Advate (octocog alfa) / Takeda - LARVOL DELTA

Impact of Heat-Stable Factor VIII Mimetics and Replacement Therapies on Inhibitor Titer using a Modified Nijmegen-Bethesda Test Across Bovine Chromogenic FVIII Assays (ISTH 2026) - "Samples were supplemented with Mim8 (0, 5, 8, 10, and 20 µg/mL), emicizumab (0, 25, 50, 75, and 150 µg/mL) or rFVIII products (Altuviiio and Advate) at 0.05, 0.2, 0.8, and 1.0 IU/mL. Comparable trends were observed between the new bovine CRYOcheck chromogenic FVIII assay and both the Coatest and Siemens assays in inhibitor testing. DOI*10.1016/j.rpth.2026.104826"

Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

The coagulation potential in the copresence of Mim8 and warfarin (ISTH 2026) - "We previously reported that prolonged PT-INR led to a reduction in the coagulation potential of emicizumab (Kajimoto et al...Methods We used blood samples obtained from a patient with severe hemophilia A who had been receiving a thrice-weekly prophylactic regimen of standard factor VIII concentrate (Advate ® )...These results suggest Mim8 alone may provide adequate hemostasis in people with hemophilia A receiving warfarin therapy without FVIII supplementation. DOI*10.1016/j.rpth.2026.104345"

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

Potent inhibition of clot growth by low dose of oral factor Xa inhibitors: factor VIII as guide for personalized treatment and as antidote. (ISTH 2026) - "Aims To study the effects of oral factor Xa inhibitors (Xabans) on clot growth Methods We documented a potent inhibition of growth in plasma spiked with Xabans (Rivaroxaban, Apixaban and Edoxaban)...In experiments with variation in Factor VIII, we showed the level to be a determinant of clot growth from absent (hemophilia A) to at least 200%, obtained by spiking plasma with FVIII (Advate)...In the same line of arguing we propose that treatment with a Factor VIII concentrate is an easily accessible antidote strategy in Xaban- treated patients that bleed. DOI*10.1016/j.rpth.2026.106525"

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

Use of Recombinant von Willebrand Factor (Vonicog alfa) for Bleed Treatment in Children under 12 – a single centre experience (ISTH 2026) - "Bleeding episodes either due to trauma or surgery can be treated with a combination of tranexamic acid, desmopressin or von Willebrand factor (VWF) replacement...All children with a baseline FVIII:c <30iu/dL were given a single dose of recombinant FVIII (Octocog alfa) alongside the initial dose of Vonicog alfa...Table or Figure Upload (1) Patient demographics and baseline von Willebrand parameters Page 2 Table or Figure Upload (2) Indication, dosing and outcomes for patients treated with Vonicog alfa. Page 3 DOI*10.1016/j.rpth.2026.105059"

Clinical • Hematological Disorders • Hemophilia • Immunology

Preclinical Safety and Thrombotic Risk Assessment of the siRNA TGM-148 for Bleeding Disorders (ISTH 2026) - "mTGM- 148 was co-administered with standard factor replacement therapies (Advate, BeneFIX) or bypassing agents (NovoSeven, FEIBA), with measurement of D-dimer levels. TGM-148 is advancing toward CTA submission in 2026. DOI*10.1016/j.rpth.2026.104873"

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

Did Efanesoctocog alfa meet our expectations in the prophylaxis of patients with hemophilia A? (ISTH 2026) - "During adolescence, he was treated with octocog alfa and subsequently switched to damoctocog alfa pegol prophylaxis at a regimen of 3000 IU intravenously twice weekly. One patient spontaneously bled into the right ankle after the 5th dose of Efa, absence of individual pharmacokinetic assessment and therefore applies prophylaxis twice a week. Table or Figure Upload (1) Table 1 Baseline characteristics of patients with severe hemophilia A switched to Efa Page 2 Table or Figure Upload (2) Figure 1 FVIII activity over time following switch to Efa in a patient with severe hemophilia A DOI*10.1016/j.rpth.2026.104909"

