Sevenfact (eptacog beta activated) / LFB SA, HEMA Biologics - LARVOL DELTA

Recombinant factor VIIa: new insights into the mechanism of action through product innovation. (PubMed, Res Pract Thromb Haemost) - "Eptacog alfa and eptacog beta share the same amino acid sequence but differ in posttranslational modifications. Further research is required to assess the clinical relevance of these differences. A better understanding of the mechanisms by which rFVIIa promotes hemostasis in patients will provide insights when evaluating clinical outcomes of safety and efficacy for innovative bypassing therapies."

Journal • Review • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases • PROCR

Real-World Effectiveness of Eptacog Beta [Coagulation Factor VIIa (Recombinant)] in the USA (EAHAD 2025) - No abstract available

Clinical • Real-world • Real-world effectiveness • Real-world evidence

Bleed treatment with eptacog beta (rFVIIa) results in a low incidence of rebleeding in adult and adolescent patients with haemophilia A or B with inhibitors. (PubMed, Haemophilia) - "Subjects with haemophilia with inhibitors who used eptacog beta to treat mild/moderate BEs experienced a low incidence of rebleeding. Rebleeds that did occur were effectively controlled with eptacog beta (median, one dose) without the need for hospitalization."

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

Pain Reduction Following Treatment of Bleeding Episodes with Eptacog Beta in Adults and Adolescents with Hemophilia A or B Complicated By Inhibitors (ASH 2024) - P3 | "Our findings provide new benchmark measurements of clinically relevant pain relief in PwHABI. We propose that the reductions in pain observed in this study might be adopted as a possible endpoint for substantive effect when designing clinical trials of bypassing agents in PwHABI."

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

ATHN 16: SEVENFACT® for Bleeding Events in Hemophilia With Inhibitors (clinicaltrials.gov) - P4 | N=55 | Recruiting | Sponsor: American Thrombosis and Hemostasis Network | Trial completion date: Jun 2025 ➔ Mar 2027 | Trial primary completion date: Nov 2024 ➔ Sep 2026

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

Real-world effectiveness of eptacog beta in patients with haemophilia and inhibitors: A multi-institutional case series. (PubMed, Haemophilia) - "In this case series, eptacog beta was safe, effective, and economical as first-line therapy, treatment of refractory BEs, management of perioperative bleeding, or prophylaxis in haemophilia patients with inhibitors."

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

Retrospective Experience Assessing the Real-World Utilization and Effectiveness of SEVENFACT® (clinicaltrials.gov) - P=N/A | N=17 | Completed | Sponsor: Laboratoire français de Fractionnement et de Biotechnologies | Recruiting ➔ Completed

Real-world • Real-world evidence • Trial completion • Hematological Disorders • Hemophilia • Rare Diseases

High Bleed Control with a Single 225 µg/kg Eptacog Beta Dose in Adult and Adolescent Hemophilia A or B Persons with Inhibitors (ISTH 2024) - P3 | "Twenty-five out of 27 subjects treated 213 mild/moderate BEs using the 225µg/kg IDR, and a single-dose efficacy of 84% was observed. Single-dose efficacy with the 225µg/kg IDR for 182 joint and 31 non-joint BEs was 86.5% and 72.4%, respectively. Similar single-dose efficacy was observed for 58 target joint and 124 non-target joint BEs (84.6% and 87.3%, respectively) and for 182 spontaneous and 31 traumatic BEs (85.8% and 76.7%, respectively; Figure 2)."

