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Study 201 was the single-arm phase 2 of 74 patients behind naxitamab's approval for relapsed or refractory high-risk neuroblastoma in bone or bone marrow: given with GM-CSF as an outpatient, the antibody produced responses in half of patients. Without a randomised comparison against dinutuximab, equal efficacy is unproven.
Naxitamab Study 201, trial NCT03363373 sponsored by Y-mAbs and reported in 2020 with the full cohort published in Nature Communications in 2025, was the pivotal single-arm study behind naxitamab's approval for relapsed or refractory high-risk neuroblastoma in bone or bone marrow. In 74 patients treated with naxitamab and GM-CSF the objective response rate was 50 percent, with complete responses in 38 percent at the interim analysis and durable responses, and the drug can be given as an outpatient. OnCo links it to paediatric neuroblastoma, naxitamab, Nai-Kong V. Cheung and the bottleneck of rare and paediatric cancers without markets. Without a randomised comparison against dinutuximab, whether naxitamab's outpatient convenience comes with equal efficacy is the open question.
Numbers are from the trial as recorded here; see the source links in the table below. This is orientation, not medical advice: ask your team how closely the trial population matches you.
Shares Naxitamab, High-risk neuroblastoma, Neuroblastoma (paediatric).
Shares High-risk neuroblastoma, Neuroblastoma (paediatric), Rare and paediatric cancers without markets.
Shares High-risk neuroblastoma, Neuroblastoma (paediatric), Rare and paediatric cancers without markets.
Shares High-risk neuroblastoma, Neuroblastoma (paediatric), Rare and paediatric cancers without markets, Monoclonal antibodies.
Shares High-risk neuroblastoma, Neuroblastoma (paediatric), Rare and paediatric cancers without markets.
Shares Y-mAbs Therapeutics, Naxitamab, High-risk neuroblastoma, Neuroblastoma (paediatric).
Shares High-risk neuroblastoma, Neuroblastoma (paediatric), Rare and paediatric cancers without markets.