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

The Laboratory Monitoring of Altuvoct (Efanesoctocog Alfa): Recommendations From the Laboratory Working Party of the United Kingdom Haemophilia Centres Doctor's Organisation. (PubMed, Haemophilia) - "In the absence of FVIII mimetic therapy, FVIII inhibitors must be assessed using heat-treated patient plasma and a one-stage APTT assay to measure residual FVIII in a Bethesda or Nijmegen-modified Bethesda assay (1B). In the presence of concomitant FVIII-mimetic therapy, FVIII inhibitors must be assessed using heat-treated patient plasma and a chromogenic FVIII assay that uses bovine FX and FIXa substrates to measure residual FVIII in a Bethesda or Nijmegen-modified Bethesda assay (1B)."

Journal • Hematological Disorders • Hemophilia • Rare Diseases

Cost Effectiveness of Efanesoctocog Alfa Versus Factor VIII Extended Half-Life in Adolescent and Adult Patients with Hemophilia A in the USA. (PubMed, Pharmacoeconomics) - "Our analysis suggests that efanesoctocog alfa was dominant versus prophylaxis with standard and elevated factor VIII extended half-life dosing regimens. Efanesoctocog alfa was associated with better joint health and, hence, contributed to fewer bleeds, lower costs, and higher quality-adjusted life-years."

HEOR • Journal • 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 • Hemophilia • Rare Diseases

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

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

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 • Psychiatry • 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 • Pediatrics • Rare Diseases

SYNOVIIIUS: Efanesoctocog Alfa Prophylaxis in Patients With Hemophilia A With Synovial Hypertrophy (clinicaltrials.gov) - P4 | N=35 | Recruiting | Sponsor: Sanofi | Not yet recruiting ➔ Recruiting | Trial completion date: Jun 2027 ➔ Oct 2027 | Initiation date: May 2025 ➔ Sep 2025 | Trial primary completion date: Mar 2027 ➔ Oct 2027

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

Efanesoctocog Alfa Prophylaxis in a Patient With Combined Mild Hemophilia A and Type 1 von Willebrand Disease. (PubMed, Pediatr Blood Cancer) - No abstract available

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

Group revenue for Q3 2025 reached SEK 7,776 M, up 21 per cent [compared to Q3 2024] at constant exchange rates (CER). This growth was driven by higher in-market performance of Sobi products, particularly for Altuvoct, Doptelet and Gamifant. (PRNewswire)

Commercial • Hemophagocytic lymphohistiocytosis • Hemophilia A • Immune Thrombocytopenic Purpura

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: Jan 2024 ➔ Nov 2024

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

A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A (clinicaltrials.gov) - P=N/A | N=200 | Active, not recruiting | Sponsor: Sanofi | Recruiting ➔ Active, not recruiting | Trial completion date: Jun 2028 ➔ Sep 2030

Enrollment closed • Trial completion date • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

LIBERTY: Study to Provide Continued Access to Treatment for Patients Completing a Previous Trial With Efanesoctocog Alfa (clinicaltrials.gov) - P3 | N=104 | Recruiting | Sponsor: Swedish Orphan Biovitrum | N=78 ➔ 104 | Trial completion date: Aug 2026 ➔ Mar 2027 | Trial primary completion date: Aug 2026 ➔ Mar 2027

Enrollment change • Trial completion date • Trial primary completion date • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Is Incubation for 30 Minutes at 56°C Sufficient to Fully Inactivate Efanesoctocog Alfa? (PubMed, Haemophilia) - No abstract available

Journal

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

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

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