NovoSeven (eptacog alfa) / Novo Nordisk - LARVOL DELTA

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

Case Report of a Pediatric Patient with Congenital Factor VII Deficiency (4%) using Human Recombinant Factor VIIa Eptacog-Beta as Secondary Prophylaxis (THSNA 2026) - "Currently, patients with congenital factor VII deficiency only have the option of using recombinant factor VIIa Eptacog alfa. This case report suggests that the use of Recombinant Factor VIIa Eptacog beta in pediatric patients with congenital factor VII deficiency as prophylaxis should be explored considering the lack of options for this 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."

Case report • Clinical • CNS Disorders • Epilepsy • Gastrointestinal Disorder • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Musculoskeletal Diseases • Osteoarthritis • Pediatrics • Rare Diseases

FASTEST: Recombinant Factor VIIa (rFVIIa) for Hemorrhagic Stroke Trial (clinicaltrials.gov) - P3 | N=860 | Not yet recruiting | Sponsor: Joseph Broderick

New P3 trial • Cardiovascular • Cerebral Hemorrhage • Hematological Disorders

FASTEST: Recombinant Factor VIIa (rFVIIa) for Hemorrhagic Stroke Trial (clinicaltrials.gov) - P3 | N=860 | Recruiting | Sponsor: Joseph Broderick, MD | Trial completion date: Dec 2025 ➔ Jan 2028 | Trial primary completion date: Dec 2025 ➔ Jan 2028

Trial completion date • Trial primary completion date • Cardiovascular • Cerebral Hemorrhage • Hematological Disorders

FASTEST: Recombinant Factor VIIa (rFVIIa) for Hemorrhagic Stroke Trial (clinicaltrials.gov) - P3 | N=860 | Recruiting | Sponsor: Joseph Broderick, MD | Not yet recruiting ➔ Recruiting

Enrollment open • Cardiovascular • Cerebral Hemorrhage • Hematological Disorders

FASTEST: Recombinant Factor VIIa (rFVIIa) for Hemorrhagic Stroke Trial (clinicaltrials.gov) - P3 | N=860 | Not yet recruiting | Sponsor: Joseph Broderick, MD | Trial completion date: Mar 2025 ➔ Aug 2025 | Trial primary completion date: Mar 2025 ➔ Aug 2025

Trial completion date • Trial primary completion date • Cardiovascular • Cerebral Hemorrhage • Hematological Disorders

FASTEST: Recombinant Factor VIIa (rFVIIa) for Hemorrhagic Stroke Trial (clinicaltrials.gov) - P3 | N=860 | Not yet recruiting | Sponsor: Joseph Broderick, MD | Trial completion date: Aug 2025 ➔ Mar 2025 | Trial primary completion date: Aug 2025 ➔ Mar 2025

Trial completion date • Trial primary completion date • Cardiovascular • Cerebral Hemorrhage • Hematological Disorders

FASTEST: Recombinant Factor VIIa (rFVIIa) for Hemorrhagic Stroke Trial (clinicaltrials.gov) - P3 | N=860 | Not yet recruiting | Sponsor: Joseph Broderick, MD | Trial completion date: Aug 2026 ➔ Aug 2025

Trial completion date • Cardiovascular • Cerebral Hemorrhage • Hematological Disorders

FASTEST: Recombinant Factor VIIa (rFVIIa) for Hemorrhagic Stroke Trial (clinicaltrials.gov) - P3 | N=860 | Not yet recruiting | Sponsor: Joseph Broderick, MD | Trial completion date: Jan 2026 ➔ Aug 2026 | Trial primary completion date: Jan 2025 ➔ Aug 2025

Trial completion date • Trial primary completion date • Cardiovascular • Cerebral Hemorrhage • Hematological Disorders

FASTEST: Recombinant Factor VIIa (rFVIIa) for Hemorrhagic Stroke Trial (clinicaltrials.gov) - P3 | N=860 | Active, not recruiting | Sponsor: Joseph Broderick, MD | Recruiting ➔ Active, not recruiting

Enrollment closed • Cardiovascular • Cerebral Hemorrhage • Hematological Disorders

Recurrence of FVIII Inhibitor during Surgery in a Patient with Severe Hemophilia A Receiving Emicizumab Prophylaxis. (PubMed, Acta Med Okayama) - "Activated partial thromboplastin time was prolonged on postoperative day 2, prompting an immediate switch to eptacog alfa. The patient recovered without bleeding. This case underscores the necessity of anti-idiotype monoclonal antibodies for accurate monitoring."

