetics, Safety, and Tolerability of a Single Intravenous Injection of Efanesoctocog Alfa in Adults with Type 2N or Type 3 von Willebrand Disease. (PubMed, J Thromb Haemost) - P1 | "Efanesoctocog alfa is well-tolerated and maintains high FVIII activity levels for a prolonged period in patients with Type 2N or Type 3 VWD."

Journal • P1 data • PK/PD data • Hematological Disorders • Hemophilia • Hemophilia A • 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

Letter in Response to the Article "A Global Comparative Field Study to Evaluate the Factor VIII Activity of Efanesoctocog Alfa by One-Stage Clotting and Chromogenic Substrate Assays at Clinical Haemostasis Laboratories". (PubMed, Haemophilia) - No abstract available

Journal

Altuvoct: Innovative Medicinal Products Benefit from Innovative Approaches to Regulatory Assessment. (PubMed, Life (Basel)) - "Data from two single-arm ongoing studies were submitted: the XTEND-1 study enrolled 159 subjects aged 12-72 years, and the XTEND-kids study enrolled 74 subjects aged <12 years; all subjects had severe haemophilia A. Single-arm studies are not amenable to conventional statistical analysis of 'effect of cause', and so a supplementary analysis was conducted on the basis of 'cause of effect', making use of the scheme described by Toulmin coupled to an analysis of causal inference. Overall, the claim that Altuvoct is indicated to treat people aged ≥2 years with severe (and moderate) haemophilia A was considered to be supported by the results of the submitted studies and associated modelling exercises; the benefit-risk evaluation of Altuvoct was found to be positive in the target population."

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

PHYSICIANS OPTIMISTIC ABOUT NEXT EVOLUTION IN HEMOPHILIA PATIENT CARE (EHA 2025) - "Advancements in therapy are moving beyond factor replacement, with novel approaches such as gene therapy and non-factor therapies, including emicizumab, marstacimab, concizumab, fitusiran, and Mim8...The greatest challenges were cited as selecting the best treatment option (30%), patient adherence (28%) and securing insurance coverage (20%) - especially for newer options.45% of hematologists have made changes to the way they manage hemophilia A patients in the past year with more adoption of emicizumab and efanesoctocog alfa, for which they report high satisfaction... Managing hemophilia patients throughout their lifetime is challenging for hematologists as they aim to improve patient quality of life and lifestyle. Newer options are providing better efficacy for different patient types and with favorable administration. As these options are approved and launched, hematologists expect many patients will be candidates and they will quickly begin prescribing these agents as..."

Clinical • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Musculoskeletal Pain • Pain • Rare Diseases

CHATGPT4O® AND WEEKLY PROPHYLAXIS WITH FVIIIC: WHAT CAN IT CONTRIBUTE? (EHA 2025) - "Within the VIII factors of extended half-life, Esperoct® and JIvi® include in their technical data sheets the weekly prophylaxis at different doses (75 IU/Kg and 60 IU/Kg, respectively). These FVIIICs have demonstrated efficacy and safety in clinical trials. Altuvoct® is a revolution and inaugurates a new class of CFVIII, decoupling its half-life from that of VWF. All in all, personalization of hemophilia treatment is a key aspect as well as economic management."

Hematological Disorders • Hemophilia • Rare Diseases

Joint Health Outcomes with Efanesoctocog Alfa in Adults/Adolescents from XTEND-1 Continuing XTEND-ed (ISTH 2025) - P3 | "HJHS total score improved or remained constant in 88% of evaluable participants (n=115). Table or Figure Upload"

Clinical • HEOR • Hematological Disorders • Hemophilia • Hemophilia A • 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

Observational Study of Prophylactic Efanesoctocog Alfa for Joint Health in Hemophilia A: PROTECT-ALT (ISTH 2025) - P3 | "Standardized MRI-based International Prophylaxis Study Group (IPSG) scoring will be utilized along with the Hemophilia Joint Health Score (HJHS) and the Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) for monitoring joint health (Figures 1 and 2); due to challenges with MRI in children <6 years of age, Musculoskeletal Ultrasound (MSKUS) will be used in this population. Table or Figure Upload"

Clinical • Observational data • Hematological Disorders • Hemophilia • Hemophilia A • Musculoskeletal Diseases • Rare Diseases • Rheumatology

