Drug updated on 12/11/2024
| Dosage Form | Tablet (oral; 50 mg, 100 mg) |
| Drug Class | Complement factor D inhibitors |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated as add-on therapy to ravulizumab or eculizumab for the treatment of extravascular hemolysis (EVH) in adults with paroxysmal nocturnal hemoglobinuria (PNH).
Latest News
Summary
- This summary is based on the review of one systematic review/meta-analysis. [1]
- The systematic review evaluated danicopan, pegcetacoplan, and iptacopan for managing eculizumab refractory paroxysmal nocturnal hemoglobinuria (PNH). However, specific effectiveness outcomes for these drugs were not provided, preventing direct comparisons.
- The review emphasizes the need for individualized treatment approaches based on the mechanisms of eculizumab refractoriness and PNH breakthrough, without indicating a universally superior drug.
- No effectiveness data or subgroup outcomes for different population types (e.g., age, gender, comorbidities) were detailed in the study.
- There is no safety information available in the reviewed studies.
- There is no population type or subgroup information available in the reviewed studies.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Voydeya (danicopan) Prescribing Information. | 2024 | Alexion Pharmaceuticals, Inc., Boston, 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 |
Clinical Practice Guidelines
| Document Title | Year | Source |
|---|---|---|
| Expert consensus on the management of pharmacodynamic breakthrough-hemolysis in treated paroxysmal nocturnal hemoglobinuria | 2024 | Hematology |