Drug updated on 3/28/2024
| Dosage Form | Injection (subcutaneous; 1,080 mg/20 mL (54 mg/mL)) |
| Drug Class | Complement inhibitors |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- For the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH).
Summary
- Pegcetacoplan (Empaveli) is a complement 3 (C3) inhibitor used for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH).
- Two systematic reviews/meta-analyses were reviewed, providing evidence on the efficacy and safety of pegcetacoplan in treating PNH.
- The first study indicates that pegcetacoplan may be beneficial for specific populations who do not respond adequately to other treatments such as eculizumab and ravulizumab, which are humanized monoclonal antibodies targeting complement 5 (C5).
- According to both studys, studies have shown an increase in hemoglobin levels among PNH patients treated with pegcetacoplan; one study reported an average increase of 2.37g/L while another showed an average rise of 3.68g/L.
- In addition to improving hemoglobin levels, pegcetacoplan also reduced absolute reticulocyte count and lactate dehydrogenase (LDH) levels in all patients receiving this therapy according to one study's findings.
- Regarding safety outcomes from these documents: adverse events were reported by some participants across different studies but no deaths were associated directly with Empaveli use during these trials - indicating it has a favorable safety profile overall when used for its indicated purpose within this patient population group suffering from PNH disease condition(s).
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Empaveli (pegcetacoplan) Prescribing Information. | 2023 | Apellis Pharmaceuticals, Inc., Waltham, MA |
Systematic Reviews / Meta-Analyses
| Document Title | Year | Source |
|---|---|---|
| Clinical, pharmacoeconomic, and stakeholder input combined report. | 2023 | CADTH |
| Complement inhibition in paroxysmal nocturnal hemoglobinuria (PNH): a systematic review and expert opinion from central Europe on special patient populations. | 2023 | Advances in Therapy |
| Australian public assessment report for pegceacoplan. | 2022 | Australian Government: Department of Health |
| Pegcetacoplan in paroxysmal nocturnal hemoglobinuria: a systematic review on efficacy and safety. | 2022 | Research and Practice in Thrombosis and Haemostasis |