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

Structural Enablers of Rare Disease Treatment Coverage in Latin America and the Caribbean: Lessons from Emicizumab. (PubMed, J Mark Access Health Policy) - "Countries with stronger structural characteristics-particularly in resource generation, service delivery, and governance-tended to achieve broader and more sustained coverage, although institutional capacity alone was not sufficient in all cases. Our results emphasize the need to strengthen health governance and adopt specific policies for rare diseases in the region."

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

Milds Matter-Feasibility of Emicizumab Prophylaxis in Mild Hemophilia. (PubMed, Haemophilia) - No abstract available

Journal • Hematological Disorders • Hemophilia • Rare Diseases

SUCCESSFUL HEMODIALYSIS INITIATION IN ACQUIRED HEMOPHILIA A MANAGED WITH SUSOCTOCOG ALFA AND EMICIZUMAB (ISN-WCN 2026) - "Hemostatic therapy was prioritized to safely continue HD.Results Initial hemostasis with recombinant activated factor VII (rFVIIa; eptacog alfa) was insufficient. To our knowledge, this is the first report documenting HD initiation and maintenance in AHA using the sequential susoctocog alfa followed by emicizumab. This case highlights an infection-conscious practical strategy for frail patients-prioritizing rapid procedural hemostasis with rpFVIII when rFVIIa response is inadequate, followed by emicizumab prophylaxis to sustain bleed prevention and minimize steroid or IST exposure."

Glomerulonephritis • Hematological Disorders • Hemophilia • Hemophilia A • Infectious Disease • Nephrology • Pneumonia • Pulmonary Disease • Rare Diseases • Renal Disease • Respiratory Diseases

Non-clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B. (PubMed, Cochrane Database Syst Rev) - "Evidence from RCTs shows that prophylaxis using non-clotting factor therapies compared with on-demand treatment may reduce bleeding events, increase the percentage of individuals with zero bleeds, increase the incidence of non-serious adverse events, and improve HRQoL. Comparative assessments with other prophylaxis regimens, assessment of long-term joint outcomes, and assessment of economic outcomes will improve evidence-based decision-making for the use of these therapies in bleed prevention."

Clinical • Journal • Review • Cystic Fibrosis • Genetic Disorders • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Immunology • Oncology • Pain • Pulmonary Disease • Rare Diseases • Respiratory Diseases

Clinical Experience with Emicizumab and Rituximab as First-Line Treatment in a Case Series of Acquired Hemophilia A. (PubMed, Hematol Rep) - " Our case series suggests that early initiation of emicizumab for patients with AHA is effective in preventing severe bleeding and subsequent immobility, and it can be combined with rituximab-containing IST to achieve remission, potentially with fewer adverse effects than standard IST. Further studies are warranted to establish the optimal treatment protocol involving emicizumab and IST for AHA."

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

Impact of Emicizumab on Coagulation Potential in Plasma From Healthy Women and Carriers of Haemophilia A During Late Pregnancy. (PubMed, Haemophilia) - "These findings ex vivo indicated that Emi at clinically relevant concentrations does not meaningfully enhance the hypercoagulable state characteristic of late pregnancy. In vivo studies are required to confirm the safety of maternal prophylaxis."

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

WILL-EMI: A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebrand Disease (clinicaltrials.gov) - P3 | N=75 | Recruiting | Sponsor: Hoffmann-La Roche

Trial initiation date • Hemophilia

Evaluating pen-injector handling and PROs in patients switching from emicizumab to Mim8 in FRONTIER5 (THSNA 2026) - P3 | "The HDHPA questionnaire results found the Mim8 pen-injector easy to use with an overall strong user preference over their previous injection system. At Week 26 of Mim8 prophylaxis, following a direct switch from emicizumab with no washout, there were no unexpected findings related to PROs. Previously presented at the International Society on Thrombosis and Haemostasis (ISTH) Congress, Washington, DC, USA, 21-25 June 2025."