Clinical • Hematological Disorders • Hemophilia • Rare Diseases

EFFICACY AND SAFETY OF EPTACOG BETA FOR BLEED TREATMENT IN ADULTS AND ADOLESCENTS WITH HEMOPHILIA B AND INHIBITORS DURING PERSEPT 1 (THSNA 2024) - "While assigned to the 225µg/kg IDR, Subject 1 experienced a treatment-related adverse event (increased body temperature) of moderate severity, which resolved following administration of ibuprofen and other non-steroidal anti-inflammatory medications.  Both eptacog beta IDRs provided safe and effective control of mild or moderate BEs in PwHBI during PERSEPT 1. Most BEs experienced by PwHBI were successfully treated at 12 h post-initial eptacog beta infusion, and all BEs in PwHBI treated with the 225µg/kg IDR had resolved at 24 h. These results support the efficacy and safety of eptacog beta for BE treatment in adult and adolescent PwHBI."

Clinical • Hematological Disorders • Hemophilia • Immunology • Pain • Rare Diseases

INSIGHT INTO ATHN 16: TREATMENT OF BLEEDING EVENTS IN PATIENTS WITH CONGENITAL HEMOPHILIA A OR B WITH INHIBITORS TO EVALUATE THE SAFETY OF COAGULATION FACTOR VIIA (RECOMBINANT)-JNCW (THSNA 2024) - P4 | "All study participants with severe hemophilia received prophylactic emicizumab with one starting emicizumab prophylaxis during the trial. To date, the safety profile of eptacog beta for the treatment of BEs in the ATHN 16 participants is consistent with previously published reports."

Clinical • Cardiovascular • Hematological Disorders • Hemophilia • Infectious Disease • Oncology • Rare Diseases • Thrombosis

REBLEED INCIDENCE WITH EPTACOG BETA TREATMENT THROUGH 24 AND 48 HOURS IN CHILDREN (UNDER 12 YEARS) WITH HEMOPHILIA A OR B WITH INHIBITORS (THSNA 2024) - P3 | "BE treatment using either eptacog beta IDR led to low rebleeding incidence (<3%) through 24 and 48 hours in PwHABI <12 years during PERSEPT2."

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

Retrospective Experience Assessing the Real-World Utilization and Effectiveness of SEVENFACT® (clinicaltrials.gov) - P=N/A | N=20 | Recruiting | Sponsor: Laboratoire français de Fractionnement et de Biotechnologies | Trial primary completion date: Jan 2024 ➔ Apr 2024

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

Eptacog beta for the management of patients with haemophilia A and B with inhibitors: A European perspective. (PubMed, Haemophilia) - "In this review, we summarise current data regarding the mode of action, clinical efficacy and safety of eptacog beta for the management of haemophilia A and B in patients with inhibitors from a European perspective. In addition to providing a valuable summary of the analyses of the clinical data for eptacog beta conducted for the EMA, our review summarises the potential differentiators for eptacog beta compared with other current bypassing agents."

Journal • Review • Hematological Disorders • Hemophilia • Rare Diseases

Stability of Eptacog Beta After Reconstitution (EAHAD 2024) - No abstract available

Retrospective Experience Assessing the Real-World Utilization and Effectiveness of SEVENFACT® (clinicaltrials.gov) - P=N/A | N=20 | Recruiting | Sponsor: Laboratoire français de Fractionnement et de Biotechnologies | Not yet recruiting ➔ Recruiting

Enrollment open • Hematological Disorders • Hemophilia • Rare Diseases

Low Rebleed Incidence with Eptacog Beta Treatment through 24 and 48 Hours in Children (<12 years) with Hemophilia a or B with Inhibitors (ASH 2023) - P3 | "These findings align with those previously reported for PERSEPT 1 in adolescent and adult patients. Combined with the high efficacy at 24 hours (>96%) observed in both trials and both IDRs, these results suggest that eptacog beta can provide clinicians and caregivers with a treatment option that can establish and maintain hemostatic control of mild or moderate BEs in PwHABI of all ages."

