ArticleBlood advances2022
Efanesoctocog alfa for hemophilia A: results from a phase 1 repeat-dose study.
Article in Blood advances, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 25 papers, 2 of them syntheses that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
25 citing papers in PubMed, 2 syntheses or guidelines pooled it, 40 citations in OpenAlex.
- Efanesoctocog Alfa versus Standard and Extended Half-Life Factor VIII Prophylaxis in Adolescent and Adult Patients with Haemophilia A without Inhibitors.Advances in therapy · 2025Pooled it
- Efanesoctocog Alfa Versus Emicizumab in Adolescent and Adult Patients With Haemophilia A Without Inhibitors.Advances in therapy · 2025Pooled it
- Efanesoctocog Alfa Population Pharmacokinetics and Repeated Time-To-Event Analysis of Bleeds in Adults, Adolescents, and Children with Severe Hemophilia A.Journal of clinical pharmacology · 2025Trial
- Treatment of Bleeding Episodes With Efanesoctocog Alfa in Previously Treated Patients With Severe Hemophilia A in the Phase 3 XTEND-1 Study.American journal of hematology · 2025Trial
- Perioperative Management With Efanesoctocog Alfa in Patients With Haemophilia A in the Phase 3 XTEND-1 and XTEND-Kids Studies.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Trial
- A Psychometric Analysis of the Haemophilia Quality of Life Questionnaire for Adults Physical Health Subscale and Total Scores: Data From the Phase 3 XTEND-1 Trial in Patients With Severe Haemophilia A.Haemophilia : the official journal of the World Federation of HemophiliaTrial
- Experiences with Efanesoctocog Alfa: Exit Interviews with Caregivers of Previously Treated XTEND-Kids Phase III Trial Patients with Haemophilia A.The patient · 2026Article
- Peptide and Protein Tags for the Half-Life Extension of Therapeutic Agents: A Review Article.The protein journal · 2026Review
- Cost Effectiveness of Efanesoctocog Alfa Versus Factor VIII Extended Half-Life in Adolescent and Adult Patients with Hemophilia A in the USA.PharmacoEconomics · 2026Article
- Individual Comparative PK Evaluation of Single-dose Octocog Alfa, Rurioctocog Alfa Pegol, and Efanesoctocog Alfa in Adults with Severe Hemophilia A.TH open : companion journal to thrombosis and haemostasis · 2026Article
- More real-world evidence on the impact of emicizumab.Research and practice in thrombosis and haemostasis · 2025Article
- Prospective, Observational Study of the Clinical Outcomes of FVIII Treatment in Adults and Adolescents with Severe Haemophilia A.TH open : companion journal to thrombosis and haemostasis · 2025Article
- Advances in biopharmaceutical products for hemophilia.iScience · 2024Review
- Efanesoctocog alfa: the renaissance of Factor VIII replacement therapy.Haematologica · 2024Review
- Exploring a novel long-acting glucagon-like peptide-1 receptor agonist built on the albumin-binding domain and XTEN scaffolds.Heliyon · 2024Article
- [Recent advances in the replacement therapy for Hemophilia].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2023Article
- Pharmacokinetics of recombinant factor VIII in adults with severe hemophilia A: fixed-sequence single-dose study of octocog alfa, rurioctocog alfa pegol, and efanesoctocog alfa.Research and practice in thrombosis and haemostasis · 2023Article
- Efanesoctocog Alfa: First Approval.Drugs · 2023Review
- Celebrating a year of clinical and translational research in Blood Advances.Blood advances · 2022Article
- Review
Corrections and comments
- Erratum issued
Authors and funding
6 authors at 2 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Efanesoctocog alfa (rFVIIIFc-VWF-XTEN; BIVV001) is a new class of factor VIII (FVIII) replacement that breaks the von Willebrand factor-imposed FVIII half-life ceiling. In a phase 1/2a study, single-dose efanesoctocog alfa was well tolerated, and no safety concerns were identified. We evaluated the safety, tolerability, and pharmacokinetics of repeat-dose efanesoctocog alfa in a phase 1 study in previously treated adults (≥150 exposure days) with severe hemophilia A. Participants received 4 once weekly doses of efanesoctocog alfa (cohort 1, 50 IU/kg; cohort 2, 65 IU/kg). All enrolled participants (cohort 1, n = 10; cohort 2, n = 14) completed the study. Inhibitor development to FVIII was not detected. After the last dose of efanesoctocog alfa, geometric mean (range) FVIII activity half-life, area under the activity-time curve, and steady-state maximum concentration for cohort 1 and cohort 2 were 41.3 (34.2-50.1) and 37.3 (28.9-43.8) hours, 8290 (5810-10 300) and 11 200 (7040-15 800) hours × IU/dL, and 131 (96-191) and 171 (118-211) IU/dL, respectively. There was minimal accumulation after 4 doses. Mean FVIII activity for cohort 1 and cohort 2, respectively, was 46% and 69% on day 3 postdose and 10% and 12% on day 7 postdose. Overall, 4 once-weekly doses of efanesoctocog alfa were well tolerated, no safety concerns were identified, and no bleeds were reported during the treatment period. Once-weekly efanesoctocog alfa provided high sustained FVIII activity within the normal to near-normal range for 3 to 4 days postdose and may improve protection against bleeds in patients with hemophilia A. The trial is study 2018-001535-51 in the EU Clinical Trials Register.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.