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

Successful hemodialysis initiation in acquired hemophilia a managed with susoctocog alfa and emicizumab. (PubMed, CEN Case Rep) - "Recombinant activated factor VII (rFVIIa, eptacog alfa) failed to control the bleeding; however, recombinant porcine sequence factor VIII (rpFVIII, susoctocog alfa) proved effective, enabling the safe placement of a long-term indwelling catheter for hemodialysis. To the best of our knowledge, there have been no prior reports of AHA cases requiring hemodialysis that were successfully treated with susoctocog alfa and emicizumab. This case highlights the potential role of these agents in optimizing hemostatic strategies for AHA patients undergoing hemodialysis."

Journal • Acute Kidney Injury • Glomerulonephritis • Hematological Disorders • Hemophilia • Hemophilia A • Immunology • Infectious Disease • Nephrology • Pneumonia • Rare Diseases • Renal Disease • Respiratory Diseases

Unexplained bleeding and a hidden inhibitor: A case of late-onset acquired hemophilia A (ASH 2025) - "The patient was treated initially with recombinant porcine Factor VIII (Obizur) without sustained response, then switched to recombinant activated Factor VII (NovoSeven) for hemostasis. Immunosuppressive therapy with prednisone and rituximab was initiated... AHA should be considered in elderly patients with unexplained bleeding and isolated prolonged aPTT, even following trauma. Increased awareness and timely intervention can significantly improve outcomes in this rare but potentially fatal disorder."

Clinical • Cardiovascular • Chronic Kidney Disease • CNS Disorders • Diabetes • Diabetic Nephropathy • Hemophilia • Hemophilia A • Hypertension • Immunology • Ischemic stroke • Metabolic Disorders • Nephrology • Rare Diseases • Renal Disease • Type 2 Diabetes Mellitus

Evaluating reversal strategies for long-acting factor XI antibodies: RFVIIa®, FEIBA®, Kcentra®, novel FXI mutants, and an anti-idiotypic antibody (ASH 2025) - "BAY1831865), and theactive site binding FXIa inhibitor osocimab...With contact activation (ellagic acid or kaolin), the relative potency of thrombin inhibitionshifted: AB011 again showed the strongest inhibition on par with FXI-/- plasma, followed by gruticibart,and osocimab... FXI inhibitors produce anticoagulant effects across global coagulation assays that can bepartially or completely reversed by bypassing agents, particularly NovoSeven® and FEIBA®; however PT,TGA and viscoelastic testing also reveal the potential to generate a transient procoagulant/prothromboticstate if plasma concentrations of these commercially available agents are too high. The magnitude of theprocoagulant activity is dependent on the bypassing agent as well as the specific FXI inhibitor, suggestingthat reversal strategies should be tailored to the mechanism of the different FXI inhibitors (i.e. A2, A3, orcatalytic domain) to achieve optimal outcomes."

Hematological Disorders • Hemophilia • Rare Diseases

The Mayo Clinic enterprise experience and outcomes of perioperative utilization of recombinant factor VIIa (rFVIIa, NovoSeven®) (ASH 2025) - "Adjunctive medications to control bleeding included emicizumab, FEIBA, and rituximab.Mean estimated blood loss was 136 mL (SD 211 mL), and mean length of stay was 5.7 days (SD 6.4).Surgical risk strongly correlated with perioperative outcomes: blood loss increased from 19 mL in verylow-risk surgeries to 352 mL in very high-risk surgeries, while mean length of stay increased from 4.5days (risk 1) to 19 days (risk 4) and 11 days (risk 5).Actual rFVIIa dosing ranged from 7.2 to 7211.5 mcg/kg per dose, with a median of 67.4 mcg/kg and amean of 852.8 mcg/kg, reflecting variability in clinical practice. In conclusion, perioperative rFVIIa was most frequently used for Factor VII deficiency but was alsoadministered to patients with Factor VIII inhibitor and Glanzmann's thrombasthenia, as well as severalrare congenital and acquired bleeding disorders. Postoperative thrombotic events were rare, while ISTH-defined bleeding complications and readmissions were common. Most..."

