The U.S. Food and Drug Administration (FDA) has approved Unituxin (dinutuximab), part of a first-line therapy for high-risk neuroblastoma.
The new treatment is approved for use in combination with surgery, chemotherapy and radiation therapy for patients who achieve at least a partial response to prior first-line multiagent, multimodality therapy.
Unituxin is only the third drug that has received initial FDA approval for a pediatric cancer in more than 20 years.
Neuroblastoma, a rare cancer that forms from immature nerve cells, typically occurs in children younger than five years of age. Patients with high-risk neuroblastoma have a 40 to 50 percent chance of long-term survival despite aggressive therapy.
“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, in the agency’s news release. “[This drug] fulfills a critical need by providing a treatment option that prolongs survival in children with high-risk neuroblastoma.”
The FDA granted Unituxin priority review and orphan product designation. Unituxin’s approval was based on the demonstration of improved event-free survival (EFS) and overall survival (OS) in a multicenter, open-label, randomized trial.
The safety and efficacy of the drug were evaluated in a clinical trial of 226 pediatric patients 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.
Read the entire news release here.
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Filed Under: Drug Discovery
