Drug updated on 12/11/2024
| 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 People with Hemophilia A with Inhibitors (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% confidence interval (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 people with hemophilia A (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 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 |