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

ALTITUDE: Long-term Study Evaluating Joint Health in People With Haemophilia A Receiving Real-world Prophylactic Treatment With Efanesoctocog Alfa (clinicaltrials.gov) - P4 | N=252 | Active, not recruiting | Sponsor: Swedish Orphan Biovitrum | Recruiting ➔ Active, not recruiting

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

TG-INSIGHT With Joint POCUS, Hemostatic Potential in Patients With Severe Hemophilia A on Novel Replacement and Substitution FVIII Therapies (clinicaltrials.gov) - P=N/A | N=60 | Recruiting | Sponsor: University of Texas Southwestern Medical Center | Not yet recruiting ➔ Recruiting

Enrollment open • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Long-term safety of treatment of hemophilia A: a comprehensive review. (PubMed, Expert Opin Drug Saf) - "This review summarizes long-term safety data across the current therapeutic landscape for hemophilia A. Evidence from pivotal clinical trials, extension studies, real-world registries, and recent international congress reports was evaluated to assess the safety profiles of standard- and extended-half-life FVIII concentrates, efanesoctocog alfa, non-factor therapies such as emicizumab, emerging rebalancing agents targeting natural anticoagulant pathways, and adeno-associated viral vector-based gene therapies. Gene therapy offers durable FVIII expression after a single administration but raises concerns about hepatotoxicity, expression durability, and long-term safety. Ongoing pharmacovigilance and individualized treatment selection remain essential as therapeutic options continue to expand."

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

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=79 | Active, not recruiting | Sponsor: Sanofi | Recruiting ➔ Active, not recruiting

Enrollment closed • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

E-PROTECT: Efanesoctocog Prophylaxis Or The Optimization Of Treatment: Real-World Experience Of Taylorizing Clotting Therapy In Hemophilia A (clinicaltrials.gov) - P=N/A | N=553 | Not yet recruiting | Sponsor: Nantes University Hospital

New trial • Real-world evidence • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Experiences with Efanesoctocog Alfa: Exit Interviews with Caregivers of Previously Treated XTEND-Kids Phase III Trial Patients with Haemophilia A. (PubMed, Patient) - P3 | "Caregiver-reported outcomes suggest that efanesoctocog alfa may meaningfully reduce disease and treatment burden, with improvements observed across children's daily functioning and emotional wellbeing, and caregiver experience."

Interview • Journal • P3 data • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Evaluating Costs and Efficacy of rFVIII Prophylaxis Using Matching-Adjusted Indirect Comparisons in Hemophilia A. (PubMed, TH Open) - "Methods Matching-adjusted indirect comparisons (MAICs) were performed to compare simoctocog alfa with efanesoctocog alfa (ALTUVIIIO) and turoctocog alfa pegol (Esperoct), and results were integrated with a previously published MAIC comparing simoctocog alfa with efmoroctocog alfa (ELOCTATE), damoctocog alfa pegol (JIVI), and rurioctocog alfa pegol (ADYNOVATE). In comparison with efanesoctocog alfa (Group A), no significant differences were observed in the proportion of individuals with zero bleeds, whereas treated total annualized bleeding rate (ABR) and treated spontaneous ABR favored efanesoctocog alfa. Conclusion This indirect comparison suggests that personalized prophylaxis with simoctocog alfa may offer economic advantages versus EHL rFVIII products in individuals with severe hemophilia A. Clinical outcomes were broadly comparable across comparators, although treated total and spontaneous ABRs were significantly lower with efanesoctocog alfa (Group A)."

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

Successful immune tolerance induction using efanesoctocog alfa in severe hemophilia A: A case series. (PubMed, J Thromb Haemost) - "Transition to once-weekly individualized prophylaxis maintained functional FVIII activity and therapeutic FVIII trough levels (4%-6%), allowing discontinuation of emicizumab. Sustained bleed-free outcomes were achieved with individualized prophylactic dosing without inhibitor recurrence. These cases support efanesoctocog alfa-based ITI as a promising strategy for rescue and retreatment patients with complex inhibitor histories."

