Kovaltry (octocog alfa) / Bayer - LARVOL DELTA

Impact of Inhibitor Development on the Cost Effectiveness of Prophylactic Treatment with Recombinant Factor VIII in Previously Untreated Patients with Severe Hemophilia A. (PubMed, Pharmacoecon Open) - "Prophylactic treatment with rAHF-PFM is likely more cost effective than BAY 81-8973 for Chinese PUPs with severe hemophilia A. This finding is primarily driven by the lower incidence of inhibitor development and fewer bleeding episodes associated with rAHF-PFM in the model."

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

Real-World Data on Hemophilia a Patients Transitioning from Bay 81-8973 to Bay 94-9027 and from Bay 14-2222 to Bay 81-8973 and then to Bay 94-9027 in the Athndataset (ASH 2023) - "Patients with Hemophilia A are transitioning more and more from standard half-life (SHL) products to extended half-life (EHL) recombinant factor VIII (rFVIII) productsObjective: To evaluate the effect of Hemophilia A patients transitioning from BAY 81-8973 (Kovaltry ®) to BAY 94-9027 (Jivi ®) and from BAY 14-2222 (Kogenate FS ®) to BAY 81-8973 and then to BAY 94-9027 in a real-world setting. The data show that hemophilia A patients who transitioned to BAY 94-9027 from BAY 81-8973, or those that transitioned from BAY 14-2222 to BAY 81-8973 and then to BAY 94-9027 did experience similar or decreased total annual bleed rates with a decrease in their prophylaxis regimen frequency when transitioning to BAY 94-9027 in the real world. The therapeutic burden of frequent infusions can be reduced when patients transition between rFVIII product classes (SHL to EHL), without change in their annual bleed rate. These data should be interpreted with caution owing to..."

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

Real-World Effectiveness Data on Mild and Moderate Hemophilia a Patients Using Bay 81-8973 in the Athndataset (ASH 2024) - "ABRs were generally low for both on demand and prophylactically treating patients in both disease severities, supporting the benefit of using standard of care prophylaxis treatment in all hemophilia A patients. Without clinical evidence, the numerically slightly higher ABR in the prophylaxis vs on-demand groups, might be explained by a potentially more severe bleeding phenotype, requiring prophylaxis treatment in mild and moderate hemophilia A patients."

Clinical • Real-world • Real-world effectiveness • Real-world evidence • Cardiovascular • Hematological Disorders • Hemophilia • Hemophilia A • Hepatitis B • Hepatitis C • Hepatology • Human Immunodeficiency Virus • Infectious Disease • Inflammation • Rare Diseases • Thrombosis

Real-World Data on Hemophilia a Patient Usage of Bay 81-8973 and Bay 94-9027 Stratified By Body Mass Index Categories in the Athndataset (ASH 2024) - "Overall, a low number of bleeds were recorded in all BMI categories; however, the underweight and healthy weight patients numerically had the highest ABRs of all, using the highest amount of IU/kg but a smaller percentage infusing at the same frequency than the overweight and obese patients. Knowing that in vivo recovery with Factor VIII tends to be lowest in underweight patients, these data support previous findings that not only patients with high but also those with low BMIs should potentially be dosed according to different body measurements.These data should be interpreted with caution owing to limitations of real-world studies."

Clinical • Real-world • Real-world evidence • Cardiovascular • Hematological Disorders • Hemophilia • Hemophilia A • Obesity • Rare Diseases • Thrombosis

Preventing Bleeds in Pediatric Patients With Hemophilia A: Which Factor Replacement Therapy Offers the Best Protection and at What Cost? (ISPOR-EU 2025) - "Second lowest number of bleeds was achieved with efanesoctocog alfa (Altuviiio/Altuvoct) with 3.90 bleeds, following Efmoroctocog alfa (Elocta), turoctocog alfa pegol (Esperoct), turoctocog alfa (NovoEight), simoctocog alfa (Nuwiq), octocog alfa (Kovaltry), Afstyla (lonoctocog alfa) and octocog alfa (Advate) with 9.80, 9.85, 9.85, 11.85, 16.85, 18.45 and 18.75 bleeds, respectively. Prevention of bleeds is of utmost importance when treating pediatric patients with hemophilia A. Choosing a treatment with the lowest possible bleeding rates can support the physical development of the patient. This analysis showed that the lowest number of bleeds and lowest costs are estimated to be reached by damoctocog alfa pegol (Jivi)."