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

Optimization of pre analytical heat treatment in samples containing Extended or Ultra-Long Half-Life Factor VIII concentrate (ISTH 2026) - "Extended half-life (EHL) FVIII: efmoroctocog alfa and ultra-long half-life (ULHL) FVIII: efanesoctocog alfa were spiked into FVIII deficient plasma at serial dilutions. Post-treatment patient samples containing SHL: octocog alfa, EHL-FVIII or ULHL-FVIII were evaluated...Although, no differences were seen using a heat-block or water-bath for SHL-FVIII, longer PHT times were needed for the heat-block for EHL & ULHL-FVIII concentrates. Laboratories should verify PHT conditions based on FVIII concentrates used in clinical care prior to inhibitor testing DOI*10.1016/j.rpth.2026.104341"

Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases

Low Inhibitor Rate and Assessment of Binding Antibodies in Previously Untreated Patients with Severe Hemophilia A Treated with Rurioctocog Alfa Pegol: Final Results from a Prospective Phase 3 Study (ISTH 2026) - P3 | "Methods Eligibility criteria encompassed severe HA (FVIII <1%), age <6 years, and <3 exposure days (EDs) to rAHF- PEG, octocog alfa, or plasma transfusion. Participants with specific binding antibodies. Page 2 DOI*10.1016/j.rpth.2026.104294"

Clinical • P3 data • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Individual Comparative PK Evaluation of Single-dose Octocog Alfa, Rurioctocog Alfa Pegol, and Efanesoctocog Alfa in Adults with Severe Hemophilia A. (PubMed, TH Open) - No abstract available

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

Cost comparison of efanesoctocog alfa vs conventional factor VIII therapies for major surgeries in patients with severe hemophilia A. (PubMed, Blood Vessel Thromb Hemost) - "The median total factor consumption per major surgery involving octocog alfa (SHL), rurioctocog alfa pegol (EHL), efmoroctocog alfa (EHL), and efanesoctocog alfa was 910, 629, 493, and 163 IU/kg, respectively. Efanesoctocog alfa resulted in $81 022 to $38 947 savings vs SHL/EHL therapies per surgery. The perioperative management with efanesoctocog alfa was estimated to be markedly more economical than that with SHL and EHL therapies due to its high-sustained factor activity."

HEOR • Journal • Reimbursement • US reimbursement • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Intrinsic activated thrombin generation for treatment efficacy and monitoring of octocog alfa and emicizumab in severe hemophilia A. (PubMed, Res Pract Thromb Haemost) - "In plasma from SHA patients, PPP Reagent INT demonstrated sixfold and fourfold greater sensitivity to emicizumab level changes compared with TF-based reagents. Intrinsic activated TGA using PPP Reagent INT provides enhanced sensitivity for monitoring SHA patients on both FVIII and non-FVIII replacement products compared with TF-based triggers, representing a promising tool for personalized monitoring."

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

Post Approval Observational Study to Learn More About How Safe Octocog Alfa is and How Well it Works in Patients With Severe Hemophilia A in India (clinicaltrials.gov) - P=N/A | N=33 | Not yet recruiting | Sponsor: Bayer | Trial completion date: Dec 2026 ➔ Sep 2027 | Initiation date: May 2026 ➔ Sep 2026 | Trial primary completion date: Dec 2026 ➔ Sep 2027

Real-world evidence • Trial completion date • Trial initiation date • Trial primary completion date • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD) (clinicaltrials.gov) - P3 | N=47 | Completed | Sponsor: Baxalta now part of Shire | Recruiting ➔ Completed | N=31 ➔ 47

Enrollment change • Trial completion • Hemophilia

Inhibitor development according to FVIII concentrates in PUPs with severe hemophilia A: update from the PedNet registry. (PubMed, J Thromb Haemost) - "Inhibitor development occurred in 31.0% of PUPs, with similar incidence across SHL-rFVIII, EHL-rFVIII, and pdFVIII. Analysis of individual concentrates showed increased inhibitor risk for KogenateFS/HelixateNexGen (SHL-rFVIII) and for the first time for Fanhdi (pdFVIII). In the absence of formal PUP studies, PedNet will continue evaluating inhibitor risk according to individual FVIII concentrates."

