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

Emicizumab for preventing intracranial hemorrhage in infants with severe hemophilia A: a cost-effectiveness analysis. (PubMed, Blood Adv) - "Scenario analysis examining prophylaxis with low-dose emicizumab resulted in an ICER of $19,600/QALY (95% CI 12,000-29,000). Probabilistic sensitivity analyses showed that standard-dose emicizumab is the cost-effective strategy in 100%, 66%, and 0% of 10,000 Monte Carlo iterations at WTP thresholds of $150,000, $104,000, and $50,000/QALY, respectively, and in 100% across all WTP thresholds for low-dose emicizumab."

HEOR • Journal • Cerebral Hemorrhage • CNS Disorders • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Risk factors for joint bleeding in severe hemophilia A and B: analysis of the Community Counts longitudinal surveillance cohort. (PubMed, Blood Vessel Thromb Hemost) - "The findings of this comprehensive analysis of a large, diverse sample drawn from hemophilia treatment centers across the United States, underscore the important contributions of patient, community, and treatment factors on joint outcomes in severe hemophilia. Use of emicizumab and few missed doses independently predicted low AJBR, highlighting the interplay of access and adherence to care."

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

BCDI-XII: Emicizumab for Severe Von Willebrand Disease (VWD) and VWD/Hemophilia A (clinicaltrials.gov) - P1 | N=40 | Recruiting | Sponsor: Bleeding and Clotting Disorders Institute Peoria, Illinois | Trial completion date: Mar 2026 ➔ Jun 2027

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

W Pharmaceutical’s Hemlibra Shows Positive Results in Hemophilia A Patients’ Exercise Safety and Bleeding Prevention (Korea IT Times) - "A team led by Kagehiro Amano from the Department of Diagnostic Medicine at Tokyo Medical University conducted a study from January 2019 to October 2021, involving 129 non-inhibitor Hemophilia A patients with an average age of 29.7 years...The annual bleeding rate (ABR) among the 129 patients was a median of 0.5 bleeds per year. Bleeding incidents related to exercise were rare, with only two cases: one traumatic bleed from a soccer ball impact and another during fishing. No adverse reactions related to Hemlibra were reported."

Clinical data • Hemophilia A

Sustained Survival Benefit of Emicizumab and Postponed Immunosuppression in Acquired Hemophilia A. (PubMed, Blood Adv) - P2 | "Compared to PS-matched historic controls receiving immediate IST but no emicizumab, GTH-AHA-EMI patients had fewer fatal infections and improved OS. NCT04188639."

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

TSUBASA study: evaluation of the quality and content of daily life of people with hemophilia A without factor VIII inhibitors on prophylactic treatment with emicizumab. (PubMed, Res Pract Thromb Haemost) - "QoL outcomes remained largely unchanged across the study period. Notable improvements were observed in physical activity levels, motivation for work/school, and anxiety related to bleeding, as reported by participants and caregivers."

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

Activated prothrombin complex concentrate in patients receiving emicizumab prophylaxis: from evidence to clinical practice. (PubMed, Res Pract Thromb Haemost) - "As more NFTs become available for bleed prophylaxis, both classes of BPAs will be needed for bleed management in people with inhibitors. Thus, optimal use of BPAs is critical and will require an understanding of the factors that influence BPA selection, such as bleed type and location, clinical response, dosing and duration of use, accessibility and availability, and patient or caregiver preference."

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

Analysis of the current situation and influencing factors of home treatment for children with hemophilia in China. (PubMed, Ann Hematol) - "The implementation of home treatment for children with hemophilia requires further improvement. Particular attention should be given to younger patients. In addition, it is essential to enhance self-injection training, boost caregivers' confidence in performing venipuncture, and improve the availability of disposable sterile supplies."

Journal • Hematological Disorders • Hemophilia • Rare Diseases

A real-world pharmacovigilance analysis of the FDA adverse event reporting system database for emicizumab. (PubMed, Thromb Res) - "Our study demonstrates the importance of early monitoring and interventions to minimize AEs and alleviate patient suffering."

Adverse events • Journal • Real-world evidence • Cardiovascular • Hematological Disorders • Hemophilia • Hemophilia A • Musculoskeletal Diseases • Musculoskeletal Pain • Orthopedics • Pain • Rare Diseases • Thrombosis

Gene Therapy for Hemophilias: Opportunities and Challenges for the Clinical Laboratory. (PubMed, Clin Chem) - "Several conclusions can be drawn about GT in hemophilia A and B based on the literature and clinical practice. First, relevant gene material (FVIII/FIX) can be incorporated into AAVV, which is then infused into patients. AAVV targets liver cells and produces FVIII/IX. Second, the selection of patients who should be free from liver disease is paramount for successful therapy. Third transgene factors can be measured by one-stage clotting (OSA) or chromogenic assays (CA). Based on results from clinical trials, OSA measures transgene factor levels that are approximately 2 times higher than CA. Although expert opinions favor the OSA for transgene FIX and CA for transgene FVIII, no firm evidence is yet available on which method is better associated with clinical outcomes."

Journal • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Hepatology • Rare Diseases

Cautious use of activated prothrombin complex concentrate in patients receiving emicizumab: new evidence and clinical considerations. (PubMed, Res Pract Thromb Haemost) - No abstract available

Journal

Acquired Hemophilia due to Immune Checkpoint Inhibitors: A Case Series Introducing Emicizumab Treatment. (PubMed, Oncologist) - "Clinicians should be vigilant about ICI-induced AHA as a potentially severe irAE. Prolonged aPTT requires thorough evaluation. Upon AHA confirmation, eradication of anti-FVIII antibodies with corticosteroids, rituximab or other immunosuppressant should be attempted. Emicizumab offers advantages over traditional replacement therapy, including ease of use and a potential reduction in immunosuppressive drug requirements-an important consideration in ICI-treated patients. Further research is needed to fully understand the pathophysiology and optimize treatment strategy for AHA."

