Drug updated on 10/30/2024
| Dosage Form | Injection (subcutaneous; 12 mg/0.4 mL, 30 mg/mL, 60 mg/0.4 mL, 105 mg/0.7 mL, 150 mg/mL) |
| Drug Class | Bispecific factor IXa- and factor X-directed antibodies |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients ages newborn and older with hemophilia A (congenital factor VIII deficiency) with or without factor VIII inhibitors.
Latest News
Summary
- This summary is based on the review of five systematic review(s)/meta-analysis(es). [1-5]
- Emicizumab vs. Bypassing Agents (BPA) in PwHAi: Emicizumab prophylaxis significantly reduced the annualized bleeding rate (ABR) for treated bleeds compared to BPA prophylaxis, with a standard mean difference of -1.58 (95% CI: -2.50 to -0.66, P = 0.0008), indicating fewer bleeding events with emicizumab.
- Emicizumab Pharmacokinetics and Efficacy: Emicizumab exhibited dose-linear pharmacokinetics with moderate interindividual variability (32%). Bleed control did not improve significantly above emicizumab concentrations of 30 μg/mL, suggesting that lower dosing may be sufficient for many patients.
- Emicizumab vs. Factor VIII in PwHA without Inhibitors: Emicizumab prophylaxis resulted in a lower total treated bleed rate compared to Factor VIII prophylaxis (Rate Ratio [RR] = 0.36; 95% credible interval [CrI]: 0.13-0.95), demonstrating superior effectiveness in reducing bleeding events.
- Emicizumab was effective in reducing treated bleed rates in both people with hemophilia A (PwHA) with and without inhibitors. Subgroup analyses showed moderate interindividual variability in trough concentrations across children and adults, with effective bleed control at emicizumab concentrations above 30 μg/mL.
- There is no safety information available in the reviewed studies.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Hemlibra (emicizumab-kxwh) Prescribing Information. | 2024 | Genentech, Inc., South San Francisco, CA |
Systematic Reviews / Meta-Analyses
| Document Title | Year | Source |
|---|---|---|
| Emicizumab prophylaxis in people with hemophilia A and inhibitors: a systematic review and meta-analysis | 2024 | Sao Paulo Medical Journal |
| Pharmacokinetics and Associated Efficacy of Emicizumab in Humans: A Systematic Review | 2021 | Clinical Pharmacokinetics |
| Intraindividual Comparisons to Determine Comparative Effectiveness: Their Relevance for G-BA's Health Technology Assessments | 2021 | Value in Health |
| Prophylaxis for hemophilia A without inhibitors: treatment options and considerations | 2020 | Expert Review of Hematology |
| Efficacy of emicizumab prophylaxis versus factor VIII prophylaxis for treatment of hemophilia A without inhibitors: network meta-analysis and sub-group analyses of the intra-patient comparison of the HAVEN 3 trial | 2019 | Current Medical Research and Opinion |