Hemlibra (emicizumab-kxwh) / Roche, JW Pharma - LARVOL DELTA

Chugai Announces 2025 1st Quarter Results (Chugai Press Release) - "Core revenue, core operating profit, and core net income at ¥288.5 billion (+21.8%), ¥139.5 billion (+36.6%), and ¥99.2 billion (+30.5%), respectively (all changes year-on-year)...Regarding revenue, domestic sales remained nearly flat with a slight decrease of 0.2% year-on-year. In the oncology field, sales decreased by 5.3% compared to the previous year. While our new product Phesgo performed well, this was offset by the decline in Perjeta and Herceptin along with the market penetration of Phesgo which includes the same active pharmaceutical ingredients. Additionally, products including our mainstay product Avastin were affected by NHI drug price revisions and biosimilars...Overseas sales increased significantly by 54.7% year-on-year, driven by a substantial increase in exports of Hemlibra and Actemra to Roche."

Commercial • Ankylosing Spondylitis • Hemophilia • HER2 Breast Cancer • Lupus • Myelodysplastic Syndrome • Ovarian Cancer • Rheumatoid Arthritis • Schizophrenia • Solid Tumor

NuPOWER: Nuwiq for Perioperative Management Of Patients With Haemophilia A on Emicizumab Regular Prophylaxis Study (clinicaltrials.gov) - P4 | N=28 | Recruiting | Sponsor: Octapharma | Trial completion date: Dec 2025 ➔ Aug 2026 | Trial primary completion date: Dec 2025 ➔ Aug 2026

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

Impact of Drugs Used in Intensive Care on Routine Coagulation Testing. (PubMed, Diagnostics (Basel)) - "In this review, we have conducted a critical analysis of the literature to define: (1) the impact of hydroxocobalamin, intravenous lipid emulsion, and propofol on chromogenic assays; (2) the impact of PEGylated compounds, emicizumab, recombinant activated factor VII, antibiotics, and sugammadex on chronometric assays; (3) the challenges associated with bridging anticoagulation in the ICU as well as the effect of N-acetylcystein, serotonin reuptake inhibitors, and tramadol on the hemostasis system. For each drug, we specify the routine coagulation assay that is impacted, whether this is linked to an in vitro interference or an in vivo effect, and the potential consequences on patient management."

Journal • Review • Critical care

Follow-up on Platelet-Targeted Gene Therapy for Hemophilia A (ASGCT 2025) - P1 | "At approximately 3.5 months after HSC transplant the patient discontinued his weekly prophylaxis regimen with Emicizumab (FVIII-mimetic) and has reported an absence of bleeding events and denied administration of rFVIII prophylaxis at least 24 months after transplant. Two patients have safely undergone treatment of severe HA with a history of inhibitors using Pleightlet LV HSC FVIII gene therapy causing platelet-targeted expression of BDDFVIII now sustained for greater than 24 and 6 months (respectively) after transplant with zero bleeding events requiring infusion of FVIII products. Regular testing for gene vector copy number, insertion site analysis, platelet FVIII levels, and coagulation function indicates the potential feasibility for this approach to treat hemophilia A in a safe and efficient manner. This study will require periodic long-term follow-up to further demonstrate the efficacy of this approach."

Gene therapy • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases • Thrombosis • CD34 • ITGA2B

Real-world bleeding rates on emicizumab: the value of using nationwide digital treatment diary data in clinical research. (PubMed, Res Pract Thromb Haemost) - "Bleeding rates among Dutch people with severe hemophilia A using emicizumab were comparable to other real-world studies. We formulated recommendations to improve the quality of patient-reported bleeding data, such as establishing guidelines for recording bleeds and improving interoperability."

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

Insights from in vitro global assays on possible doses of concomitant hemostatic agents in the presence of NXT007 in hemophilia A. (PubMed, J Thromb Haemost) - "Concomitant addition of coagulation agents increased coagulation potentials in vitro in the presence of NXT007. A dose of 10 U/kg may serve as a rough indicator for the initial dose when exploring the concomitant use of aPCC at plasma NXT007 levels of approximately 30 μg/mL. Importantly, plasma NXT007 at ≥10 μg/mL demonstrated non-hemophiliac coagulation potentials in vitro."