Checkpoint inhibition • Journal • Hematological Disorders • Hemophilia • Hemophilia A • Immunology • Oncology • Rare Diseases

SAFE Study: Safety of aPCC Following Emicizumab Prophylaxis (clinicaltrials.gov) - P3 | N=5 | Recruiting | Sponsor: Emory University | Trial completion date: Sep 2025 ➔ Sep 2026 | Trial primary completion date: Sep 2025 ➔ Sep 2026

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

A Study to Learn About How Changing Therapy From Emicizumab to Marstacimab Affects People With the Severe Hemophilia A. (clinicaltrials.gov) - P1 | N=15 | Recruiting | Sponsor: Pfizer | N=10 ➔ 15 | Trial completion date: May 2031 ➔ May 2027

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

Effectiveness of Octocog Alfa (BAY 81-8973) to Treat People With Haemophilia A Enrolled in the ATHNdataset, Including Under-Represented Subgroups. (PubMed, Eur J Haematol) - "PwHA treated with octocog alfa in the ATHNdataset, including important subgroups, reported a low number of bleeds; thus, octocog alfa offers an effective therapeutic option."

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

Clinical characteristics and outcomes of acquired hemophilia A before and after emicizumab approval in Japan. (PubMed, Blood Vessel Thromb Hemost) - "This study suggests that emicizumab effectively prevents bleeding in patients with AHA. Moreover, emicizumab may lead to shorter hospital stays, maintained ADLs, reduced costs, and improved prognosis in patients with AHA."

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

Progressive cephalohematoma in a neonate revealing severe hemophilia a owing to intron 22 inversion: a case report. (PubMed, Front Pediatr) - "After stabilization, the patient was transitioned to prophylactic emicizumab, which was well tolerated...It also highlights the potential benefits of early initiation of non-factor prophylaxis. Collaborative cross-disciplinary care is essential to achieve optimal outcomes in neonatal patients with bleeding disorders."

Journal • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases • F8

Discovery and development of bispecific antibodies. (PubMed, Protein Expr Purif) - "We use emicizumab as a case study to illustrate these developments...We examine the unique affinity behaviors of these resins and their impact on BsAb purification. This comprehensive review aims to provide researchers and industry professionals with a thorough understanding of the current landscape and future potential of bispecific antibodies in therapeutic applications, highlighting both technical challenges and innovative solutions in this rapidly evolving field."

Journal • Review • Oncology

NHS broadens access to haemophilia A drug Hemlibra (pharmaphorum) - "More people living with haemophilia A in the UK will be able to get access to a subcutaneous treatment, Roche/Chugai's Hemlibra, thanks to a decision by NHS England to make it an option for all patients. Hemlibra (emicizumab), the only treatment currently available for haemophilia A via the NHS that doesn't require an intravenous infusion, can now be used as prophylaxis in patients of all ages with moderate haemophilia A. Previously, it could only be used in patients at the severe end of the spectrum."

Reimbursement • Hemophilia A

Effective but Elusive: Emicizumab for Hemophilia A in India. (PubMed, Indian J Pediatr) - No abstract available

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

JW Pharmaceutical reports 25.4 billion won operating profit, fueled by prescription drug sales (Chosun Biz) - "JW Pharmaceutical announced on the 31st that its operating profit for the second quarter on a separate basis was 25.4 billion won, an increase of 103.3% compared to the same period last year. Revenue rose to 189.7 billion won, an 11.2% increase over the same period...According to the company, revenue in the second quarter from the institutional sector of prescription drugs increased by 13.3% year on year to 156.3 billion won, driving overall performance. During the same period, revenue from the dyslipidemia treatment 'Rivaroxaban' increased by 35.3% to 25.4 billion won, while the 'Rivaroxaban family' (Rivaroxaban·Rivarojet·Rivarobvi) increased by 18.4% to 47.4 billion won...Sales of the hemophilia treatment 'Hemlibra' reached 15.3 billion won, a 24% increase compared to the same period last year. The rheumatoid arthritis treatment 'Actemra' increased by 0.5% to 6.5 billion won."

Sales • Dyslipidemia • Hemophilia A • Rheumatoid Arthritis

Standardization of FVIII neutralization for functional Emicizumab Quantification in Presence of Various FVIII Sources. (PubMed, J Thromb Haemost) - No abstract available

Journal

Current trends and advances in the management of acquired Hemophilia A. (PubMed, Blood Rev) - "Bypassing agents are used for treatment, but promising new therapeutic options such as emicizumab are under evaluation. AHA remains a serious condition with high mortality from bleeding complications and adverse effects of immunosuppression. This review provides a comprehensive overview of current knowledge on AHA, including epidemiology, pathophysiology, diagnosis, prognostic factors and therapeutic strategies."

Journal • Review • Hematological Disorders • Hemophilia • Hemophilia A • Immunology • Infectious Disease • Oncology • Rare Diseases

Standardization of FVIII neutralization for functional Emicizumab Quantification in Presence of Various FVIII Sources. (PubMed, J Thromb Haemost) - No abstract available

Journal

Mathematical analysis of emicizumab: affinity-driven complex formation and lipid surface reactions. (PubMed, J Thromb Haemost) - "High concentrations of emicizumab decrease TF:VIIa activation of FX by reducing FX binding to both the lipid surface and TF:VIIa. Emicizumab enhances FIXa activation of FX on the lipid surface by preferentially binding to lipid-bound FX and subsequently to lipid-bound FIXa with an enhanced association rate due to colocalization on the lipid surface."

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