Drug updated on 7/25/2024
| 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
- Emicizumab (Hemlibra) is 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.
- The information was derived from 2 systematic reviews/meta-analyses documents focusing on emicizumab's pharmacokinetics, efficacy, and comparison with FVIII prophylaxis.
- In a study involving 140 volunteers and 467 patients including children (<12 years) and adults (≥12 years), emicizumab demonstrated dose-linear pharmacokinetics with moderate interindividual variability. Control of bleeds did not improve significantly above concentrations of 30 µg/mL, suggesting potential for lower dosing in several patients.
- Network meta-analysis comparing emicizumab prophylaxis to FVIII prophylaxis showed that the total treated bleed rate was significantly lower with emicizumab (Rate Ratio [RR] = 0.36). Additional intra-patient analyses confirmed superior efficacy over FVIII prophylaxis across various sub-groups from the HAVEN 3 trial.
- Emicizumab supports body weight-based dosing which may benefit individualized monitoring for cost-effective treatment while maintaining effective control of bleeding episodes compared to traditional FVIII therapy.
- Consistent pharmacokinetic profiles were observed across different age groups and the presence/absence of factor VIII inhibitors, indicating reliable performance in diverse patient populations.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Hemlibra (emicizumab) Prescribing Information. | 2022 | Genentech, Inc., South San Francisco, CA |
Systematic Reviews / Meta-Analyses
| Document Title | Year | Source |
|---|---|---|
| Pharmacokinetics and associated efficacy of emicizumab in humans: a systematic review. | 2021 | Clinical Pharmacokinetics |
| 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 |