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

Acquired Hemophilia A (AHA) associated with monoclonal gammopathy: A single institution case series (ASH 2025) - "Bleeding resolved with Novoseven, corticosteroids, and weekly Rituximab.He was transitioned to Hemlibra, completed 4 doses of Rituximab and tapered off with completeresolution of the inhibitor.Case 3:An 80-year-old female with IgG lambda MGUS presented with a spontaneous and enlarging left upperthigh hematoma with active extravasation, requiring IR guided embolization and multiple units of bloodtransfusions. This suggests that even minimal clonal plasma cell activity,characteristic of MGUS, can cause significant immune dysregulation leading to severe autoimmunecomplications like AHA.Although MGUS typically requires no treatment, persistent or refractory bleeding with sustained inhibitorlevels may warrant myeloma directed therapy targeting the plasma cell clone. Hence further awarenessabout of this novel association could also inform the treatment strategies in patients not responding toinitial measures."

Clinical • Beta-Thalassemia • Hematological Malignancies • Hemophilia • Hemophilia A • Immunology • Monoclonal Gammopathy • Multiple Myeloma • Rare Diseases • Smoldering Multiple Myeloma

In vitro comparison of eptacog beta and eptacog alfa with antithrombin lowering (simulated fitusiran) using thrombin generation assay (ASH 2025) - "These results demonstrate that in vitro both eptacog beta and eptacog alfa similarly increase TG when ATlevels are reduced to fitusiran target ranges. Eptacog alfa doses of 45µg/kg, used to treat bleeds inpatients on fitusiran, had similar TG parameters to the 75µg/kg and 125µg/kg doses of eptacog beta. Thisdata provides in vitro proof of concept supporting the use of eptacog beta for the treatment ofbreakthrough bleeds of patients on fitusiran prophylaxis."

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

Real World Experience with use of Coagulation Factor VIIa at an Academic Medical Center. (PubMed, Clin Appl Thromb Hemost) - "Our institution converted from eptacog alfa to eptacog beta as the preferred rFVIIa product. Cost avoidance was estimated at $554,400 over a 12-month period.ConclusionWe treated a small cohort of patients with eptacog beta without adverse outcomes. Other hospitals might evaluate their rFVIIa use and consider opportunities for substitution."

Journal • Real-world evidence • Retrospective data • Cardiovascular • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases • Thrombosis

Real-World Experience with Eptacog Beta for On-Label and Off-Label Indications: The Spanish Experience. (PubMed, Pharmaceuticals (Basel)) - "Preclinical studies have shown the product has a similar profile to eptacog alfa. Our experience provides further evidence of the efficacy and safety of eptacog beta for surgical prophylaxis and treatment of bleeding in patients with haemophilia A with inhibitors, but also in those with factor VII deficiency. To our knowledge, this is the first report to describe the use of eptacog beta for factor VII deficiency and acquired haemophilia."

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

Recombinant Factor VIIa Prophylaxis in 2 Brothers with Bernard-Soulier Syndrome. (PubMed, Am J Case Rep) - "Recombinant activated factor VIIa (rFVIIa, trade name: NovoSeven) has emerged as a potential alternative therapy...The older brother, aged 19, presented with persistent gingival bleeding that was unresponsive to tranexamic acid and required hospital-based interventions, including platelet transfusions...This case highlights the potential role of rFVIIa as a viable alternative to platelet transfusions in patients with recurrent bleeding. Further studies are needed to establish standardized protocols for prophylactic rFVIIa use in BSS."

