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FDA approves Unituxin for pediatric patients with high-risk neuroblastoma
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The FDA today announced the approval of dinutuximab for the treatment of pediatric patients with high-risk neuroblastoma, according to a press release from the drug’s manufacturer.
Dinutuximab (Unituxin, United Therapeutics) is an antibody designed to bond to the surface of neuroblastoma cells and is approved as part of a multimodality regimen, including surgery, chemotherapy and radiation therapy for pediatric patients who achieved at least a partial response to prior first-line multi-agent, multimodality therapy.
Richard Pazdur
“Unituxin marks the first approval for a therapy aimed specifically for the treatment of patients with high-risk neuroblastoma,” Richard Pazdur, MD, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, said in a press release. “Unituxin fulfills a critical need by providing a treatment option that prolongs survival in children with high-risk neuroblastoma.”
Besides FDA approval of dinutuximab, United Therapeutics also received a rare pediatric disease priority review voucher for this therapy, marking the second rare pediatric disease priority review voucher granted by the FDA since inception of this program.
Dinutuximab was evaluated in a randomized clinical trial of 226 pediatric participants with high-risk neuroblastoma whose tumors shrunk or disappeared after treatment with multiple-drug chemotherapy and surgery followed by additional intensive chemotherapy and who later received bone marrow transplantation support and radiation therapy.
Participants received either an oral retinoid drug, isotretinoin, or dinutuximab combined with interleukin-2 and granulocyte-macrophage colony–stimulating factor, which are thought to increase the activity of dinutuximab by stimulating the immune system and isotretinoin.
Three years after starting treatment, 63% of participants receiving the dinutuximab combination were alive and free of tumor growth or recurrence vs. 46% of participants treated with isotretinoin alone. In an updated analysis of survival, 73% of participants in the dinutuximab combination group were alive vs. 58% of patients who received isotretinoin alone.
Dinutuximab carries a boxed warning for irritating nerve cells, which causes severe pain that involves treatment with IV narcotics. Dinutuximab also can cause nerve damage and life-threatening infusion reactions, including upper airway swelling, difficulty breathing and low blood pressure, during or shortly after completion of the infusion.
Other potentially serious adverse effects associated with dinutuximab include infections, eye problems, electrolyte abnormalities and bone marrow suppression.
The most common adverse effects reported by patients who received dinutuximab included severe pain, fever, low platelet counts, infusion reactions, low BP, low levels of salt in the blood (hyponatremia), elevated liver enzymes, anemia, vomiting, diarrhea, low potassium levels in the blood, capillary leak syndrome, neutropenia, lymphopenia, hives and low blood calcium levels.