Emicizumab

(Hemlibra®)

Hemlibra®

Drug updated on 5/17/2024

Dosage FormInjection (subcutaneous; 12 mg/0.4 mL, 30 mg/mL, 60 mg/0.4 mL, 105 mg/0.7 mL, 150 mg/mL)
Drug ClassBispecific 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
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  • 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, both with or without factor VIII inhibitors. It has demonstrated dose-linear pharmacokinetics, meaning its blood concentrations increase linearly with higher doses.
  • The information was derived from one systematic review/meta-analysis that provided comprehensive insights into the drug's usage, safety profile, effectiveness comparison, and special considerations.
  • This drug has been studied across a diverse demography, including children (0 to <12 years), adolescents, and adults (>12 years). Both subgroups - those with factor VIII inhibitors as well as those without - were considered, which showcases Emicizumab’s broad applicability within the hemophilia A community.
  • There exists moderate interindividual variability in trough concentrations (32%), indicating some individual variability in response to Emicizumab. However, control of bleeds did not further improve above Emicizumab concentrations of 30 µg/mL, suggesting potential for dose optimization while maintaining efficacy.
  • In terms of safety and effectiveness comparison, based on this single study, it appears that Emicizumab’s performance is comparable or superior to existing treatments, but precise superiority cannot be established due to lack of direct comparisons within this study reviewed.
  • Special consideration should be given towards monitoring dosing regimen since there seems to be a possibility for more cost-effective dosing while maintaining efficacy due to its predictable treatment profile.