Obizur (susoctocog alfa) / Takeda, Ipsen - LARVOL DELTA

Targeting early immunologic decline in acquired hemophilia A: A case for prophylactic rituximab (ASH 2025) - "We present the case of an 81-year-old man with idiopathic AHA and a mechanical aortic valve on chronic coumadin...Following treatment with triple immunosuppressive therapy including prednisone, rituximab, and cyclophosphamide, he achieved remission...Beyond its clinical impact, this approach may also offer significant cost savings by reducing the need for costly agents such as recombinant FVIII (obizur) or emicizumab. Further investigation is warranted to define relapse thresholds, evaluate safety, and determine the broader applicability of prophylactic immunosuppression in AHA patients."

Clinical • Cardiovascular • Hematological Disorders • Hemophilia • Hemophilia A • Immunology • Rare Diseases • Thrombocytopenic Purpura

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

Susoctocog alfa for major surgery in acquired Hemophilia A (ASH 2025) - "As rescue therapy, rpFVIII was also effective in most life-threatening situations. Thesefindings may inform future guidelines and best practices for managing surgical emergencies in patientswith AHA."

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

Distinct Co-methylation Patterns in African and European Populations and Their Genetic Associations. (PubMed, Genomics Proteomics Bioinformatics) - "Between individuals of European and African ancestry, 101 CMRs exhibited population-specific DNAm patterns (Pop-CMRs), including 91 Pop-CMRs not found in previous investigations, which spanned genes (e.g., CCDC42, GYPE, MAP3K20, and OBI1) related to diseases (e.g., malaria infection and diabetes) with different prevalence and incidence rates between populations...Additionally, subsets of Pop-CMRs could be applicable in East Asian populations and peripheral blood-based tissues. This study provides insights into DNAm differences across the genome between populations and explores their associations with genetic variants and biological relevance, advancing our understanding of epigenetic roles in population specificity."

Journal • Diabetes • Infectious Disease • Malaria • Metabolic Disorders • CDC42

Concomitant Acquired Hemophilia A and Acquired Von Willebrand Syndrome from Distinctive Autoantibodies: Case Report. (PubMed, Hematol Rep) - "The patient was started on immunosuppression with prednisone, cyclophosphamide, and intravenous immunoglobulin, alongside recombinant porcine FVIII replacement, susoctocog alfa. The patient had two further relapses; the most recent was treated with Rituximab, following which remission has been maintained. Given the potential therapeutic implications, VWF testing should be considered as part of the diagnostic workup for AHA."

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

Pharmacokinetic strategies for achieving precise factor VIII control with susoctocog alfa in acquired hemophilia A. (PubMed, J Thromb Haemost) - "PK estimates from limited sampling effectively guided rpFVIII bolus and continuous infusion dosing. Continuous infusion provided steady FVIII levels and may offer a practical alternative to bolus dosing."

Journal • PK/PD data • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

TAK-672-4002: A Study of OBIZUR in Adults With Acquired Hemophilia A (AHA) in South Korea (clinicaltrials.gov) - P=N/A | N=9 | Recruiting | Sponsor: Takeda | Not yet recruiting ➔ Recruiting

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

Acquired Hemophilia A: insights from a 6-year experience in Liguria Italy (ISTH 2025) - "Among available treatments, susoctocog α, when administered at a loading dose of 100 U/kg, demonstrated high efficacy in controlling bleeding...Most diagnoses (21 out of 22) were made in central and western Liguria, highlighting a significant geographic disparity (Table 1 and Figure 1). Table or Figure Upload"

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

TAK-672-4002: A Study of OBIZUR in Adults With Acquired Hemophilia A (AHA) in South Korea (clinicaltrials.gov) - P=N/A | N=9 | Not yet recruiting | Sponsor: Takeda | Initiation date: Sep 2024 ➔ Dec 2024

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

Genetic Foundations of Nellore Traits: A Gene Prioritization and Functional Analyses of Genome-Wide Association Study Results. (PubMed, Genes (Basel)) - "The prioritized candidate genes were GFRA4, RFWD3, SERTAD2, KIZ, REM2, and ANKRD34B for reproduction; RFWD3, TMEM120A, MIEF2, FOXRED2, DUSP29, CARHSP1, OBI1, JOSD1, NOP58, and LOXL1-AS1 for the carcass; ANKRD34B and JOSD1 for growth traits; and no genes were prioritized for morphological traits. The functional analysis pinpointed the following genes: KIZ (plays a crucial role in spindle organization, which is essential in forming a robust mitotic centrosome), DUSP29 (involved in muscle cell differentiation), and JOSD1 (involved in protein deubiquitination, thereby improving growth). The enrichment of the functional candidate genes identified in this study highlights that these genes play an important role in the expression of reproduction, carcass, and growth traits in Nellore cattle."

