On November 25, 2020, the FDA granted accelerated approval to naxitamab (Danyelza, Y-mAbs Therapeutics) in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF) for relapsed or refractory high-risk neuroblastoma in the bone or bone barrow.
This indication is for pediatric patients ≥1 year of age and adult patients with neuroblastoma in the bone or bone marrow who demonstrated a partial response, minor response, or stable disease to prior therapy.
The efficacy of naxitamab was evaluated in 2 single-arm, open-label trials: Study 201 and Study 12-230. Patients received 3 mg/kg naxitamab administered as an intravenous infusion on days 1, 3, and 5 of each 4-week cycle in combination with GM-CSF subcutaneously at 250 µg/m2/day on days -4 to 0 and at 500 µg/m2/day on days 1 to 5.
In Study 201, patients were permitted to received pre-planned radiation to the primary disease site. In Study 12-230, patients were permitted to receive therapy to non-target bony lesions or soft tissue disease.
The main efficacy outcome measures were confirmed overall response rate (ORR) and duration of response (DOR).
Among 22 patients treated in the multicenter Study 201, the ORR was 45% and 30% of responders had a DOR greater or equal to 6 months. Among 38 patients treated in the single-center Study 12-230, the ORR was 34% with 23% of patients having a DOR greater or equal to 6 months.
The most common adverse events were infusion-related reactions, pain, tachycardia, vomiting, cough, nausea, diarrhea, decreased appetite, hypertension, fatigue, erythema multiforme, peripheral neuropathy, urticaria, pyrexia, headache, injection site reaction, edema, anxiety, localized edema, and irritability.—Janelle Bradley
