Drug updated on 4/16/2024
| Dosage Form | Injection (subcutaneous: 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.
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, with or without factor VIII inhibitors.
- The systematic review/meta-analysis document on Hemlibra revealed that it has a clear dose-concentration-response relationship, which allows dosing regimens based on body weight without the necessity for laboratory monitoring.
- This analysis included 15 studies involving 140 volunteers and 467 people with hemophilia A (PwHA), including children aged less than 12 years as well as adolescents and adults aged over or equal to 12 years, both with and without factor VIII inhibitors.
- It was found that emicizumab demonstrated dose-linear pharmacokinetics across various subgroups such as age group, Factor VIII inhibitor status, and dosing interval; indicating its effectiveness irrespective of these factors.
- The control of bleeds did not further improve above emicizumab concentrations of 30 µg/mL suggesting potential scope for lower dosing in a substantial proportion of PwHA thereby making treatment more cost-effective.
- Despite supporting body weight-based dosing regimen overall, individualized monitoring of emicizumab concentrations may be beneficial according to this meta-analysis document reviewed here; providing valuable insight into optimizing therapy while ensuring safety profile remains intact.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Hemlibra (emicizumab) Prescribing Information. | 2022 | Genentech, Inc., South San Francisco, CA |
Systematic Reviews / Meta-Analyses
Clinical Practice Guidelines
| Document Title | Year | Source |
|---|---|---|
| WFH guidelines for the management of hemophilia, 3rd edition. | 2021 | Haemophilia |
| Practical guidance of the gth haemophilia board on the use of emicizumab in patients with haemophilia a. https://www.thieme- | 2019 | Hamostaseologie |