Journal • Targeted Protein Degradation • NOP58 • RFWD3

Cross-reacting antiporcine factor VIII inhibitors in patients with acquired hemophilia A. (PubMed, Res Pract Thromb Haemost) - "•Recombinant porcine factor VIII (rpFVIII) is a hemostatic treatment for bleeding in acquired hemophilia A.•The efficacy of rpFVIII can be negatively affected by cross-reacting anti-human FVIII antibodies.•In this cohort, 52% of patients with acquired hemophilia A had baseline cross-reacting antibodies.•Cross-reacting antibodies were associated with decreased FVIII activity levels post-rpFVIII infusion."

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

Acquired haemophilia: Update in 2024 (PubMed, Rev Med Interne) - "Eradication of inhibitor should be established as soon as the diagnosis is confirmed with steroid alone often associated with cytotoxic agents or rituximab, depending on FVIII activity and inhibitor titer. The purpose of this review is to summarize the epidemiology, etiopathogenesis, diagnosis, treatment of AHA and discuss current recommendations."

Journal • Review • Hematological Disorders • Hemophilia • Immunology • Oncology • Rare Diseases

Real-world use of recombinant porcine sequence factor VIII in the treatment of acquired hemophilia A: EU PASS. (PubMed, Ther Adv Hematol) - P=N/A, P2/3 | "Recombinant porcine factor VIII (rpFVIII; susoctocog alfa) is indicated for the treatment of bleeding events (BEs) in adults with acquired hemophilia A (AHA)...Results from this real-world study support the use of rpFVIII for AHA, aligning with findings from the clinical trial of rpFVIII (NCT01178294) in the treatment of BEs in adults with AHA. EUPAS16055; NCT03199794."

Journal • Preclinical • Real-world • Real-world evidence • Cardiovascular • Hematological Disorders • Hemophilia • Immunology • Rare Diseases

Efficacy and safety of recombinant porcine factor VIII in Japanese patients with acquired hemophilia A. (PubMed, Int J Hematol) - P2/3 | "This phase II/III open-label study evaluated the safety and efficacy of recombinant porcine factor VIII (rpFVIII, susoctocog alfa) in adults with AHA and severe bleeding episodes in Japan (NCT04580407)...This study supports the use of rpFVIII as a novel therapy in the clinical management of patients with AHA in Japan. rpFVIII was approved for treating bleeding episodes in adults with AHA in Japan in 2024."

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

A Study of OBIZUR in Adults With Acquired Hemophilia A (AHA) in South Korea (clinicaltrials.gov) - P=N/A | N=9 | Not yet recruiting | Sponsor: Takeda

New trial • Hematological Disorders • Hemophilia • Rare Diseases

A Survey of Susoctocog Alfa (Genetical Recombination) in Participants With Acquired Haemophilia A (clinicaltrials.gov) - P=N/A | N=25 | Recruiting | Sponsor: Takeda

New trial • Hematological Disorders • Hemophilia • Rare Diseases

Identification of FVIII inhibitors by anti-FVIII ELISA assay and Bethesda assays in tandem to identify anti-Factor VIII inhibitors (ISTH 2024) - "Bovine-based reagents are used to assess for inhibitors in the presence of emicizumab. Porcine-based Bethesda assay measures FVIII inhibition with porcine FVIII therapy (e.g. obizur)... 218 anti-FVIII results were reviewed. 91% of patients showed correlation between ELISA and Bethesda assays whereas 9% did not. 5/218 (2%) patients had positive Nijmegen Bethesda with negative ELISA."

Hematological Disorders • Hemophilia • Rare Diseases

Evaluation of Factor VIII Population Pharmacokinetics and Exposure-Response to Support Dosing of Recombinant Porcine Factor VIII in Patients with Acquired Hemophilia A (ISTH 2024) - P=N/A, P2, P2/3 | "Background: Recombinant porcine factor VIII (rpFVIII; susoctocog alfa) is indicated for the treatment of bleeding episodes (BEs) in acquired hemophilia A (AHA)... A 2-compartment model with linear elimination and baseline (endogenous) levels of FVIII described FVIII PK. A typical adult patient (70 kg, 65 years, with positive hFVIII and negative pFVIII inhibitors at baseline) had 10.5 dL/h FVIII clearance, 72.8 dL volume of distribution (Vc), and 6.7 h elimination half-life. Patients with bleeding and positive baseline pFVIII inhibitors (n=13) had 10-fold and 2-fold higher median FVIII clearance and Vc, respectively, than those with negative baseline pFVIII inhibitors (n=19; Table 2); patients positive for baseline hFVIII inhibitors had 3.5-fold and 2-fold higher median FVIII clearance and Vc, respectively, than those negative for baseline hFVIII inhibitors (Table 2)."