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

TO BLEED OR NOT TO BLEED: MANAGEMENT OF ATRIAL FIBRILLATION AND LEFT ATRIAL APPENDAGE THROMBUS WITH ACQUIRED HEMOPHILIA A - Robert Monsour (ACC 2025) - "Management of rare bleeding disorders like AHA with concurrent AF requires close collaboration between Cardiology and Hematology teams to balance acute bleeding risks with longer term stroke risks."

Atrial Fibrillation • Bullous Pemphigoid • Cardiovascular • Congestive Heart Failure • Coronary Artery Disease • Dermatology • Dermatopathology • Heart Failure • Hematological Disorders • Hemophilia • Hemophilia A • Immunology • Rare Diseases • Thrombosis

Consistent clinical factor VIII equivalency is unlikely for non-factor therapies in hemophilic mice. (PubMed, Haematologica) - "Strikingly, both emicizumab- and SIA-marstacimab-treatment resulted in spontaneous rebleeds in the TVT-, TAT- and tail-clip-models, further distinguishing them from FVIII-treatment. Our data suggest that a single FVIIIequivalence is unlikely to exist for emicizumab, SIA-marstacimab and similar molecules, because their activity is dependent on local conditions and severity of the injury."

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

Trends in Treatment of Severe Haemophilia and Impact on Inhibitor Assessment by the EUHASS Registry. (PubMed, Haemophilia) - "In 2022, 44% of SHA-PUPs and 25% of SHA-PTPs received emicizumab prophylaxis. Concomitantly, observed inhibitor incidence reduced to 6% in SHA-PUPs. In SHB, EHL-rFIX treatment increased to 79% in SHB-PUPs and 69% in SHB-PTPs. Assessing inhibitor incidence for new concentrates is likely to be hampered by novel treatments causing delayed exposure to FVIII/FIX."

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

Clinical Application of Clot Waveform Analysis. (PubMed, Clin Appl Thromb Hemost) - "CWA-sTT reflects thrombin burst and evaluates hemostatic abnormalities in patients treated with emicizumab. CFWA is a variant of CWA-APTT that includes a small amount of tissue-type plasminogen activator, indicating both clotting and fibrinolysis. The CWA-APTT and modified CWA should be further investigated in various diseases for many applications in the clinical setting, including the monitoring of hemophilia patients and patients receiving anticoagulant therapy, and the differential diagnosis of diseases."

Journal • Review • Hematological Disorders • Hemophilia • Rare Diseases

A Hemophilia Joint Health Score-Based Model for the Economic Evaluation of Hemophilia A Prophylaxis Interventions. (PubMed, Pharmacoeconomics) - "This is the first cost-effectiveness model incorporating HJHS to apply sequential joint damage to hemophilia A. While EA offers clinical benefits, our analysis suggests it will not be cost-effective from a Canadian provincial Ministry of Health perspective without a significant price decrease."

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

Five-decades of successive establishment of hemophilia care in Thailand. (PubMed, Expert Rev Hematol) - "Finally, nationwide nonfactor therapy of monthly low dose emicizumab prophylaxis will be established soon. Hemophilia care in Thailand constitutes the real-world experience passing from generation to generation for five decades using our limited resources efficiently."

Journal • Review • Hematological Disorders • Hemophilia • Rare Diseases

JW Pharmaceutical's 'Hemlibra' Proven to Improve Quality of Life for Patients with Severe Hemophilia A (Korea IT Times) - "JW Pharmaceutical announced on March 31 that research presented at the annual European Association for Haemophilia and Allied Disorders (EAHAD 2025) demonstrated significant improvements in the quality of life for patients with severe Hemophilia A treated with their drug 'Hemlibra'....The team analyzed 28 patients with severe Hemophilia A (16 adults and 12 adolescents) who had been receiving prophylactic treatment with eight-factor products for more than 24 weeks and were then treated with Hemlibra for 48 weeks. The study focused on assessing quality of life and changes in bleeding events. Results showed that after treatment with Hemlibra, patients maintained stable levels of physical activity and joint health, while the occurrence of bleeding events significantly decreased."

