Drug updated on 9/4/2024
Dosage Form | Injection (intravenous; 40 mg/10 mL [4 mg/mL]) |
Drug Class | GD2-binding monoclonal antibodies |
Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated, in combination with granulocyte-macrophage colony-stimulating factor (GMCSF), for the treatment of pediatric patients 1 year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy.
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Summary
- Danyelza (naxitamab-gqgk) is indicated, in combination with granulocyte-macrophage colony-stimulating factor (GMCSF), for the treatment of pediatric patients 1 year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy.
- This summary is based on the review of three randomized controlled trials. [1-3]
- Effectiveness in High-Risk Neuroblastoma: In Trial 201 (NCT03363373), 75% of patients with high-risk neuroblastoma achieved an overall objective response, with 67% achieving a complete response. Among patients with bone marrow involvement, 92% (12/13) achieved a complete response, indicating a high level of effectiveness in this challenging population.
- Comparison to Historical Therapies: While the GD2/GD3 vaccine trial showed a 5-year progression-free survival (PFS) of 32% ± 6% and overall survival (OS) of 71% ± 7%, the specific comparative effectiveness between this and the anti-GD2 monoclonal antibody therapy used in Trial 201 is not provided, making direct comparisons difficult.
- Population Focus: The trial primarily focused on pediatric patients with high-risk neuroblastoma who had either refractory disease or insufficient response to previous treatments, demonstrating significant efficacy in this vulnerable subgroup.
- Adverse Events (AEs): In Trial 201 (NCT03363373), common adverse events included pain, hypotension, bronchospasm, cough, vomiting, diarrhea, nausea, and tachycardia, occurring in at least 50% of patients. Grade ≥3 AEs, such as pain, hypotension, urticaria, and bronchospasm, were reported in at least 10% of patients.
- Serious Adverse Events (SAEs): Six treatment-related serious adverse events were reported in five patients, including anaphylactic reaction, pyrexia, and respiratory depression, indicating a need for careful monitoring and management during treatment.
- Subgroup Findings: The studies focused on pediatric patients with high-risk neuroblastoma, particularly those with disease in bone and/or bone marrow who were refractory to initial treatments or had insufficient responses to therapy for progressive/relapsed disease. In this population, naxitamab showed a high complete response rate of 67% and an overall objective response rate of 75%. Common adverse events, including pain, hypotension, and bronchospasm, were manageable in an outpatient setting with appropriate premedications and monitoring. No specific differences in effectiveness or safety were noted among different subgroups within this pediatric population.
Product Monograph / Prescribing Information
Document Title | Year | Source |
---|---|---|
Danyelza (naxitamab-gqgk) Prescribing Information. | 2024 | Y-mAbs Therapeutics, Inc., New York, NY |
Randomized Controlled Trials
Document Title | Sex Distribution | Year | Source |
---|---|---|---|
Outpatient administration of naxitamab in combination with granulocyte-macrophage colony-stimulating factor in patients with refractory and/or relapsed high-risk neuroblastoma: Management of adverse events. | Data not availableSubjects F: null% M: null% | 2023 | Cancer Reports |
Naxitamab, a new generation anti-GD2 monoclonal antibody (mAb) for treatment of relapsed/refractory high-risk neuroblastoma (HR-NB). | Data not availableSubjects F: null% M: null% | 2020 | Journal of Clinical Oncology |
Survival impact of anti-GD2 antibody response in a phase II ganglioside vaccine trial among patients with high-risk neuroblastoma with prior disease progression. | Data not availableSubjects F: null% M: null% | 2020 | Journal of Clinical Oncology |
Sex Distribution:
![No Data](/img/Nodata_Icon.png)
Year:
2023
Source:Cancer Reports
Sex Distribution:
![No Data](/img/Nodata_Icon.png)
Year:
2020
Source:Journal of Clinical Oncology
Sex Distribution:
![No Data](/img/Nodata_Icon.png)
Year:
2020
Source:Journal of Clinical Oncology