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

Prophylactic Weekly Efanesoctocog Alfa Versus Standard-Care Factor VIII in People Living With Severe Hemophilia A : A Cost-Effectiveness Analysis. (PubMed, Ann Intern Med) - "Prophylaxis with efanesoctocog alfa is not conventionally or distributionally cost-effective for severe hemophilia A in the United States at current pricing and equity weight thresholds. National Institutes of Health; National Heart, Lung, and Blood Institute."

HEOR • Journal • Hematological Disorders • Hemophilia • Hemophilia A • Pain • Rare Diseases • Rheumatology

Press Release: Sanofi: strong Q1 performance and 2025 guidance confirmed (Sanofi Press Release) - "ALTUVIIIO (hemophilia A) sales were €251 million of which 87% 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 €33 million benefited from the launch in Japan and sales to the collaborator in Europe....Qfitlia (hemophilia A and B) was approved in the US on March 28, 2025, with first sales recorded at the beginning of Q2 2025. Dupixent sales were €3,480 million and increased by 20.3%. Global sales were driven by increased use in all approved indications, including atopic dermatitis, asthma, chronic rhinosinusitis with nasal polyposis, eosinophilic esophagitis, prurigo nodularis, chronic spontaneous urticaria, and emerging use in COPD."

Sales • Asthma • Atopic Dermatitis • Chronic Obstructive Pulmonary Disease • Chronic Rhinosinusitis With Nasal Polyps • Chronic Spontaneous Urticaria • Eosinophilic Esophagitis • Hemophilia A • Hemophilia B • Prurigo Nodularis

Haemophilia Prophylaxis in the Age of Innovation: Exploring Opportunities for Personalized Treatment. (PubMed, Haemophilia) - "Although these new therapies offer significant promise in controlling bleeding and enhancing quality of life, challenges such as variable efficacy, potential long-term risks and high costs remain. Additionally, the absence of validated surrogate markers for non-factor therapies limits their optimal use in clinical settings. The primary goal of haemophilia care is to provide optimal personalized treatment that enables patients to lead unrestricted lives. However, the introduction of these novel treatments necessitates a shift in treatment paradigms and the development of improved evaluation tools for better personalization. Choosing the most appropriate treatment requires careful clinical follow-up, taking into account patient preferences, disease burden, and individual and environmental factors that affect patients' lives. Ongoing research and real-world studies are essential to assess long-term efficacy and safety. In addition, improving educational resources-such..."

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

Summary for Patients: Prophylactic Weekly Efanesoctocog Alfa Versus Standard-Care Factor VIII in People Living With Severe Hemophilia A. (PubMed, Ann Intern Med) - No abstract available

Journal • 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 | Not yet recruiting | Sponsor: Swedish Orphan Biovitrum

New P4 trial • Real-world evidence • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Efanesoctocog Alfa in Children With Haemophilia A: European Results From the XTEND-ed Second Interim Analysis (BSH 2025) - No abstract available

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

Sobi presents clinical data at WFH 2025 Comprehensive Care Summit (PRNewswire) - "Sobi...presents clinical data aimed at updating the global haemophilia community attending this year's WFH 2025 Comprehensive Care Summit in Dubai from the 23 – 25 April. New and updated outcomes and analyses will be presented from the XTEND phase 3 clinical program testing the effectiveness of Altuvoct (efanesoctocog alfa) treatment for haemophilia A, including updates on patients' joint health and surgical outcomes in patients."

Clinical data • Hemophilia A

LIBERTY: Study to Provide Continued Access to Treatment for Patients Completing a Previous Trial With Efanesoctocog Alfa (clinicaltrials.gov) - P3 | N=78 | Recruiting | Sponsor: Swedish Orphan Biovitrum | Not yet recruiting ➔ Recruiting

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

NICE recommends Sobi’s Altuvoct for severe hemophilia A (The Pharma Letter) - "Swedish Orphan Biovitrum (STO: SOBI)...has announced that the National Institute for Health and Care Excellence (NICE) has issued its final guidance in England and Wales recommending the use of Altuvoct (efanesoctocog alfa). The high-sustained factor VIII replacement therapy will now be available for treating and preventing bleeding episodes in people with severe hemophilia A aged two years and over. Altuvoct won approval from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) after a reduction in bleeds was demonstrated in clinical trials."

