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. Naxitamab-gqgk is a humanized, monoclonal antibody that targets ganglioside GD2 which is over expressed on neuroblastoma cells and other cells of neuroectodermal origin, including the CNS and peripheral nerves.
The FDA approval was based on results from two small non comparative clinical studies in patients with relapsed or refractory neuroblastoma in the bone or bone marrow. Combined the two trials treated 60 patients and 34% to 45% responded to treatment, 23% to 30% of responders had response duration greater or equal to 6 months.
In addition to producing low blood counts as a side effect of treatment over 25% of patients experienced infusion-related reactions, nausea and vomiting, diarrhea, decreased appetite, hypertension, fatigue, erythema multiforme, peripheral neuropathy, itching, fever, injection site reactions, localized edema, and irritability. Danyelza carries a Boxed Warning for serious infusion-related reactions and neurotoxicity, including severe neuropathic pain, transverse myelitis and reversible posterior leukoencephalopathy syndrome. For more information visit ymabs.com.
- FDA grants accelerated approval to naxitamab for high-risk neuroblastoma in bone or bone marrow. [press release]. Silver Spring, MD: US Food and Drug Administration; November 25, 2020.
- FDA approves Y-mAbs’ Danyelza® (naxitamab-gqgk) for the treatment of neuroblastoma. [press release]. New York, NY: Y-mAbs Therapeutics; November 25, 2020.
- Danyelza® [package insert]. New York, NY: Y-mAbs Therapeutics; 2020.