Clinical • Cardiovascular • Hematological Disorders • Hemophilia • Hemophilia A • Musculoskeletal Diseases • Musculoskeletal Pain • Orthopedics • Rare Diseases • Thrombosis

Maintaining Factor VIII Tolerance in Patients with Severe Hemophilia A on Emicizumab: A Four-Patient Case Series (THSNA 2026) - "142: p. 5476-5477. 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

Insights into effective immune tolerance induction in hemophilia A patients with inhibitors:New findings from the interim analysis of the MOTIVATE study (THSNA 2026) - P | "Group 1 (standard ITI) reached negative inhibitors faster than Group 2 (ITI with emicizumab) for all patients and for high-responders on primary ITI. This was likely due to higher and more frequent FVIII dosing in Group 1. BEs were low and occurred in all groups, with most joint BEs occurring in Group 3."

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

Laboratory testing for patients on emicizumab: Case-based discussion and common challenges in monitoring (THSNA 2026) - "Individualizing emicizumab treatment based on thrombin generation assays requires optimization. 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

NXT007 prophylaxis in people with hemophilia A with or without FVIII inhibitors: A global Phase I/II multiple-ascending-dose study (THSNA 2026) - P1/2 | "Introduction: NXT007 is a next-generation bispecific antibody, based on emicizumab, that mimics the cofactor function of activated factor (F)VIII in people with hemophilia A (PwHA). NXT007 had a favorable safety profile at all doses. Overall treated bleed ABR was low. The presence of ADAs did not impact pharmacokinetics."

Clinical • P1/2 data • Hematological Disorders • Hemophilia • Hemophilia A • Mood Disorders • Rare Diseases

Exploratory Analysis from HAVEN 1–4 to Further Contextualize Injection-Site Reactions Among People with Hemophilia A Receiving Emicizumab (THSNA 2026) - P3 | "Overall, <1% of emicizumab injections were associated with an ISR and the proportion of participants experiencing ISRs declined over time; however, changes in visit schedules beyond the main study period may introduce a reporting bias. These results expand our understanding of the tolerability of emicizumab injections. 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."

Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Emicizumab for Severe von Willebrand Disease (VWD): the EmiVWD Study Enrollment 2026 (THSNA 2026) - P1 | "This ongoing pilot study is the first prospective investigation of emicizumab in patients with severe VWD. This study will shed light on feasibility, safety and potential efficacy of emicizumab prophylaxis in this patient population. 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."

Cardiovascular • Hematological Disorders • Hemophilia • Hemophilia A • Hepatology • Nephrology • Rare Diseases

Anticoagulation in patients with inherited bleeding disorders (THSNA 2026) - "In severe hemophilia with inhibitors, the risk of uncontrollable bleeding often precludes full‑dose anticoagulation, although emicizumab may create new options in select patients... Anticoagulation in patients with inherited bleeding disorders is complex but feasible when guided by a structured assessment of thrombotic risk, bleeding phenotype, and achievable factor levels. Bleeding disorders do not obviate the need for stroke and VTE prevention, particularly in older patients with multiple cardiovascular comorbidities. DOACs, combined with tailored factor prophylaxis targeting trough levels ≥20–30%, represent a preferred strategy for many patients, while catheter‑based AF interventions and left atrial appendage occlusion offer alternatives for those with prohibitive long‑term bleeding risk."

Clinical • Acute Coronary Syndrome • Atrial Fibrillation • Cardiovascular • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases • Venous Thromboembolism

Evaluating the Safety and Tolerability of Switching from Emicizumab to Fitusiran Prophylaxis in Males Aged ≥12 Years with Severe Hemophilia A, with and without Inhibitors: SWITCH Study (THSNA 2026) - P4 | "Study recruitment is ongoing. 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 • Hemophilia B • Rare Diseases

FRONTIER5 direct switch study: Safety of initiating Mim8 prophylaxis without washout of emicizumab (THSNA 2026) - P3 | "A direct switch from emicizumab to Mim8 prophylaxis treatment, without a washout period, in adolescents and adults with HA with/without inhibitors was well tolerated, and no safety concerns were observed. Previously presented at the International Society on Thrombosis and Haemostasis (ISTH) Congress, Washington, DC, USA, 21-25 June 2025. 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 • Immunology • Rare Diseases • Thrombosis

Surgery in the Emicizumab Era: Real-World Evidence from the Mid-Atlantic Region (THSNA 2026) - "The MAR Emi Surgery and Procedure Project underscores the value of real-world data in guiding perioperative management for Emi-treated patients. While bleeding risks remain, especially in certain procedures, careful use of factor concentrate and antifibrinolytic therapies appears effective. Continued data collection and analysis are essential to refine best practices and support clinical decision-making in this evolving therapeutic landscape and may have implications for other non-factor therapeutics."