Journal • Hematological Disorders • Thrombocytopenia

Cost-Savings Analysis of Fitusiran Prophylaxis: Reducing Breakthrough Bleeding Treatment Expenditure in the Kingdom of Saudi Arabia (ISPOR-EU 2025) - P3 | "For people with haemophilia (PwH) A without inhibitors, episodic treatments comprised octocog alfa, efmoroctocog alfa and rurioctocog alfa pegol for PwH A and nonacog alfa, albutrepenonacog alfa and eftrenonacog alfa for PwH B. Treatments included for PwH with inhibitors were factor VIII inhibitor bypassing activity (FEIBA) and eptacog alfa. In the KSA, fitusiran AT-DR prophylaxis may considerably reduce breakthrough bleed management costs in PwH versus CFC/BPA prophylaxis. Cost savings are predicted to be more substantial in PwH with inhibitors than in those without inhibitors."

HEOR • Hematological Disorders • Hemophilia • Rare Diseases

FASTEST Part 2: Recombinant Factor VIIa (rFVIIa) for Hemorrhagic Stroke Trial - Part 2 (clinicaltrials.gov) - P3 | N=350 | Recruiting | Sponsor: Joseph Broderick, MD

New P3 trial • Cardiovascular • Cerebral Hemorrhage • Hematological Disorders

Transplacental Transfer of Emicizumab: Experience with Emicizumab in a Pregnant Female with Severe Hemophilia Α and an Inhibitor (ASH 2023) - "She was not treated with immune tolerance induction and had remained on factor eight inhibitor bypassing activity (FEIBA) as prophylaxis until transferring to our center. She was transitioned to monthly emi prophylaxis and recombinant FVIIa (rFVIIa, NovoSeven) for breakthrough bleeding at age 26...Her labor was induced with oxytocin while on rFVIIa and tranexamic acid (TXA) support... In this highly unusual case, we demonstrate that emi crossed the placenta and is excreted in breast milk. We also demonstrate that the maternal FVIII inhibitor did not cross the placenta into the newborn. Drawing conclusions on the safety of emi despite placental and breast milk transmission and transmissibility of inhibitor based on a single case would be premature."

Hematological Disorders • Hemophilia • Hemophilia A • Postpartum Hemorrhage • Rare Diseases

The Influence of Coagulation Factor Biosimilars in Shortening the Activated Partial Thromboplastin Time in Patients with Hemophilia a (ASH 2024) - "Negative correlation was observed between PT and FVII values after adding Kogenate FS (r=-0.103, p<0.001), aPTT and FVIII values after adding Kogenate FS (r=-0.898, p<0.001), aPTT and FVIII values after adding NovoEight (r=-0.865, p<0.001), as well as aPTT and FVIII values after adding AryoSeven (r=-0.647, p<0.001).Conclusion : All the investigated drugs significantly shorten aPTT values and increase values of FVIII and FVII. In terms of efficacy, no difference exists between AryoSeven and NovoSeven, as well as between Kogenate FS and NovoEight only in altering PT."

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

Cost-Savings Assessment of Fitusiran Prophylaxis in Minimizing Breakthrough Bleeding Treatment Expenses in the United Arab Emirates (ISPOR-EU 2025) - P3 | "The episodic treatments included were efmoroctocog alfa, octocog alfa and rurioctocog alfa pegol for people with haemophilia (PwH) A without inhibitors and albutrepenonacog alfa, nonacog alfa and eftrenonacog alfa for PwH B without inhibitors...A scenario analysis examined the impact of vial sharing. Among PwH without inhibitors, fitusiran AT-DR enabled per-bleed savings ranging from UAE Dirham (AED) 4,625 (efmoroctocog alfa) to AED 11,521 (rurioctocog alfa pegol) in PwH A and from AED 8,935 (nonacog alfa) to AED 30,053 (albutrepenonacog alfa) in PwH B. In PwH with inhibitors, fitusiran AT-DR usage generated per-bleed savings of AED 71,846 (FEIBA) to AED 90,761 (eptacog alfa). In the UAE, fitusiran AT-DR prophylaxis may considerably reduce costs associated with episodic treatments for breakthrough bleeds in PwH compared with CFC/BPA prophylaxis. PwH with inhibitors might have larger cost savings than those without inhibitors."

HEOR • Hematological Disorders • Hemophilia • Rare Diseases