NICE • Hemophilia A

Efanesoctocog Alfa Population Pharmacokinetics and Repeated Time-To-Event Analysis of Bleeds in Adults, Adolescents, and Children with Severe Hemophilia A. (PubMed, J Clin Pharmacol) - P1/2, P3 | "Bleeding episodes in participants in XTEND-1 and XTEND-Kids were characterized by an RTTE model with a Weibull base hazard and effect of FVIII activity modeled by a power effect. The RTTE model showed the probability of being bleed-free in 1 year with efanesoctocog alfa 50 IU/kg QW regimen was >70% across all age groups, consistent with the observed clinical outcomes in the Phase 3 trials of highly effective protection from bleeding episodes in patients with severe hemophilia A, which validates the model's prediction of the long-term bleed hazard."

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

Perioperative Management With Efanesoctocog Alfa in Patients With Haemophilia A in the Phase 3 XTEND-1 and XTEND-Kids Studies. (PubMed, Haemophilia) - P3 | "Efanesoctocog alfa provided highly effective perioperative protection in patients with severe haemophilia A."

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

Miracle drug costing £510k per year to be offered on NHS for incurable disease (Express) - "Patients with an incurable, inherited condition are set to benefit from a £510,000-a-year treatment that can prevent bleeding episodes...Haemophilia A is a rare disorder that means sufferers do not have enough of the clotting factor VIII in their blood or it does not work properly...The drug, efanesoctocog alfa, is given as a weekly injection...The National Institute for Health and Care Excellence (NICE) has recommended it is offered on the NHS to patients aged two and over with severe haemophilia A, which affects an estimated 1,900 people in the UK....However, manufacturer Swedish Orphan Biovitrum has agreed a confidential discount with NHS England."

NICE • Hemophilia A

Sobi receives MHRA approval for Altuvoct in severe or moderate haemophilia A (PMLive) - "Sobi’s Sanofi-partnered efanesoctocog alfa has been approved by the Medicines and Healthcare products Regulatory Agency (MHRA) to treat haemophilia A...The drug has been authorised under the brand name Altuvoct to treat and prevent bleeding in patients aged two years and older with severe or moderate cases of the genetic bleeding disorder...The MHRA’s decision on the drug was based on results from the late-stage XTEND-1 study of 159 patients aged 12 years and older with severe haemophilia A....Also supporting the regulator’s decision were positive results from the phase 3 XTEND-Kids trial..."

MHRA approval • Hemophilia A

In vitro combined haemostatic efficacy of emicizumab and extended half-life factor VIII compounds. (PubMed, Haemophilia) - "TGA can be used to monitor combined treatment with emicizumab and either efmoroctocog alfa or efanesoctocog alfa, which is not possible with currently available FVIII reagents for the latter. As expected, there is no synergistic effect between the mimetic and FVIII at therapeutical doses. Both efmoroctocog alfa and efanesoctocog alfa show similar in vitro procoagulant activity in terms of thrombin generation."

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

Treatment of Bleeding Episodes With Efanesoctocog Alfa in Previously Treated Patients With Severe Hemophilia A in the Phase 3 XTEND-1 Study. (PubMed, Am J Hematol) - P3 | "Median total dose was 50.9 IU/kg per bleeding episode. Results of this analysis further demonstrated that once-weekly efanesoctocog alfa provides highly effective bleed protection and treatment of bleeding episodes in participants with severe hemophilia A. Trial Registration: NCT04161495."

