The U.S. Food and Drug Administration (FDA) has approved Unituxin (dinutuximab) to be used in combination with interleukin-2 and granulocyte-macrophage colony-stimulating factor (GCSF) for first-line therapy for pediatric patients with high-risk neuroblastoma

Unituxin is an antibody that binds to the surface of neuroblastoma cells. Unituxin is to be used as part of a multimodality regimen for patients who achieved at least a partial response to prior first-line multiagent, multimodality therapy, including surgery, chemotherapy, and radiotherapy.

“Unituxin marks the first approval for a therapy aimed specifically for the treatment of patients with high-risk neuroblastoma,” said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research.

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“Unituxin fulfills a critical need by providing a treatment option that prolongs survival in children with high-risk neuroblastoma.”

Approval is based on a study that included 226 pediatric participants with high-risk neuroblastoma whose tumors shrunk or disappeared after receiving multiagent chemotherapy and surgery followed by additional intensive chemotherapy, bone marrow transplantation support, and radiotherapy.

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In the study, patients were randomly assigned to receive isotretinoin or Unituxin in combination with interleukin-2 and GCSF. Results showed that at 3 years, 63% of those who received Unituxin were alive and free of tumor growth or recurrence versus 46% of those treated with isotetinoin.

The most common adverse events associated with Unituxin treatment were severe pain, fever, thrombocytopenia, infusion reactions, hypotension, hyponatremia, elevated liver enzymes, anemia, diarrhea, hypokalemia, capillary leak syndrome, neutropenia, lymphopenia, hives, and hypocalcemia.

Unituxin carries a Boxed Warning alerting health care professionals and patients that Unituxin can cause severe pain that requires treatment with IV narcotics and can also cause life-threatening infusion reactions and nerve damage.

According to the National Cancer Institute, there are an estimated 650 new cases of neuroblastoma diagnoses in the United States each year, and patients with high-risk disease only have a 40-50% chance of long-term survival despite receiving aggressive treatment.