The U.S. Food and Drug Administration (FDA) has approved Unituxin (dinutuximab) to be used as a first-line therapy for babies with a rare form of pediatric cancer.
The drug has been approved for the treatment of high-risk neuroblastoma, which often occurs in young children, the FDA reported. The cancer forms from immature cell nerves and usually begins in the adrenal glands, but can also be seen in the abdomen, chest or nerve tissue near the spine. It occurs in one out of 100,000 children, and there are about 650 new cases diagnosed in the U.S. every year.
"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. "Unituxin fulfills a critical need by providing a treatment option that prolongs survival in children with high-risk neuroblastoma."
The promising drug binds to the surface of neuroblastoma cells, and is being approved as part of a multimodality regimen that also includes treatment methods such as surgery and chemotherapy.
The safety and efficacy of the drug was tested in a clinical trial of 226 pediatric participants suffering from high-risk neuroblastoma whose tumors shrank or disappeared "after treatment with multiple-drug chemotherapy and surgery followed by additional intensive chemotherapy and who subsequently received bone marrow transplantation support and radiation therapy," the FDA stated.
The participants were randomly assigned to receive either an oral retinoid drug, isotretinoin (RA), or Unituxin in combination with interleukin-2 and granulocyte-macrophage colony-stimulating factor. After three years, 63 percent of the patients who received the Unituxin combination were alive and tumor-free, compared with only 46 percent of those who were treated with RA alone.
The drug causes severe pain by irritating nerve cells, requiring the patient to be treated with intravenous narcotics. It can also cause "nerve damage and life-threatening infusion reactions, including upper airway swelling, difficulty breathing, and low blood pressure, during or shortly following completion of the infusion. Unituxin may also cause other serious side effects including infections, eye problems, electrolyte abnormalities and bone marrow suppression," the FDA stated.
