{"entity":{"id":"naxitamab","kind":"drug","name":"Naxitamab","aka":[],"tldr":"Naxitamab is a humanised anti-GD2 antibody from Memorial Sloan Kettering, given as an outpatient with GM-CSF for relapsed neuroblastoma in bone or marrow.","summary":"Accelerated approval November 2020 for relapsed/refractory high-risk neuroblastoma in bone or bone marrow (Study 201: ORR 50%; MSK 12-230: ORR 45%). Phase 2 in primary refractory disease with stepped-up GM-CSF: 75% CR in a 32-patient series (2025). Outpatient 30-minute infusions but severe pain and hypertension. Y-mAbs.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Naxitamab","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Naxitamab"}],"tags":[],"related":[],"cancers":["neuroblastoma","neuroblastoma-high-risk"],"sections":[],"technologies":["monoclonal-antibody"],"targets":["gd2"],"drugs":[],"companies":["y-mabs"],"institutions":[],"pathways":[],"terms":[],"trials":["naxitamab-201"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Danyelza","modality":"Monoclonal antibody (anti-GD2, humanised)","mechanism":"Humanised 3F8 IgG1 anti-GD2 with higher affinity; ADCC/CDC enhanced by GM-CSF-primed granulocytes.","approvals":[{"region":"US","year":2020,"indication":"Relapsed/refractory high-risk neuroblastoma in bone or bone marrow with partial response, minor response or stable disease, with GM-CSF (accelerated)"}],"mechanismSteps":[],"dosing":{"route":"IV over 30-60 minutes (outpatient)","schedule":"3 mg/kg days 1, 3, 5 of each 4-week cycle with GM-CSF days -4 to 5; premedication with antihistamine, antipyretic, gabapentin, opioids","monitoring":"Pain, hypertension (including delayed), infusion reactions, neurotoxicity (transverse myelitis, RPLS), hypotension"},"toxicity":[{"event":"Pain","anyGradePct":100,"grade3PlusPct":65,"note":"Study 201 / 12-230 pooled"},{"event":"Hypertension","anyGradePct":42,"grade3PlusPct":28},{"event":"Infusion-related reaction","grade3PlusPct":32},{"event":"Neurotoxicity","anyGradePct":20}],"access":[],"regulatoryEvents":[{"date":"2020-11-25","type":"accelerated-approval","region":"US","note":"Accelerated approval (Study 201, 12-230) The confirmatory requirement was still open 5.8 years later, when the FDA's table was read.","indication":"In combination with granulocyte-macrophage colony-stimulating factor (GM-CSF), 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."}]},"route":"/drugs/naxitamab/","neighbours":{"cancer":[{"id":"childhood-cancers","kind":"cancer","name":"Childhood cancers (all types)","route":"/cancers/childhood-cancers/"},{"id":"neuroblastoma-high-risk","kind":"cancer","name":"High-risk neuroblastoma","route":"/cancers/neuroblastoma-high-risk/"},{"id":"neuroblastoma","kind":"cancer","name":"Neuroblastoma (paediatric)","route":"/cancers/neuroblastoma/"}],"technology":[{"id":"monoclonal-antibody","kind":"technology","name":"Monoclonal antibodies","route":"/technologies/monoclonal-antibody/"}],"target":[{"id":"gd2","kind":"target","name":"GD2 (disialoganglioside)","route":"/targets/gd2/"}],"company":[{"id":"y-mabs","kind":"company","name":"Y-mAbs Therapeutics","route":"/companies/y-mabs/"}],"trial":[{"id":"naxitamab-201","kind":"trial","name":"Naxitamab Study 201","route":"/trials/naxitamab-201/"},{"id":"nct07011654","kind":"trial","name":"Study of Naxitamab and Sacituzumab Govitecan in Patients With Metastatic Triple-negative Breast Cancer (TNBC)","route":"/trials/nct07011654/"}],"pairing":[{"id":"anti-gd2-plus-chemo-relapse","kind":"pairing","name":"Anti-GD2 antibody + irinotecan-temozolomide (chemoimmunotherapy)","route":"/pairings/anti-gd2-plus-chemo-relapse/"}],"bottleneck":[{"id":"b-rare-cancers","kind":"bottleneck","name":"Rare and paediatric cancers without markets","route":"/bottlenecks/b-rare-cancers/"}]}}