NCCN Adds Naxitamab to Practice Guidelines for High-Risk Neuroblastoma

Naxitamab-gqgk (Danyelza) has been added to the National Comprehensive Cancer Network’s (NCCN) Clinical Practice Guidelines in Oncology as a Category 2A treatment for high-risk neuroblastoma.¹

“We are very pleased with NCCN’s update of the NCCN Guidelines to include naxitamab-gqgk. We believe this decision reinforces the importance of naxitimab-gqgk as a leading anti-GD2 therapy of choice for physicians treating patients with relapsed/refractory high-risk neuroblastoma,” said Doug Gentilcore, senior vice president at Y-mAbs Therapeutics, in a press release.

The FDA granted naxitamab, a humanized monoclonal antibody targeting GD2, plus granulocyte-macrophage colony-stimulating factor (GM-CSF) accelerated approval in November 2020 for adult and pediatric patients aged 1 year and older with relapsed/refractory high-risk neuroblastoma in the bone or bone marrow that demonstrated a partial response, minor response, or stable disease to prior therapy.² This accelerated approval was supported by findings from the phase 2 Study 201 (NCT03363373) and phase 1 Study 12-230 (NCT01757626).

About Study 201

Study 201 is a phase 2 study investigating naxitamab plus GM-CSF in adult and pediatric patients with high-risk neuroblastoma in the bone and/or bone marrow with primary refractory disease or an incomplete response to salvage treatment.³ Patients are receiving treatment for up to 101 weeks following the first naxitamab administration.

Findings from the study were presented at the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting.⁴ Here, 22 patients were included in the analysis. The overall response rate (ORR) was 68% (n = 15; 95% CI, 45%-86%), with 59% (n = 13; 95% CI, 36%-79%) of patients achieving a complete response (CR). In 20 patients with disease in the bone compartment, the ORR was 80% (n = 16; 95% CI, 46%-88%) with a CR rate of 70% (n = 14; 95% CI, 46%-88%). In 9 patients with disease in the bone marrow compartment, the ORR and CR rate were both 78% (n = 7; 95% CI, 40%-97%).

Findings from a safety analysis were published in 2023. At least 50% of patients experienced pain, hypotension, bronchospasms, cough, vomiting, diarrhea, nausea, and tachycardia. Grade 3 or higher pain, hypotension, urticaria, and bronchospasm were reported in at least 10% of patients. However, adverse events were generally manageable in the outpatient setting with patient education and close monitoring.⁵

REFERENCES:

1. Y-mAbs announces update to National Comprehensive Cancer Network® (NCCN®) Clinical Practice Guidelines in Oncology for neuroblastoma to include naxitamab-gqgk (DANYELZA®). News release. Y-mAbs Therapeutics. May 7, 2025. Accessed May 8, 2025. https://tinyurl.com/bp7td4hd

2. FDA approves Y-mAbs’ DANYELZA® (naxitamab-gqgk) for the treatment of neuroblastoma. News release. Y-mAbs Therapeutics, Inc. November 25, 2020. Accessed May 9, 2025. https://bit.ly/378009V

3. Naxitamab for high-risk neuroblastoma patients with primary refractory disease or incomplete response to salvage treatment in bone and/​or bone marrow. ClinicalTrials.gov. Updated February 20, 2025. Accessed May 8, 2025. https://clinicaltrials.gov/study/NCT03363373

4. Kushner B, Morgenstern DA, Nysom K, et al. Efficacy of naxitamab in patients with refractory/relapse (R/R) high-risk neuroblastoma (HR-NB) by bone/bone marrow (BM) evaluation, potential sites of residual disease. J Clin Oncol. 39, 10022-10022(2021). doi:10.1200/JCO.2021.39.15_suppl.10022

5. Mora J, Chan GC, Morgenstern DA, et al. 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. Cancer Rep. 2023 Jan;6(1):e1627. doi: 10.1002/cnr2.1627. Epub 2022 May 17.