Clinical data • Hemophilia A

Estimation of the budget impact of the dual dispensing circuit of emicizumab in France: The HemiValue study. (PubMed, J Epidemiol Popul Health) - "The dual dispensing circuit reduces the financial and logistical burdens associated with obtaining emicizumab. These results highlight the importance of innovative dispensing models that aim to ease constraints for patients and their caregivers while working towards greater equity in accessibility."

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

Impact of Emicizumab on Quality of Life and Clinical Outcomes in Patients with Hemophilia A in Guatemala (ISPOR 2025) - "The use of emicizumab in Guatemalan patients with hemophilia A resulted in improved clinical outcomes and substantial quality-of-life enhancements. These findings highlight the need to expand access to emicizumab as a comprehensive therapeutic option for this population, particularly for those with complex clinical scenarios or poor prior treatment management."

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

Population-Level Impact of Emicizumab for Hemophilia A Patients in Six Latin American (LATAM) Countries: A 25-Year Model (ISPOR 2025) - "Emicizumab in hemophilia A is associated with reducing disease burden and increasing the patient's quality of life, resulting in a significant short- and long-term cost reduction for healthcare systems in LATAM countries."

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

Early diagnosis and prophylactic treatment with emicizumab in premature infants with severe hemophilia A. (PubMed, Pediatr Neonatol) - No abstract available

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

A survey on clinical practice in monitoring and management of bleeding in children with haemophilia A on emicizumab prophylaxis in the PedNet centres. (PubMed, Thromb Res) - No abstract available

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

Acquired Hemophilia-A Case Series and Review. (PubMed, J Clin Med) - "Initial immunosuppressive therapy (IST) with corticosteroids achieved remission in 45% of patients, while additional treatment with Rituximab or Cyclophosphamide was required in others. Emicizumab shows promise, particularly in refractory cases and fragile populations. Future research is needed to evaluate long-term outcomes and spontaneous remission rates, especially in special populations like post-partum women and the elderly."

Journal • Review • Hematological Disorders • Hemophilia • Hemophilia A • Immunology • Musculoskeletal Diseases • Oncology • Orthopedics • Rare Diseases

SAFE Study: Safety of APCC Following Emicizumab Prophylaxis (clinicaltrials.gov) - P3 | N=5 | Recruiting | Sponsor: Emory University | N=20 ➔ 5

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

A plain language summary on indirectly comparing bleeding after valoctocogene roxaparvovec gene therapy to bleeding with emicizumab prophylaxis. (PubMed, Expert Rev Hematol) - No abstract available

Journal • Gene Therapies

A literature review of major surgery experience with emicizumab in people with hemophilia A without factor VIII inhibitors. (PubMed, Res Pract Thromb Haemost) - "No deaths were reported. This review provides a consolidated narrative of the currently reported experiences of emicizumab-treated people with hemophilia A without FVIII inhibitors undergoing major surgery, helping to support the future management decisions of emicizumab-treated people with hemophilia A during surgery."

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

Application value of thromboelastography in assessing coagulation function in children with severe hemophilia A after emicizumab therapy: a single-center study (PubMed, Zhongguo Dang Dai Er Ke Za Zhi) - "The bleeding condition of children with severe HA can be effectively controlled after EMI treatment. Routine APTT testing cannot reflect true coagulation function, whereas TEG testing is clinically valuable in assessing the coagulation function of children with severe HA undergoing EMI treatment."

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

POCUS: Hemostatic Potential and Joint Health in Patients With Severe Hemophilia A on Novel Replacement Therapies (clinicaltrials.gov) - P4 | N=28 | Recruiting | Sponsor: University of Texas Southwestern Medical Center | Trial completion date: Jul 2025 ➔ Jul 2027 | Trial primary completion date: Jul 2025 ➔ Jul 2027

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

A retrospective single-center study on the effectiveness and tolerability of emicizumab in patients with hemophilia a with and without inhibitors. (PubMed, Expert Rev Hematol) - "Tolerability was favorable with only one patient reportinga non-significant AE. Emicizumab prophylaxis effectively reducesABRs and enhances joint health in severe HA patients, irrespective of inhibitorpresence, providing a convenient, well-tolerated alternative to FVIII therapy."

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