Summary

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• 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.