Clinical • HEOR • Real-world • Real-world evidence • Surgery • Cardiovascular • Hematological Disorders • Hemophilia • Hemophilia A • Orthopedics • Rare Diseases • Thrombosis

Bleeding rates, healthcare utilization, and costs among patients with hemophilia a without inhibitors treated with concomitant octocog alfa or extended half-life factor VIII while on emicizumab prophylaxis. (PubMed, J Med Econ) - "Findings are limited by the retrospective design and reliance on claims data, which may omit home-treated bleeds, and misrepresent FVIII utilization due to billing complexities and assumptions about dispensed product use. While the choice of concomitant FVIII does not substantially influence ABRb or bleed-related HCRU, significantly lower pharmacy costs with octocog alfa compared with EHL agents highlight its potential cost saving and support its consideration as a preferred on-demand treatment option in patients with HA without inhibitors on emicizumab prophylaxis."

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

In Vitro Spiking Comparison of Coagulation Potential Between Emicizumab and Mim8 in Whole Blood and Plasma From a Single Patient With Severe Hemophilia A Receiving FVIII Prophylaxis and Warfarin. (PubMed, Haemophilia) - No abstract available

Journal • Preclinical • 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

Monochorionic diamniotic twin brothers with severe hemophilia A: a case report. (PubMed, Front Pediatr) - "Emicizumab was started as the primary anti-hemorrhagic prophylaxis, with good response and no major bleeding events in the first year of therapy...Targeted coagulation profiling and factor assays are mission-critical for newborns from twin pregnancies when a hematological disorder is suspected. Coagulation factor assays are essential to confirm the diagnosis of hemophilia A. An early diagnosis of hemophilia is crucial for the timely initiation of an appropriate management plan."

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

Role of Bispecific Antibody in Hemophilia (ICKSH 2026) - "This represented not only a remarkable scientific achievement but also a poten tial solution for effective prophylactic therapy in people with hemophilia who have developed anti -FVIII alloantibodies (PWHI), since the activity of the therapeutic molecule is not affected by anti -FVIII inhibitors due to the lack of homology between the sequences of emicizumab and FVIII. A first -in-human study of NXT007, a next -generation, activated factor VIII -mimetic bispecific antibody, in healthy participants . J Thromb Haemost 2025; 23: 3098 -3110 ."

Hematological Disorders • Hemophilia • Hemophilia A • Immunology • Inflammation • Rare Diseases

Recent Advances in the Diagnosis and Management of Acquired Hemophilia A (ICKSH 2026) - "Emicizumab, a bispecific factor VIII mimetic antibody, has emerged as a promising adjunctive or alternative strategy for bleeding prevention in selected patients, particularly those at high risk for recurrent bleeding or treatment -related toxicity...In parallel, immunosuppressive strategies are evolving toward more individualized, risk -adapted approaches that balance inhibitor eradication with the risk of infection and other treatment -related complications. Bridging the gap between guideline recommendations and real -world practice through early recognition, appropriate selection of hemostatic agents, and patient -centered treatment strategies remains essential to improving outcomes in acquired hemophilia A."

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

Comparative Efficacy and Safety of Non-Clotting Factor Prophylaxis Versus. on-Demand Therapy in Hemophilia: A Meta-Analysis of Randomized Controlled Trials. (PubMed, Clin Appl Thromb Hemost) - "Prophylaxis increased the likelihood of achieving zero treated bleeds [RR = 4.11; 95% CI: (1.48%, 11.45%), I2 = 88.5%, p < 0.0001]. An exploratory network meta-analysis comparing fitusiran, emicizumab, and concizumab reported no statistically significant difference in the ABR for all treated bleeds.ConclusionCompared to on-demand therapy, non-factor prophylactic therapies significantly reduce bleeding episodes, improve quality of life, and increase the likelihood of zero bleeds in patients with hemophilia."

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