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

Final data from the German AHEAD study: effectiveness and safety of octocog alfa and rurioctocog alfa pegol for hemophilia A in a real-world setting. (PubMed, Ther Adv Hematol) - "In patients with severe HA, octocog alfa prophylaxis was associated with reduced bleeding rates compared with on-demand therapy. Deutsches Register Klinischer Studien (German Clinical Trials Register): DRKS00000556."

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

COST-MINIMIZATION ANALYSIS OF OCTOCOG ALFA VERSUS EMICIZUMAB PROPHYLAXIS IN PATIENTS WITH HEMOPHILIA A WITHOUT INHIBITORS IN CENTRAL AMERICA, ECUADOR AND PERÚ (ISPOR 2026) - "OBJECTIVES: To estimate annual treatment acquisition costs and conduct a cost minimization comparison of drug consumption between octocog alfa (Brand Kovaltry®) and emicizumab for prophylaxis in patients with hemophilia A without inhibitors in El Salvador, Panamá, Peru, Ecuador and Guatemala A cost minimization analysis was conducted from a payer perspective over one year, comparing dosing of octocog alfa to emicizumab for Year 1 and Year 2 separately. The cost minimization analysis indicates that octocog alfa is a cost-saving alternative, offering over 65% annual cost savings compared to emicizumab in El Salvador, Panamá, Peru and Guatemala and. In the case of Ecuador, offering over 50% annual cost savings compared to emicizumab"

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

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

Post Approval Observational Study to Learn More About How Safe Octocog Alfa is and How Well it Works in Patients With Severe Hemophilia A in India (clinicaltrials.gov) - P=N/A | N=33 | Not yet recruiting | Sponsor: Bayer

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

A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD) (clinicaltrials.gov) - P3 | N=39 | Recruiting | Sponsor: Baxalta now part of Shire | Trial primary completion date: Jul 2019 ➔ May 2020 | Trial completion date: Jul 2019 ➔ May 2020

Trial completion date • Trial primary completion date • Hemophilia

A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD) (clinicaltrials.gov) - P3 | N=39 | Recruiting | Sponsor: Baxalta now part of Shire | Not yet recruiting ➔ Recruiting | Initiation date: Nov 2016 ➔ Dec 2017

Enrollment open • Trial initiation date • Hemophilia

A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD) (clinicaltrials.gov) - P3 | N=39 | Not yet recruiting | Sponsor: Baxalta US Inc.

New P3 trial • Hemophilia

A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD) (clinicaltrials.gov) - P3 | N=31 | Recruiting | Sponsor: Baxalta now part of Shire | Trial completion date: Jan 2025 ➔ Mar 2026 | Trial primary completion date: Jan 2025 ➔ Mar 2026

Trial completion date • Trial primary completion date • Hemophilia

A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD) (clinicaltrials.gov) - P3 | N=34 | Recruiting | Sponsor: Baxalta now part of Shire | Trial completion date: Mar 2024 ➔ Jan 2025 | Trial primary completion date: Mar 2024 ➔ Jan 2025

Trial completion date • Trial primary completion date • Hemophilia

A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD) (clinicaltrials.gov) - P3 | N=34 | Recruiting | Sponsor: Baxalta now part of Shire | Trial completion date: Mar 2023 ➔ Mar 2024 | Trial primary completion date: Mar 2023 ➔ Mar 2024

Trial completion date • Trial primary completion date • Hemophilia

A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD) (clinicaltrials.gov) - P3 | N=34 | Recruiting | Sponsor: Baxalta now part of Shire | Trial completion date: Dec 2022 ➔ Mar 2023 | Trial primary completion date: Jun 2022 ➔ Mar 2023

Trial completion date • Trial primary completion date • Hemophilia