Drug updated on 3/28/2024
| Dosage Form | Injection (intravenous; 300 mg/30 mL) |
| Drug Class | Complement inhibitors |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- To treat paroxysmal nocturnal hemoglobinuria in adults and pediatric patients one month of age and older.
- To treat atypical hemolytic uremic syndrome in adults and pediatric patients one month of age and older.
- To treat atypical hemolytic uremic syndrome in adults and pediatric patients one month of age and older.
Summary
- Ravulizumab (Ultomiris) is used to treat paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome in adults and pediatric patients one month of age and older.
- Three systematic reviews/meta-analyses were reviewed, providing insights into the safety, efficacy, financial burden, biomarker associations with interventions, role of genetic mutations in prognosis for ravulizumab treatment.
- The first review found that both eculizumab and ravulizumab are comparable in terms of safety and efficacy; however, ravulizumab was preferred by patients due to its lower financial burden and less frequent dosing schedule.
- Soluble complement 5b-9 (sC5b), especially when present in urine samples could potentially be used as a biomarker to assess response to treatment with Ultomiris according to the same study.
- Genetic mutations such as those affecting complement factor I (CFI), membrane cofactor protein (MCP), or complement factor H (CFH) may increase risk of disease recurrence after discontinuation of therapy; hence caution should be exercised while considering cessation among these patient subgroups per findings from the first study reviewed.
- The third document suggests that terminal complement inhibition using drugs like Ultomiris appears beneficial for patients suffering from atypical haemolytic uraemic syndrome based on low-quality evidence drawn from five single-arm studies compared against historical data but calls for further research given high bias potential inherent within non-randomized trials design employed so far.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Ultomiris (ravulizumab) Prescribing Information. | 2022 | Alexion Pharmaceuticals, Inc., Boston, MA |
Systematic Reviews / Meta-Analyses
| Document Title | Year | Source |
|---|---|---|
| Clinical, pharmacoeconomic, and stakeholder input combined report. | 2023 | CADTH |
| Eculizumab Versus Ravulizumab for the Treatment of Atypical Hemolytic Uremic Syndrome: A Systematic Review. | 2023 | Cureus |
| Complement Inhibition in Paroxysmal Nocturnal Hemoglobinuria (PNH): A Systematic Review and Expert Opinion from Central Europe on Special Patient Populations. | 2023 | Advances in Therapy |
| Combined clinical and pharmacoeconomic report: ravulizumab (Ultomiris). | 2022 | CADTH |
| Assessment report: Ultomiris. | 2021 | EMA |
| Interventions for atypical haemolytic uraemic syndrome. | 2021 | Cochrane Database of Systematic Reviews |