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

Factor VIII Protein Modifications Influence the Immune Recognition of FVIIIFc-VWF-XTEN in Hemophilia A Patient Samples (THSNA Travel Awardee) (THSNA 2026) - "FVIII protein modifications differentially alter binding of affinity-matured FVIII antibodies in vitro, both in inhibitor and non-inhibitor samples. Whether the observed changes in antibody binding with FVIIIFc-VWF-XTEN would translate to more rapid immune tolerance induction in inhibitor patients or reduced antibody formation in PUPs warrants further investigation. No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author."

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

Type 2N von Willebrand Disease in Pregnancy and Postpartum: A Case Report (THSNA 2026) - "Due to the high-risk nature of her pregnancy and poor outcome from the previous 2 pregnancies, treatment was initiated with recombinant vWF replacement (VonvendiÒ) biweekly to maintain hemostasis antepartum. Frequent infusions and inadequate FVIII recovery led to treatment transition to efanesoctocog alfa (AltuviiioÒ) at 17 weeks gestation with goal FVIII trough of >10%...Re-bleeding occurred after discontinuation of treatment and required additional therapy about every 2-3 weeks with WilateÒ and tranexamic acid...Further investigation and research should be conducted on the bleeding tendencies of patients with 2N von Willebrand Disease, especially surrounding pregnancy and postpartum care. No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author."

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

Heat inactivation of Efanesoctocog alfa (Altuviiio) in patient's plasma to accurately determine Factor VIII Inhibitor titer by Bethesda assay. (THSNA 2026) - "An extended 120-minute heat treatment at 56°C appears sufficient to inactivate endogenous and therapeutic FVIII activity prior to Bethesda assay in samples collected from HA patients receiving Efa, thus allowing for accurate measurement of inhibitor titer. No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author."

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

Assay Performance and Heat-Inactivation Challenges of Efanesoctocog Alfa in Factor VIII Activity and Inhibitor Testing (THSNA 2026) - "This study confirms the variability in Efa measurement with FVIII:C and CH8 assays. Efa causes assay-dependent variability in Factor VIII activity measurements and interferes with inhibitor testing. Optimized heat-inactivation guidelines are needed for accurate titers in patients receiving Efa."

Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Bleed Treatment Outcomes from the XTEND-ed Study in Patients Aged 50 Years and Older (THSNA 2026) - P3 | "Results of this post hoc analysis in patients aged ≥50 years in the XTEND-ed study show that efanesoctocog alfa provides highly effective bleed protection and improvements in patient-reported outcomes without any new safety signals. Funding: Study funded by Sanofi and Sobi. No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author."

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

Thrombin Generation in Pediatric Haemophilia A Patients on Extended Half-Life FVIII versus Non-FVIII Therapies. (PubMed, Haemophilia) - "Pediatric patients on EHL-FVIII prophylaxis demonstrated higher ETP in vitro using PPP compared to those on EMI prophylaxis. These findings highlight the need for further systematic investigations to explore the implications of these differences in bleed control."

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

Challenges and Opportunities in Post-Marketing Reporting of Factor VIII Inhibitors With Efanesoctocog Alfa. (PubMed, Haemophilia) - No abstract available

Journal • P4 data

A Psychometric Analysis of the Haemophilia Quality of Life Questionnaire for Adults Physical Health Subscale and Total Scores: Data From the Phase 3 XTEND-1 Trial in Patients With Severe Haemophilia A. (PubMed, Haemophilia) - P3 | "The Haem-A-QoL PH is a reliable, valid, and responsive measure to assess the impact of interventions in the treatment of haemophilia A."

HEOR • Journal • P3 data • Hematological Disorders • Hemophilia • Hemophilia A • Pain • Rare Diseases

2025: strong sales and EPS growth. (GlobeNewswire) - "ALTUVIIIO (haemophilia A) sales were €324 million of which 85% was in the US...Rest of World sales of €48 million benefited from the launches in Japan and Taiwan...Rezurock (chronic graft-versus-host disease) sales were €113 million and decreased by 6.1%...Sales in Europe were -€4 million, mainly from a one-time credit in the UK. In Rest of World, sales were €12 million, the overwhelming majority from the launch in China; Cablivi (acquired thrombotic thrombocytopenic purpura) sales were €69 million and increased by 1.4%, driven by more patients being treated in the US and Europe offset by an element of price impact in the US....Wayrilz (immune thrombocytopenia) sales were €6 million, all in the US, following approval in August 2025; Qfitlia (haemophilia A and B) sales were €4 million, all in the US, following approval in March 2025."

Sales • Chronic Graft versus Host Disease • Hemophilia A • Hemophilia B • Immune Thrombocytopenic Purpura

Press release: 2025: strong sales and EPS growth. Continued profitable growth expected in 2026 (The Manila Times) - "Pharma launches increased sales by 49.4%, reaching €1.1 billion, primarily driven by Ayvakit and ALTUVIIIO"

Commercial • Aggressive Systemic Mastocytosis • Hemophilia A

Outcomes in participants switching from FVIII replacement therapy to efanesoctocog alfa prophylaxis in XTEND-1: a post hoc analysis. (PubMed, Ther Adv Hematol) - P3 | "Collectively, the results of this post hoc analysis demonstrate the benefits of once-weekly efanesoctocog alfa prophylaxis over SHL or EHL FVIII prophylaxis on bleed rates, injection frequency and consumption. Observational study: 242HA201/OBS16221; XTEND-1: NCT04161495 (https://clinicaltrials.gov/study/NCT04161495)."

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

ACTIVIIITY: Goal Attainment and Physical Activity in People With Hemophilia A (clinicaltrials.gov) - P=N/A | N=3 | Terminated | Sponsor: Sanofi | N=35 ➔ 3 | Trial completion date: Aug 2026 ➔ Sep 2025 | Recruiting ➔ Terminated | Trial primary completion date: Aug 2026 ➔ Sep 2025; Early discontinuation based on sponsor decision not driven by any safety concerns.

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

On Factor VIII Assay Discrepancies in Post-infusion Samples Obtained from Patients Treated with Efanesoctocog Alfa. (PubMed, Hamostaseologie) - "Further analysis demonstrated different behavior of spiked and post-infusion samples within the FVIII CSA. Future studies will reveal underlying mechanisms and assess if drug-specific calibration will sufficiently correct for this phenomenon."

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

The First Three Japanese Haemophilia A Patients in Whom Factor VIII Inhibitors Were Observed During Prophylactic Treatment With Efanesoctocog Alfa. (PubMed, Haemophilia) - No abstract available

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

MFDS Approves Rare Hemophilia A Treatment “Altuviiio Injection“ (The Asia Business Daily) - "The Ministry designated this drug as a target for the Global Innovative Product Fast-Track (GIFT) review system and expedited its review and approval."

Korea approval • Hemophilia A

Pharmacokinetic Comparison of Efanesoctocog Alfa vs Other EHL-rFVIII Products in Participants With Severe Haemophilia A (clinicaltrials.gov) - P1 | N=24 | Recruiting | Sponsor: Swedish Orphan Biovitrum | Trial completion date: Oct 2025 ➔ May 2026 | Trial primary completion date: Sep 2025 ➔ Apr 2026

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

A Study to Investigate the Course of Synovial Hypertrophy in Patients With Haemophilia A on Efanesoctocog Alfa Prophylaxis (clinicaltrials.gov) - P4 | N=37 | Active, not recruiting | Sponsor: Swedish Orphan Biovitrum | Recruiting ➔ Active, not recruiting

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

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