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

Analysis of Pooled Real-World Utilization and Outcomes Data of rVIII-Single Chain Compared With Standard and Extended Half-Life FVIII Products for Prophylaxis of Hemophilia A in France, Germany, and Italy (ISPOR-EU 2025) - "Prophylaxis with rVIII-SingleChain may reduce consumption and improve bleed protection compared with SHL FVIII products, and may provide comparable protection to EHL FVIII products."

Clinical • Real-world • Real-world evidence • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Drug Use Investigation of Kovaltry in Hemophilia A Patients (clinicaltrials.gov) - P=N/A | N=230 | Completed | Sponsor: Bayer | Active, not recruiting ➔ Completed

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

Canadian Clinical Experience on Switching From Standard Half-life Recombinant Factor VIII (rFVIII), Octocog Alfa, to Extended Half-life rFVIII, Damoctocog Alfa Pegol, in Persons With Haemophilia A ≥ 12 Years Followed in a Comprehensive Haemophilia Care Program in Canada. (PubMed, Haemophilia) - "This is a plain language summary on switching from the medicine octocog alfa to a new medicine damoctocog alfa pegol (BAY 94-9027, Jivi) for the treatment of haemophilia A in Canada."

Journal • 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

A comparison of My Precise Dose and WAPPS for optimizing prophylaxis in children with hemophilia A (ISTH 2025) - "Aims To compare the generated PK profiles and dosing estimations by WAPPS-Hemo and MPD for prophylaxis in children with hemophilia A treated with BAY 81-8973...(1 IU/dL: WAPPS-Hemo 12.1 (9.3, 17.1) vs. MPD 7.3 (5.5, 9.9), P<0.001; 3 IU/dL: WAPPS-Hemo 36.3 (27.8, 51.3) vs. MPD 21.9 (16.4, 30.0), P<0.001) . Table or Figure Upload"

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

AFEEL: Low Dose Emicizumab vs Low Dose Factor VIII in Prophylaxis in Hemophilia A Patients (clinicaltrials.gov) - P2/3 | N=20 | Not yet recruiting | Sponsor: Dhaka Medical College

New P2/3 trial • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

A comparison of My Precise Dose and WAPPS-Hemo as dosing tools for optimizing prophylaxis in children with hemophilia A treated with BAY 81-8973. (PubMed, Thromb Res) - "The pediatric popPK model such as MPD tailored for conditions can more precisely represent the PK characteristics and metabolic conditions of Chinese children in real-world settings, highlighting its potential utility in the clinical care of hemophilia patients."

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

BAY 81-8973 Demonstrates Long-Term Safety and Efficacy in Children With Severe Haemophilia A: Results From the LEOPOLD Kids Extension Study. (PubMed, Eur J Haematol) - P3 | "Long-term BAY 81-8973 treatment is well tolerated and maintains low ABRs for all bleeds and joint bleeds in children with severe haemophilia A."

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

Cost-Effectiveness Analysis of Different Recombinant Factor VIII for Prophylactic Treatment of Previously Untreated Patients With Severe Hemophilia A in China (ISPOR-EU 2024) - "Based on the best available evidence, rAHF-PFM is likely to be economically superior compared to BAY 81-8973 in PUPs with severe hemophilia A for lifetime prophylactic treatment in China."

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

Development and internal validation of a clinical prediction model for individualized dosing of BAY 81-8973, A full-length recombinant factor VIII, in pediatric patients with haemophilia A. (PubMed, Thromb Res) - "This is the first predictive model designed to predict individualized dosing of BAY 81-8973 in pediatric patients with Hemophilia A. These results are useful in the PK-guided prophylaxis among pediatric patients and hold great potential to improve their long-term clinical outcomes."

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

Development of a Rapid and Fully Automated Factor VIII-Inhibitor Assay, Insensitive for Emicizumab, and a Lowest Level of Quantification of 0.2BU/mL. (PubMed, J Thromb Haemost) - "We present development data of a sensitive and specific, rapid, automated FVIII inhibitor assay generating results within 20 minutes, is less resource intensive than standard assays, with potential to improve assay variability."

Journal • Hematological Disorders • Hemophilia • Rare Diseases

Population pharmacokinetics model of BAY81-8973 in Chinese children with severe haemophilia A (ISTH 2024) - "A two-compartment PopPK model adequately described FVIII activity. VWF:Ag and blood type were significant descriptors of clearance (CL) and volume of central compartment (V1). Fat free mass (FFM) was a significant descriptor of CL, volume of peripheral compartment (V1 & V2)."