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

Race against time: Evaluating eptacog beta for bleeding cessation in pediatric patients (ASHP 2023) - No abstract available

Clinical • Pediatrics

Retrospective Experience Assessing the Real-World Utilization and Effectiveness of SEVENFACT® (clinicaltrials.gov) - P=N/A | N=20 | Not yet recruiting | Sponsor: Laboratoire français de Fractionnement et de Biotechnologies | Initiation date: Jul 2023 ➔ Oct 2023

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

Efficacy and safety evaluation of eptacog beta (coagulation factor VIIa [recombinant]-jncw) for the treatment of hemophilia a and B with inhibitors. (PubMed, Expert Rev Hematol) - "This has become particularly important given the use of subcutaneous medications (e.g. emicizumab) for prophylaxis of bleeding. Clinicians should consider eptacog beta as a BPA for all PwHABI."

Journal • Review • Hematological Disorders • Hemophilia • Rare Diseases

Efficacy and Safety of Eptacog Beta (Recombinant Activated FVII) in Patients with Hemophilia A or B with Inhibitors According to Time to Initial Infusion: a Post-Hoc Analysis from PERSEPT-1 (ISTH 2023) - P3 | "Across 468 BEs treated, the overall success proportion for the primary endpoint was 85.3% (95% CI 77.0-93.5) with 81.0% (70.9-91.0) and 90.3% (82.9-97.7) in the 75 and 225μg/kg IDRs, respectively. Initial infusion at < 30min was associated with numerically higher success proportion for both IDRs, compared with an initial infusion at 30-60 and ≥60min; but the differences did not reach statistical significance. No significant differences were found when comparing both IDRs (Figure 1)."

Clinical • Retrospective data • Cardiovascular • Hematological Disorders • Hemophilia • Immunology • Musculoskeletal Diseases • Musculoskeletal Pain • Pain • Rare Diseases

Preliminary Analysis of ATHN 16: Real-World Safety of Eptacog Beta (ISTH 2023) - P4 | "Background: Eptacog beta (rFVIIa-jncw) received regulatory approval in the U.S. in 2020 for the treatment of bleeding events (BEs) in persons with hemophilia A/B with inhibitors (PwHABI), 12 years of age and older. Between August 2021 and October 2022, eleven subjects have been enrolled, ten of whom are on prophylactic emicizumab. Twenty-eight bleeding events have been treated (57% spontaneous, 36% traumatic, 8% other (surgery, activity/exercise) in four of the eleven enrolled participants. One adverse event (AE), unrelated to study product, was reported and resolved."

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

Retrospective Experience Assessing the Real-World Utilization and Effectiveness of SEVENFACT® (clinicaltrials.gov) - P=N/A | N=20 | Not yet recruiting | Sponsor: Laboratoire français de Fractionnement et de Biotechnologies

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

ATHN 16: SEVENFACT® for Bleeding Events in Hemophilia With Inhibitors (clinicaltrials.gov) - P4 | N=55 | Recruiting | Sponsor: American Thrombosis and Hemostasis Network | Trial primary completion date: Oct 2023 ➔ Nov 2024

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

Pain relief with Eptacog beta in haemophilia patients with inhibitors (#46) (GTH 2023) - "Conclusion Eptacog beta resulted in clinically relevant pain reduction and bleed resolution across all age groups in CHABwI. A higher analgesic requirement and longer time to pain relief were observed in younger patients, possibly due to difficulty in assessing pain in this younger patient population."

Clinical • Hematological Disorders • Hemophilia • Pain • Rare Diseases

EFFICACY AND SAFETY OF EPTACOG BETA (RECOMBINANT HUMAN FVIIA) ACCORDING TO AGE IN PERSONS WITH HAEMOPHILIA A/B WITH INHIBITORS UNDERGOING SURGICAL PROCEDURES (EAHAD 2023) - P3 | "This post-hoc subgroup analysis shows that eptacog beta is effective and well-tolerated in perioperative care irrespective of patient age (<12 vs ≥12y), supporting the use of eptacog beta for bleed management (prevention and treatment) in major and minor surgical procedures in all PwHABI."

Clinical • Hematological Disorders • Hemophilia • Rare Diseases