FDA Granted Accelerated Approval to Danyelza for Neuroblastoma


On November 25, 2020, the United States Food and Drug Administration (FDA) granted accelerated approval to Danyelza (naxitamab) administered in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF) for the treatment of relapsed or refractory high-risk neuroblastoma in the bone or bone barrow.

Neuroblastoma is a type of cancer that develops in the nerve tissues of the adrenal gland, neck, chest, or spinal cord. Although neuroblastoma is rare (it affects roughly 650 children and adolescents in the United States each year), it is the most common cancer diagnosed in infants. Based on the age of the patient and the specific characteristics of the cancer, neuroblastoma is classified as low-, intermediate-, or high-risk. Patients with high-risk neuroblastoma typically receive high doses of chemotherapy as part of their treatment.

Danyelza is a type of immunotherapy drug known as a monoclonal antibody. It works by helping the body’s immune system to find and destroy cancer cells.

The FDA approval was based on patients treated in two multicenter studies. In the first study 22 patients had an overall response rate of 45% and 30% of responders had a duration of response of greater or equal to 6 months. The second trial yielded similar results; 34% of 38 patients responded to treatment and 23% of patients experienced a response duration of greater or equal to 6 months.

The most common side effects 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.