Drug updated on 12/11/2024
| Dosage Form | Injection (intravenous; 300 mg/30 mL [10 mg/mL], 300 mg/3 mL [100 mg/mL], 1,100 mg/11 mL [100 mg/mL]) |
| Drug Class | Complement inhibitors |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for the treatment of adult and pediatric patients one month of age and older with paroxysmal nocturnal hemoglobinuria (PNH)
- Indicated for the treatment of adult and pediatric patients one month of age and older with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA)
- Indicated for the treatment of adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive
- Indicated for the treatment of adult patients with neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP4) antibody positive.
Latest News
Summary
- This summary is based on the review of five systematic review(s)/meta-analysis(es). [1-5]
- The studies involved general populations with Paroxysmal Nocturnal Hemoglobinuria (PNH), Atypical Hemolytic Uremic Syndrome (aHUS), and Myasthenia Gravis (MG), with PNH patients treated with complement inhibitors and aHUS patients showing a higher risk of recurrence associated with genetic mutations (complement factor I (CFI), membrane cofactor protein (MCP), and complement factor H (CFH)). Additionally, 40% of aHUS patients are under 18 years old.
- In aHUS, the identification of genetic mutations necessitates prompt initiation of monoclonal antibody treatment to mitigate recurrence risks, emphasizing the importance of genetic screening in this population.
- The findings also indicate that the general MG population is treated with innovative therapies, including complement inhibitors and FcRn blockers, suggesting a shift toward personalized treatment approaches based on individual patient needs.
- In PNH, no specific safety outcomes were highlighted for ravulizumab compared to other drugs, indicating a lack of detailed safety data.
- In aHUS, serious adverse events occurred in 42% of patients treated with Eculizumab, along with two cases of meningococcal infection. Ravulizumab was associated with four deaths (7%) after 26 weeks, but no specific adverse events were mentioned in the study.
- There is no population type or subgroup information available in the reviewed studies.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Ultomiris (ravulizumab-cwvz) Prescribing Information. | 2024 | Alexion Pharmaceuticals, Inc., Boston, MA |
Systematic Reviews / Meta-Analyses
| Document Title | Year | Source |
|---|---|---|
| Efficacy of complement inhibitors for patients with paroxysmal nocturnal hemoglobinuria: a systematic review and meta-analysis | 2023 | Therapeutic Advances in Hematology |
| Eculizumab Versus Ravulizumab for the Treatment of Atypical Hemolytic Uremic Syndrome: A Systematic Review | 2023 | Cureus |
| Efficacy of innovative therapies in myasthenia gravis: A systematic review, meta-analysis and network meta-analysis | 2023 | European Journal of Neurology |
| Complement Inhibition in Paroxysmal Nocturnal Hemoglobinuria (PNH): A Systematic Review and Expert Opinion from Central Europe on Special Patient Populations | 2023 | Advances in Therapy |
| Interventions for atypical haemolytic uraemic syndrome | 2021 | The Cochrane Database of Systematic Reviews |
Clinical Practice Guidelines
| Document Title | Year | Source |
|---|---|---|
| Update on the diagnosis and treatment of neuromyelitis optica spectrum disorders (NMOSD) – revised recommendations of the Neuromyelitis Optica Study Group (NEMOS). Part II: Attack therapy and long-term management | 2024 | Journal of Neurology |
| A critical appraisal of clinical practice guidelines for pharmacological treatments of paroxysmal nocturnal hemoglobinuria. | 2023 | Journal of Advances in Biology & Biotechnology |
| Guideline for the management of myasthenic syndromes | 2023 | Therapeutic Advances in Neurological Disorders |