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

New and novel pharmacotherapies for hemophilia A: an update. (PubMed, Expert Opin Pharmacother) - "In replacement therapy, efanesoctocog alfa maintains normal-to-near-normal factor VIII (FVIII) levels weekly by bypassing endogenous von Willebrand factor dependence. In non-replacement therapies, the focus centers on rebalancing agents - the anti-tissue factor pathway inhibitor (TFPI) monoclonal antibodies concizumab and marstacimab, and the antithrombin-targeting small interfering RNA (siRNA) fitusiran - as well as next-generation FVIII-mimetics like denecimig...Rebalancing therapies present potential thromboembolic risks, complex breakthrough bleed protocols, and standard laboratory assay interference (requiring antithrombin monitoring or specialized assays). Critical goals for contemporary hemophilia management include tailoring therapies through multidisciplinary collaboration, monitoring subclinical joint disease via point-of-care ultrasound, and implementing standardized, real-world protocols for emergency hemostasis."

Journal • Review • Cardiovascular • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases • Rheumatology

Treatment satisfaction and patient preference for efanesoctocog alfa over previous hemophilia A treatment: Results from the XTEND-1 phase 3 clinical trial. (PubMed, Curr Ther Res Clin Exp) - P3 | "There were no strong correlations with treatment satisfaction improvements at Week 52 and improvement in quality of life or bleeding episodes. Prophylactic efanesoctocog alfa was preferred to prior prophylactic regimens by most patients and improved patient satisfaction at 52 weeks."

Journal • P3 data • 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 Blood Health | Trial completion date: Aug 2032 ➔ Aug 2033 | Initiation date: Oct 2025 ➔ Dec 2026 | Trial primary completion date: Dec 2030 ➔ Dec 2031

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

CHALE II: Study of Surgical Practices in Hemophilia A Patients Treated With Efanesoctocog Alfa (Altuvoct) (clinicaltrials.gov) - P=N/A | N=160 | Recruiting | Sponsor: Hospices Civils de Lyon

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

Impact of Heat-Stable Factor VIII Mimetics and Replacement Therapies on Inhibitor Titer using a Modified Nijmegen-Bethesda Test Across Bovine Chromogenic FVIII Assays (ISTH 2026) - "Samples were supplemented with Mim8 (0, 5, 8, 10, and 20 µg/mL), emicizumab (0, 25, 50, 75, and 150 µg/mL) or rFVIII products (Altuviiio and Advate) at 0.05, 0.2, 0.8, and 1.0 IU/mL. Comparable trends were observed between the new bovine CRYOcheck chromogenic FVIII assay and both the Coatest and Siemens assays in inhibitor testing. DOI*10.1016/j.rpth.2026.104826"

Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Real World Experience of Efanesoctocog Alfa in Irish Children with Severe Haemophilia A and High Intensity Sporting Activities (ISTH 2026) - "Five children switched from emicizumab, five switched from elocta and two were on both emicizumab and elocta. Conclusions Efanesoctocog alfa offers effective protection with good trough levels for older children with severe haemophilia A and high intensity sporting activities. DOI*10.1016/j.rpth.2026.105037"

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

The conversion factor for monitoring efanesoctocog alfa is not always 2.5 (ISTH 2026) - "UK guidance suggests that laboratories should: use appropriate assays; only quote a correction factor when a CSA is used in the presence of emicizumab; and report that Altuvoct is overestimated by 1.5-3.0 times. Table or Figure Upload (1) Figure 1: Conversion factors for each sample, and results of applying a correction factor of 2.5 (dotted blue line: line of identity). Page 2 DOI*10.1016/j.rpth.2026.104825"

Hematological Disorders • Hemophilia • Rare Diseases

Septic arthritis in a patient with severe haemophilia A. (ISTH 2026) - "Routine prophylaxis with emicizumab and efanesoctocog alfa continued. It highlights the need of early-stage suspicion and the importance of coordinated multidisciplinary management. DOI*10.1016/j.rpth.2026.106558"

