Drug updated on 9/4/2024
Dosage Form | Injection (subcutaneous; 1,080 mg/20 mL (54 mg/mL)) |
Drug Class | Complement inhibitors |
Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH).
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Summary
- Empaveli (pegcetacoplan) is indicated for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH).
- This summary is based on the review of four systematic review(s)/meta-analysis(es). [1-4]
- Hemoglobin Levels and LDH Reduction: Pegcetacoplan treatment in PNH patients resulted in an average hemoglobin increase of 3.68 g/L (PADDOCK study) and 2.37 g/L (Hillmen et al.), with LDH levels showing a mean reduction of -15 ± 43 U/L (Hillmen et al.) and stability within <1.5× upper limit of normal (De Castro et al.).
- Transfusion Independence and Comparative Effectiveness: Pegcetacoplan improved transfusion independence in PNH patients. When compared with other complement inhibitors (eculizumab, ravulizumab), pooled decreases in LDH were -1462.0 U/L for ≤26 weeks and -1696.5 U/L for >26 weeks of treatment, with hemoglobin increases of 1.4 g/dL and 1.9 g/dL, respectively. At least 50% of patients achieved transfusion avoidance across all durations.
- Quality of Life: Complement inhibitors, including pegcetacoplan, led to clinically meaningful improvements in FACIT-F scores, with pooled increases from 6.8 before to 9.5 after 26 weeks of treatment.
- Pegcetacoplan Adverse Events: Adverse events were reported in patients treated with pegcetacoplan across studies: 2 out of 6 patients (De Castro et al.), 7 out of 23 patients (PADDOCK study), and 7 out of 41 patients (Hillmen et al.).
- Safety Comparison: Specific safety outcomes comparing pegcetacoplan to eculizumab and ravulizumab were not detailed in the reviewed studies.
- Population Types and Subgroup Considerations: This section focuses on adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH), with 59.83% of participants being female in pegcetacoplan studies. There is also an emphasis on patients unresponsive to eculizumab, with individualized treatment plans recommended based on the mechanism of refractoriness.
Product Monograph / Prescribing Information
Document Title | Year | Source |
---|---|---|
Empaveli (pegcetacoplan) Prescribing Information. | 2024 | Apellis Pharmaceuticals, Inc., Waltham, MA |
Systematic Reviews / Meta-Analyses
Document Title | Year | Source |
---|---|---|
Treatment of eculizumab refractory paroxysmal nocturnal hemoglobinuria: a systematic review about current treatment options and future direction. | 2023 | SAGE Open Medicine |
Efficacy of complement inhibitors for patients with paroxysmal nocturnal hemoglobinuria: aA systematic review and meta-analysis. | 2023 | Therapeutic Advances in Hematology |
Complement inhibition in paroxysmal nocturnal hemoglobinuria (PNH): a systematic review and expert opinion from central Europe on special patient populations. | 2023 | Advances in Therapy |
Pegcetacoplan in paroxysmal nocturnal hemoglobinuria: a systematic review on efficacy and safety. | 2022 | Research and Practice in Thrombosis and Haemostasis |