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

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

The von Willebrand factor D’''D3 region in BIVV001 alters the immune response to factor VIII (ISTH 2025) - "There was no difference in total IgG titers between rFVIII variants. Table or Figure Upload"

Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases • F8

Treatment of Bleeding Episodes with Efanesoctocog Alfa in Children: XTEND-ed Second Interim Analysis (ISTH 2025) - P3 | "There were two treatment-related adverse events (injection-related reaction; headache) and no treatment-related serious adverse events. Table or Figure Upload"

Clinical • Hematological Disorders • Hemophilia • Hemophilia A • Pain • 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

Long-term Study Evaluating Joint Health in People With Haemophilia A Receiving Real-world Prophylactic Treatment With Efanesoctocog Alfa (clinicaltrials.gov) - P4 | N=250 | Recruiting | Sponsor: Swedish Orphan Biovitrum | Not yet recruiting ➔ Recruiting

Enrollment open • Real-world evidence • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

ISTH: new data highlight innovation from Sanofi’s pipeline in rare diseases (Sanofi Press Release) - "New data from 18 abstracts, including five oral presentations, will be presented at the 33rd congress of the International Society on Thrombosis and Haemostasis (ISTH) in Washington D.C., US from June 21 to 25, 2025, highlighting Sanofi as a leader in hemophilia committed to rare blood diseases. Data to be presented expand on the potential of rilzabrutinib to address the underlying immune dysregulation of immune thrombocytopenia (ITP) and strengthen Sanofi as a leader in hemophilia with ALTUVIIIO and the newly launched Qfitlia, aimed at providing more treatment options to help improve patients’ lives."

Clinical data • Hemophilia • Thrombocytopenia

PROTECT-ALT: A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A in Taiwan (clinicaltrials.gov) - P=N/A | N=100 | Recruiting | Sponsor: Sanofi | Initiation date: Nov 2024 ➔ Jan 2024

Trial initiation date • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Balancing Hemostasis and Thrombosis in Pediatric Hodgkin Lymphoma With Co-Existing Hemophilia A (ASPHO 2025) - "Lymphoma was treated per AHOD0031 with Doxorubicin, Bleomycin, Vincristine, Etoposide, Prednisone, and Cyclophosphamide...Efanesoctocog alfa was chosen for the ability to titrate and monitor factor VIII levels over emicizumab... This case highlights the first reported instance and the challenges in managing concurrent HL and hemophilia A in a pediatric patient. Prophylaxis with factor replacement enabled successful delivery of myelosuppressive chemotherapy without significant bleeding complications. This case underscores the importance of individualized care that balances hemostasis and thrombosis in patients with complex coexisting conditions."

Clinical • Cardiovascular • Classical Hodgkin Lymphoma • Hematological Disorders • Hematological Malignancies • Hemophilia • Hemophilia A • Hodgkin Lymphoma • Lymphoma • Oncology • Pediatrics • Rare Diseases • Thrombocytopenia • Thrombosis

Efanesoctocog alfa not cost-effective for prophylaxis in hemophilia A (Medical Xpress) - "For U.S. patients with severe hemophilia A, prophylaxis with efanesoctocog alfa is neither conventionally nor distributionally cost-effective, according to a study published online April 22 in the Annals of Internal Medicine....The researchers found that efanesoctocog alfa and standard-care factor VIII accrued 19.7 and 14.6 discounted lifetime quality-adjusted life years (QALYs) at costs of $22.1 and $11.2 million, respectively; the incremental cost-effectiveness ratio per QALY was $2.13 million. The distributional cost-effectiveness analysis-derived threshold equity weight was 6.9, which exceeded U.S. equity standards that are commonly used. Across all scenario analyses, the results were consistent."

HEOR • Hemophilia A