Clinical • Hematological Disorders • Hemophilia • Hemophilia A • Human Immunodeficiency Virus • Immunology • Infectious Disease • Inflammation • Inflammatory Arthritis • Musculoskeletal Diseases • Musculoskeletal Pain • Orthopedics • Rare Diseases • Rheumatology • CD4

Indirect treatment comparison of emicizumab versus efanesoctocog alfa prophylaxis in pediatric people with severe hemophilia A (ISTH 2026) - P3 | "Table or Figure Upload (1) Table 1. Adjusted (MAIC-weighted) comparison of bleed types between emicizumab and efanesoctocog alfa Page 2 DOI*10.1016/j.rpth.2026.104840"

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

Impact of cost-effectiveness in patients with hemophilia A who switched their prophylactic medicines to Efanesoctcog-alfa (ISTH 2026) - "Background Efanesoctocog-alfa(Efa) is a novel factor concentrate product, which is called "high sustained factor(HSF) concentrate", and can sustain >40% of factor VIII concentration in plasma for 3 or 4 days by infusing once a week...However, from a cost-effectiveness perspective, this study found that switching from emicizumab to Efa was preferable...One thousand yen is equivalent to approximately 6.329 dollars. Page 2 DOI*10.1016/j.rpth.2026.104879"

Clinical • Cost effectiveness • HEOR • Hematological Disorders • Hemophilia • Hemophilia A • Preventive care • Rare Diseases

TG-INSIGHT With Joint POCUS, Hemostatic Potential in Patients With Severe Hemophilia A on Novel Replacement and Substitution FVIII Therapies (clinicaltrials.gov) - P=N/A | N=60 | Not yet recruiting | Sponsor: University of Texas Southwestern Medical Center

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

When One Size Does Not Fit All: Prophylaxis Choice and Switching Patterns in Severe Haemophilia A in the Era of Emicizumab and Efanesoctocog Alfa (ISTH 2026) - "This data suggests that usage will predominantly be for breakthrough bleeding and perioperative haemostasis. DOI*10.1016/j.rpth.2026.104868"

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

Concordance between FVIII activity and antigen levels in patients treated with Efanesoctocog alfa: benefits of drug-specific standards (ISTH 2026) - "Methods FVIII:Act and antigen were measured in 35 plasma samples collected from 12 hemophilia A patients in treatment with Efanesoctocog alfa either alone or in presence of emicizumab. FVIII values are expressed as %. Page 2 DOI*10.1016/j.rpth.2026.104834"

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

Indirect Comparison of Marstacimab, Emicizumab, and Efanesoctocog Alfa in Hemophilia A Prophylaxis (ISTH 2026) - "Formal network meta-analysis needed for definitive conclusions. Table or Figure Upload (1) table 1 Page 2 Table or Figure Upload (2) Figure 1 DOI*10.1016/j.rpth.2026.104831"

Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Unmet needs despite standard-of-care prophylaxis in Hemophilia A: improved outcomes after switching to Efanesoctocog Alfa. (PubMed, Blood Adv) - "The ECHO (MR-004, No. In conclusion, efa effectively addresses complex unmet needs, including in emicizumab-treated patients. However, the discrepancy between results of local OSAs and drug-calibrated CSA highlights challenges in laboratory monitoring."

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

Novel ITI concept using Efanesoctocog alfa in a modified Atlanta Protocol – Update on the Gephard protocol (ISTH 2026) - "All patients received concomitant bleeding prophylaxis with emicizumab. Prospective, multicenter studies are required to confirm these observations and to establish standardized dosing strategies. DOI*10.1016/j.rpth.2026.103638"

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

Retrospective, Real-World Study of Annual Bleeding Rate Changes Reported in the Microhealth Digital App by Patients with Hemophilia A Transitioning to Efanesoctocog Alfa (ISTH 2026) - "Aims To evaluate patient-reported hemostatic outcomes and adherence in US patients transitioning from standard half-life (SHL) products, extended half-life (EHL) products, or factor mimetic (emicizumab), to efa, in the real-world setting. Table or Figure Upload (1) Table 1. ABR for PWHA without inhibitors who transitioned to efa as reported in the Microhealth digital application Page 2 DOI*10.1016/j.rpth.2026.104845"

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