PK/PD data • Preclinical • Hematological Disorders • Hemophilia • Rare Diseases

BLEEDING DUET: A CASE OF SIMULTANEOUS ACQUIRED FACTOR VIII DEFICIENCY AND ACQUIRED VON WILLEBRAND SYNDROME (THSNA 2024) - "The patient was started on immunosuppression with oral prednisone, IV cyclophosphamide and IVIG. She also received factor VIII replacement, Obizur... To the authors knowledge, this is the first documented instance of two acquired bleeding disorders that occurred simultaneously in a single individual. Here we present the diagnostic steps taken to identify and then prove the presence of two distinct acquired autoimmune mediated bleeding entities and documented positive longitudinal response to immunosuppression."

Clinical • Cardiovascular • Colorectal Cancer • Gastroenterology • Gastrointestinal Disorder • Hematological Disorders • Hematological Malignancies • Hemophilia • Immunology • Multiple Myeloma • Oncology • Pain • Rheumatology • Solid Tumor • Targeted Protein Degradation

Measurement of recombinant porcine factor VIII in patients with congenital haemophilia A and inhibitors in the presence of emicizumab. (PubMed, Haemophilia) - "This study indicates that rpFVIII can be measured in the presence of emicizumab with a bCSA. A calibration curve for the measurement of rpFVIII with bCSA should be established."

Journal • Preclinical • Hematological Disorders • Hemophilia • Rare Diseases

Recombinant porcine factor VIII in patients with congenital haemophilia A with inhibitors undergoing surgery: Phase 3, single-arm, open-label study. (PubMed, Haemophilia) - P3 | "Good haemostasis was achieved with rpFVIII during the immediate perioperative period. The study was terminated early because the study sponsor and health authorities determined that the risk of anamnestic reactions outweighs the benefits in this study population."

Journal • P3 data • Preclinical • Surgery • Hematological Disorders • Hemophilia • Rare Diseases

Diagnosis and laboratory monitoring of hemophilia A. (PubMed, Hematology Am Soc Hematol Educ Program) - "A Bethesda or modified Bethesda assay should be performed to quantify any inhibitor, and if susoctocog alfa (rpFVIII) is available, then an assay for cross-reacting antibodies should also be performed. Measurement of rpFVIII requires a 1-stage clotting assay, and measurement of patient FVIII while on emicizumab requires a chromogenic assay that does not contain human FX. Close communication is required between the treating clinicians and the laboratory to ensure that the correct tests are performed while patients are receiving treatments."

Journal • Hematological Disorders • Hemophilia • Immunology • Inflammatory Arthritis • Rare Diseases

Agreement between one stage and chromogenic assays in samples from patients receiving recombinant porcine FVIII (Obizur, Susoctocog-alfa). (PubMed, Int J Lab Hematol) - "The relationship between OSA and CSA was not consistent between method or patient. Previously there has been reports of underestimation by CSA in in vitro spiked samples. Investigation into concentration of phospholipids in the APTT reagents may explain some of these variations."

Journal • Preclinical • Hematological Disorders • Hemophilia • Rare Diseases

Real-world safety and effectiveness of recombinant porcine sequence factor VIII in acquired haemophilia A: A non-interventional, post-authorization safety study. (PubMed, Haemophilia) - P=N/A | "In this real-world study of rpFVIII for AHA, no new safety signals were identified compared with previous clinical trial findings. Eighty percent of bleeds resolved with rpFVIII treatment."

Journal • Preclinical • Real-world • Real-world evidence • Cardiovascular • Hematological Disorders • Hemophilia • Rare Diseases

OBINUTUZUMAB INDUCTION AND MAINTENANCE IN PATIENTS WITH RELAPSED/REFRACTORY WALDENSTRÖM MACROGLOBULINAEMIA (EHA 2023) - P2 | "Background: Chemoimmunotherapy based on rituximab remains the cornerstone of treating symptomatic patients with Waldenström macroglobulinaemia...Aims: The OBI-1 study (EUDRACT, 2016-005053-20) evaluated the efficacy and safety of obinutuzumab induction and maintenance in patients with relapsed or refractory Waldenström macroglobulinaemia... Our findings suggest that obinutuzumab induction and maintenance may have significant clinical activity in relapsed and refractory Waldenström macroglobulinaemia. Investigated chemotherapy-free induction and maintenance offered prolonged survival with an acceptable toxicity profile that warrants further investigation of obinutuzumab in Waldenström macroglobulinaemia. Induction, relapsed/refractory, Waldenstrom's macroglobulinemia, Maintenance"

Clinical • IO biomarker • Congestive Heart Failure • Heart Failure • Hematological Disorders • Hematological Malignancies • Indolent Lymphoma • Infectious Disease • Lymphoma • Lymphoplasmacytic Lymphoma • Multiple Myeloma • Neutropenia • Non-Hodgkin’s Lymphoma • Novel Coronavirus Disease • Oncology • Thrombocytopenia • Waldenstrom Macroglobulinemia