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

Efanesoctocog alfa measurement data from exercise between NEQAS & ECAT participants Autumn 2024. (ISTH 2025) - "Participants were asked to measure factor VIII in these samples by using their routine one-stage and/or chromogenic assay. Results In total 200 participants returned results (174 For one-stage assays and 148 for chromogenic assays).The table below is summary of the one stage assay results returned for samples EFA 24:01-24:04, (n≥10) Table or Figure Upload"

Emicizumab provides excellent brain protection in a large unbiased retrospective cohort (ISTH 2025) - "Additionally, 7 patients participated in clinical trials, receiving either valoctocogene roxaparvovec (n = 4), BIVV001 (n = 1) or Mim8 (n = 2) (Figure 1). In contrast, two intracranial bleeds occurred in patients on FVIII prophylaxis, one of which was fatal. Table or Figure Upload"

Retrospective data • Cerebral Hemorrhage • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Real world comparison of different one stage FVIII reagents for monitoring of efanesoctocog alfa (ISTH 2025) - "CL results compared well to M1 and M3 results but there was poor agreement with M2. Table or Figure Upload"

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

Efanesoctocog Alfa: One-Stage Clotting Assay validation and result comparison for APTT reagents. (ISTH 2025) - "Results All validation parameters were acceptable: linear calibration curve (R2 >0.985), assay precision (CV% <5%), method comparison with SynthASil and analyser comparison with Actin FSL (Sysmex CN3500) showing no statistical difference for non-Efanesoctocog alfa treatment samples (p=0.0675, 0.899).Comparable FVIII:C activity was measured using Actin FSL and SynthAFax. Significantly lower results were measured with SynthASil for spiking and post treatment samples (Fig1)"

Hematological Disorders • Hemophilia • Rare Diseases

Miniaturized Activated Partial Thromboplastin Time (APTT) Assay for Analysis of Small Sample Volumes (ISTH 2025) - "APTT correlated with FVIII activity measured by CA (r: -0.72 to -0.77, R2: 0.51 to 0.59, log-log scale). Notably, TG in mouse plasma correlated with activity of Lonoctocog alfa and Moroctocog alfa in plasma, but not with Efanesoctocog alfa (r=0.52, R2= 0.27)."

Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Minor Surgeries Outcomes with Efanesoctocog Alfa: 4 Years’ Experience in the XTEND Clinical Program (ISTH 2025) - P3 | "No transfusions were required; no treatment-related adverse events were reported. Table or Figure Upload"

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

Matched Adjusted Indirect Comparisons (MAIC) and Artificial Intelligence: Complementary? CASE REPORT (ISTH 2025) - "Aims To compare 2 products in HA , without inhibitor, aged =12 years (Efanesoctocog Alfa-XTEND-1 vs Emicizumab-HAVEN 3) with ChatGPT4o and compare the conclusion with MAIC carried out by Dra. If we prioritise maximum bleeding reduction and improvements in physical and joint health, Efa would be the choice. Considering Emicizumab's cost of 32 euros/mg (https://www.farmaceuticos.com/botplus/)) ChatGPT indicates that the cost per IU (International Unit) of Efa should be = 0.675 euros/IU to be considered more efficient vs Emi in terms of cost per ABR reduction (Graphic 2) Table or Figure Upload"

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

Persistent Musculoskeletal Challenges Despite Emicizumab Benefits in Adult Haemophilia A Patients (ISTH 2025) - "Seven patients who were offered access to emicizumab deliberately chose to remain on FVIII therapy, while three others are participating in clinical trials (MIM8, Efanesoctocog alfa). The HJHS score available for 39 patients averaged 40. During follow-up, most patients expressed frustration about persistent pain and functional limitations after switching to emicizumab Table or Figure Upload"

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

Major Surgical Outcomes with Efanesoctocog Alfa: 4 Years’ Experience in the XTEND Clinical Program (ISTH 2025) - P3 | "Efanesoctocog alfa was well tolerated. Table or Figure Upload"

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

Thrombin generation is higher in pediatric Hemophilia A on extended half-life FVIII vs emicizumab (ISTH 2025) - "Results Twenty-three pediatric patients with moderate to severe Hemophilia A were on prophylactic emicizumab (N=11, mean age 8.9) and EHL-FVIII therapies (N=12, 8 on efanesoctocog alfa, mean age 12.5). Similarly, median PT was significantly higher in EHL-FVIII prophylaxis group (38.68 (6.68-88.21) nM) than the emicizumab prophylaxis group (13.66 (2.72-24.28) nM; p=0.0022). Table or Figure Upload"

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