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

Sobi publishes Q4 2024 report: A solid ending to a strong year (PRNewswire) - "Haematology revenue increased 22 per cent at CER to SEK 4,487 M (3,640), mainly driven by strong sales of Doptelet of SEK 1,147 M (727), Vonjo of SEK 416 M (322), Aspaveli/Empaveli of SEK 269 M (186) and launch sales of Altuvoct of SEK 302 M (2); Immunology revenue decreased 12 per cent at CER to SEK 2,564 M (2,905), explained by low Synagis sales of 68 M (897), partially offset by Beyfortus royalty of SEK 1,207 M (890) and sales of Kineret of SEK 777 M (621)."

Sales • Hemophilia A • Idiopathic Arthritis • Immune Thrombocytopenic Purpura • Immunology • Infectious Disease • Inflammatory Arthritis • Myelofibrosis • Myeloproliferative Neoplasm • Paroxysmal Nocturnal Hemoglobinuria • Rheumatoid Arthritis • Thrombocytopenia • Thrombocytopenic Purpura

ACTIVIIITY: Goal Attainment and Physical Activity in People With Hemophilia A (clinicaltrials.gov) - P=N/A | N=35 | Recruiting | Sponsor: Sanofi | Not yet recruiting ➔ Recruiting | Trial primary completion date: Apr 2026 ➔ Aug 2026

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

Press Release: Q4 sales growth of 10.3%, 2024 business EPS guidance exceeded, and strong business EPS rebound expected in 2025 (GlobeNewswire) - "ALTUVIIIO (hemophilia A) sales were €230 million of which more than 85% were in the US...Nexviazyme/Nexviadyme (Pompe disease) sales were €184 million and increased by 42.0%, driven by Europe (+68.6%) and Rest of World (+55.0%). In the US (+26.3%)...Rezurock (chronic graft-versus-host disease) sales were €132 million and increased by 53.5%, driven by the US (+43.4%) and by launches in Europe (sales of €8 million) and in Rest of World (sales of €5 million)...Cablivi (acquired thrombotic thrombocytopenic purpura) sales were €73 million and increased by 24.1%, driven by an increased number of patients being identified for appropriate treatment, in the US and from launches in Europe and in Rest of World...Eloctate sales were €81 million and decreased by 21.4%, mainly due to patients converting to ALTUVIIIO in the US and in Japan. Aubagio sales were €78 million and decreased by 35.5%..."

Sales • Chronic Graft versus Host Disease • Hemophilia A • Multiple Sclerosis • Pompe Disease • Thrombocytopenic Purpura

Single center experience of efanesoctocog alfa in adult and pediatric patients with hemophilia A. (EAHAD 2025) - No abstract available

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

Cost-Efficacy Analysis of Rurioctocog Alfa Pegol (PROPEL) vs. Efanesoctocog in Haemophilia A using Artificial Intelligence (AI) (EAHAD 2025) - No abstract available

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

Clinical outcomes over 2 years of efanesoctocog alfa in children with severe haemophilia A: European results from the second interim analysis of XTEND-ed (EAHAD 2025) - No abstract available

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

NEQAS BC and ECAT collaborative exercise with Efanesoctocog alfa spiked plasma for FVIIIC testing by One Stage and Chromogenic assays. (EAHAD 2025) - No abstract available

Initial clinical experience with Efanesoctocog alfa in orthopedic surgery at University Hospital Ostrava, Czech Republic (EAHAD 2025) - No abstract available

Clinical • Surgery • Orthopedics

Treatment of Bleeding Episodes with Efanesoctocog Alfa in Adults and Adolescents with Severe Haemophilia A: Second Interim Analysis of the XTEND-ed Long-term Extension Study (EAHAD 2025) - No abstract available

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

Efanesoctocog Alfa for the Perioperative Management of Patients with Severe Haemophilia A: 4 years of Experience in the XTEND Clinical Program (EAHAD 2025) - No abstract available

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