The U.S. Food and Drug Administration (FDA) has approved dinutuximab (Unituxin), a monoclonal antibody targeting glycolipid GD2, as part of first-line therapy for pediatric patients with high-risk neuroblastoma. A chimeric monoclonal antibody that binds to the surface of neuroblastoma cells, dinutuximab is being approved for use as part of a multimodality regimen, including surgery, chemotherapy, and radiation therapy for patients who have achieved at least a partial response to prior first-line multiagent, multimodality therapy.
“Dinutuximab marks the first approval for a therapy aimed specifically for the treatment of patients with high-risk neuroblastoma,” said Richard Pazdur, MD, Director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “Dinutuximab fulfills a critical need by providing a treatment option that prolongs survival in children with high-risk neuroblastoma.”
The FDA granted dinutuximab priority review and orphan product designation. With this approval, the FDA also issued a rare pediatric disease priority review voucher to United Therapeutics, which confers priority review to a subsequent drug application that would not otherwise qualify for priority review.
The safety and efficacy of dinutuximab were evaluated in a 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 subsequently received bone marrow transplantation support and radiation therapy. Participants were randomly assigned to receive either an oral retinoid drug, isotretinoin, or dinutuximab in combination with interleukin-2 (Proleukin), granulocyte-macrophage colony-stimulating factor, and isotretinoin.
Three years after treatment assignment, 63% of participants receiving the dinutuximab combination were alive and free of tumor growth or recurrence, compared to 46% of participants treated with isotretinoin alone. In an updated analysis of survival, 73% of participants who received the dinutuximab combination were alive compared with 58% of those receiving isotretinoin alone. ■