Clinical • PK/PD data • Hematological Disorders • Hemophilia • Pediatrics • Rare Diseases

TITLE: REAL-WORLD EFFICACY DATA ON ADOLESCENT HEMOPHILIA A PATIENTS TRANSITIONING TO BAY 81-8973 OR BAY 94-9027 IN THE ATHNDATASET (THSNA 2024) - "The data show that adolescents with hemophilia A who transitioned to BAY 94-9027 or BAY 81-8973 experienced overall a decrease in their prophylaxis regimen frequency and mean total annual bleed rates. The therapeutic burden of frequent infusions can be reduced and treatment efficacy can be improved when adolescent patients transition to either BAY94-9027 or BAY81‑8973. These data should be interpreted with caution owing to limitations of real-world studies."

Clinical • Real-world • Real-world effectiveness • Real-world evidence • Cardiovascular • Hematological Disorders • Hemophilia • Pediatrics • Rare Diseases • Thrombosis

Drug Use Investigation of Kovaltry in Hemophilia A Patients (clinicaltrials.gov) - P=N/A | N=231 | Active, not recruiting | Sponsor: Bayer | Trial completion date: Mar 2024 ➔ Sep 2025

Trial completion date • Hematological Disorders • Hemophilia • Rare Diseases

A Post Approval Commitment Study to Gain More Information on How Safe and Effective KOVALTRY is in Chinese Children, Adolescents /Adults With Severe Hemophilia A (clinicaltrials.gov) - P4 | N=45 | Completed | Sponsor: Bayer | Recruiting ➔ Completed | Trial completion date: Sep 2028 ➔ Mar 2024 | Trial primary completion date: Sep 2028 ➔ Mar 2024

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

A Post Approval Commitment study to evaluate the efficacy, safety, and pharmacokinetics of KOVALTRY in Chinese children, adolescents/adults with severe hemophilia A. (clinicaltrialsregister.eu) - P4 | N=48 | Sponsor: Bayer Healthcare Company Limited

New P4 trial • Hematological Disorders • Hemophilia • Rare Diseases

Canadian clinical experience on switching from standard half-life recombinant factor VIII (rFVIII), octocog alfa, to extended half-life rFVIII, damoctocog alfa pegol, in persons with haemophilia A ≥ 12 years followed in a Comprehensive Hemophilia Care Program in Canada. (PubMed, Haemophilia) - "This study provides routine clinical evidence supporting the benefits of switching from octocog alfa to damoctocog alfa pegol for patients with severe haemophilia A."

Journal • Hematological Disorders • Hemophilia • Rare Diseases

Structural characterization of coagulation factor VIII (ISTH 2023) - " FL-FVIII purified from commercial sources i.e. recombinant FL-FVIII (rFL-FVIII) (Kovaltry) and plasma-derived FL-FVIII (pFL-FVIII) (Hemoctin) were subjected to cryo-EM (cryo-electron microscopy) and AFM (atomic force microscopy) studies... The globular regions of FL-FVIII fitted well within low-resolution cryo-EM maps obtained for rFL-FVIII and pFL-FVIII. The B-domain was still missing from these maps but appeared to be part of scattered density surrounding the central core. Similar observations of B-domain wrapping around the core globular domains were made in the AFM images."

Development of a Rapid and Fully Automated FVIII Inhibitor Assay Insensitive to the Presence of Emicizumab (ISTH 2023) - " In the Rapid FVIII Inhibitor Assay, VWF-free recombinant-FVIII (Kovaltry®, Bayer®) is used as inhibitor-substrate which dramatically lowers incubation time. After heat inactivation of patient samples, multiple dilutions of the samples were measured fully automatically. All samples, both positive and negative, returned inhibitor results which were in line with the inhibitor potency previously reported in these samples. Conclusion(s): This chromogenic variant of the novel Rapid FVIII Inhibitor Assay exhibits potential to be a game changer in FVIII-inhibitor detection concerning accuracy, availability and standardisation irrespective of the therapy (replacement or non-replacing) being used.Learning Objectives: Upon completion, participants will be able to define the limitations of the classic Factor VIII Inhibitor assays with respect to complicated liquid handling and analytical variability."

Title: Real-World data on Hemophilia A patients with inhibitors switching to either BAY 94-9027 or BAY 81-8973 in the ATHNdataset (ISTH 2023) - "A total of 205 patients were treating with BAY 94-9027 and 354 with BAY 81-8973. Fifteen (7.3%) patients had an inhibitor titer ≥0.6 BU/ml before switching to BAY 94-9027. Titers ranged from 0.6 BU/ml to 56 BU/ml."

Clinical • Real-world • Real-world evidence • Cardiovascular • Hematological Disorders • Hemophilia • Rare Diseases • Thrombosis