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

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

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

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

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

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 • 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 • Rare Diseases

Assay of Efanesoctocog Alfa in 200 Centres: Data Form Collaborative NEQAS BC (United Kingdom) and ECAT (Netherlands) Exercise Autumn 2024. (PubMed, Haemophilia) - "The NEQAS BC and ECAT exercise confirmed the field study: over-estimation for Actin FS OSA and all CA assays at all EFA levels plus an under-estimation for SynthasIL OSA at EFA levels of: 20, 50 and 100 IU/dL. Centres using Actin FSL on a Siemens/Sysmex or Stago analyser plus centres using Synthafax on a Werfen analysers had median recoveries within ±25% compared to the assigned potency for samples at EFA levels of: 20; 50 and 100 IU/dL. Centres using CK Prest and Pathromtin had median recoveries within ±25% compared to the assigned potency for samples at an EFA levels of 100 IU/dL."

Journal

Sobi to Showcase Scientific advances and Commitment to Haematology at ASH 2025 (PRNewswire) - "Sobi's innovative portfolio including data from efanesoctocog alfa, pegcetacoplan, avatrombopag, emapalumab, and pacritinib....Overview of treatment advances with complement Inhibitors in patients with Paroxysmal Nocturnal Haemoglobinuria; Optimising PNH treatment with the complement inhibitor Pegcetacoplan: A case report; User experience with Pegcetacoplan on-body Injector in patients with Paroxysmal Nocturnal Hemoglobinuria."

Clinical data • Aplastic Anemia • Chronic Myelomonocytic Leukemia • Hemophagocytic lymphohistiocytosis • Hemophilia A • Immune Thrombocytopenic Purpura • Myelofibrosis • Paroxysmal Nocturnal Hemoglobinuria

In Silico Assessment of Limited Blood Sampling Strategies for Individualised Pharmacokinetic-guided Dosing of Efanesoctocog Alfa in Haemophilia A Patients. (PubMed, Haemophilia) - "Several LSSs demonstrated adequate predictive performance for PK-guided dosing of efanesoctocog alfa."

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

Efanesoctocog Alfa Improves Outcomes for Patients With Hemophilia A (DocWire) - "Patients received efanesoctocog alfa for a median of 14.0 months at a median dose of 50 IU/kg, and most received a once-weekly injection....In the prophylaxis cohort, the annualized bleeding rate reduced from 1.5 to 1.2 in the SHL group, from 2.2 to 0.6 in the EHL group, and from 1.6 to 0.7 in the emicizumab group. The longer half-life of efanesoctocog alfa allowed for less frequent injections and greater protection against joint bleeds among patients who switched from SHL (n=12) or EHL therapies (n=12) to efanesoctocog alfa for prophylaxis or on-demand treatment. Similarly, patients who switched from emicizumab to efanesoctocog alfa had improved control of joint bleeds and improved bleed protection during exercise. Patients did not require blood transfusions or experience unexpected bleeding events."

Real-world • Hemophilia A